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Artificial Intelligence Helps Diagnose Lung Disease in Infants and Outperforms Trainee Doctors
VIENNA — Artificial Intelligence (AI) can assist doctors in assessing and diagnosing respiratory illnesses in infants and children, according to two new studies presented at the European Respiratory Society (ERS) 2024 Congress.
Researchers can train artificial neural networks (ANNs) to detect lung disease in premature babies by analyzing their breathing patterns while they sleep. “Our noninvasive test is less distressing for the baby and their parents, meaning they can access treatment more quickly, and may also be relevant for their long-term prognosis,” said Edgar Delgado-Eckert, PhD, adjunct professor in the Department of Biomedical Engineering at The University of Basel, Switzerland, and a research group leader at the University Children’s Hospital, Switzerland.
Manjith Narayanan, MD, a consultant in pediatric pulmonology at the Royal Hospital for Children and Young People, Edinburgh, and honorary senior clinical lecturer at The University of Edinburgh, United Kingdom, said chatbots such as ChatGPT, Bard, and Bing can perform as well as or better than trainee doctors when assessing children with respiratory issues. He said chatbots could triage patients more quickly and ease pressure on health services.
Chatbots Show Promise in Triage of Pediatric Respiratory Illnesses
Researchers at The University of Edinburgh provided 10 trainee doctors with less than 4 months of clinical experience in pediatrics with clinical scenarios that covered topics such as cystic fibrosis, asthma, sleep-disordered breathing, breathlessness, chest infections, or no obvious diagnosis.
The trainee doctors had 1 hour to use the internet, although they were not allowed to use chatbots to solve each scenario with a descriptive answer.
Each scenario was also presented to the three large language models (LLMs): OpenAI’s ChatGPT, Google’s Bard, and Microsoft’s Bing.
Six pediatric respiratory experts assessed all responses, scoring correctness, comprehensiveness, usefulness, plausibility, and coherence on a scale of 0-9. They were also asked to say whether they thought a human or a chatbot generated each response.
ChatGPT scored an average of 7 out of 9 overall and was believed to be more human-like than responses from the other chatbots. Bard scored an average of 6 out of 9 and was more “coherent” than trainee doctors, but in other respects, it was no better or worse than trainee doctors. Bing and trainee doctors scored an average of 4 out of 9.
“Our study is the first, to our knowledge, to test LLMs against trainee doctors in situations that reflect real-life clinical practice,” Narayanan said. “We did this by allowing the trainee doctors to have full access to resources available on the internet, as they would in real life. This moves the focus away from testing memory, where LLMs have a clear advantage.”
Narayanan said that these models could help nurses, trainee doctors, and primary care physicians triage patients quickly and assist medical professionals in their studies by summarizing their thought processes. “The key word, though, is “assist.” They cannot replace conventional medical training yet,” he told Medscape Medical News.
The researchers found no obvious hallucinations — seemingly made-up information — with any of the three LLMs. Still, Narayanan said, “We need to be aware of this possibility and build mitigations.”
Hilary Pinnock, ERS education council chair and professor of primary care respiratory medicine at The University of Edinburgh who was not involved in the research, said seeing how widely available AI tools can provide solutions to complex cases of respiratory illness in children is exciting and worrying at the same time. “It certainly points the way to a brave new world of AI-supported care.”
“However, before we start to use AI in routine clinical practice, we need to be confident that it will not create errors either through ‘hallucinating’ fake information or because it has been trained on data that does not equitably represent the population we serve,” she said.
AI Predicts Lung Disease in Premature Babies
Identifying bronchopulmonary dysplasia (BPD) in premature babies remains a challenge. Lung function tests usually require blowing out on request, which is a task babies cannot perform. Current techniques require sophisticated equipment to measure an infant’s lung ventilation characteristics, so doctors usually diagnose BPD by the presence of its leading causes, prematurity and the need for respiratory support.
Researchers at the University of Basel in Switzerland trained an ANN model to predict BPD in premature babies.
The team studied a group of 139 full-term and 190 premature infants who had been assessed for BPD, recording their breathing for 10 minutes while they slept. For each baby, 100 consecutive regular breaths, carefully inspected to exclude sighs or other artifacts, were used to train, validate, and test an ANN called a Long Short-Term Memory model (LSTM), which is particularly effective at classifying sequential data such as tidal breathing.
Researchers used 60% of the data to teach the network how to recognize BPD, 20% to validate the model, and then fed the remaining 20% of the data to the model to see if it could correctly identify those babies with BPD.
The LSTM model classified a series of flow values in the unseen test data set as belonging to a patient diagnosed with BPD or not with 96% accuracy.
“Until recently, this need for large amounts of data has hindered efforts to create accurate models for lung disease in infants because it is so difficult to assess their lung function,” Delgado-Eckert said. “Our research delivers, for the first time, a comprehensive way of analyzing infants’ breathing and allows us to detect which babies have BPD as early as 1 month of corrected age.”
The study presented by Delgado-Eckert received funding from the Swiss National Science Foundation. Narayanan and Pinnock reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — Artificial Intelligence (AI) can assist doctors in assessing and diagnosing respiratory illnesses in infants and children, according to two new studies presented at the European Respiratory Society (ERS) 2024 Congress.
Researchers can train artificial neural networks (ANNs) to detect lung disease in premature babies by analyzing their breathing patterns while they sleep. “Our noninvasive test is less distressing for the baby and their parents, meaning they can access treatment more quickly, and may also be relevant for their long-term prognosis,” said Edgar Delgado-Eckert, PhD, adjunct professor in the Department of Biomedical Engineering at The University of Basel, Switzerland, and a research group leader at the University Children’s Hospital, Switzerland.
Manjith Narayanan, MD, a consultant in pediatric pulmonology at the Royal Hospital for Children and Young People, Edinburgh, and honorary senior clinical lecturer at The University of Edinburgh, United Kingdom, said chatbots such as ChatGPT, Bard, and Bing can perform as well as or better than trainee doctors when assessing children with respiratory issues. He said chatbots could triage patients more quickly and ease pressure on health services.
Chatbots Show Promise in Triage of Pediatric Respiratory Illnesses
Researchers at The University of Edinburgh provided 10 trainee doctors with less than 4 months of clinical experience in pediatrics with clinical scenarios that covered topics such as cystic fibrosis, asthma, sleep-disordered breathing, breathlessness, chest infections, or no obvious diagnosis.
The trainee doctors had 1 hour to use the internet, although they were not allowed to use chatbots to solve each scenario with a descriptive answer.
Each scenario was also presented to the three large language models (LLMs): OpenAI’s ChatGPT, Google’s Bard, and Microsoft’s Bing.
Six pediatric respiratory experts assessed all responses, scoring correctness, comprehensiveness, usefulness, plausibility, and coherence on a scale of 0-9. They were also asked to say whether they thought a human or a chatbot generated each response.
ChatGPT scored an average of 7 out of 9 overall and was believed to be more human-like than responses from the other chatbots. Bard scored an average of 6 out of 9 and was more “coherent” than trainee doctors, but in other respects, it was no better or worse than trainee doctors. Bing and trainee doctors scored an average of 4 out of 9.
“Our study is the first, to our knowledge, to test LLMs against trainee doctors in situations that reflect real-life clinical practice,” Narayanan said. “We did this by allowing the trainee doctors to have full access to resources available on the internet, as they would in real life. This moves the focus away from testing memory, where LLMs have a clear advantage.”
Narayanan said that these models could help nurses, trainee doctors, and primary care physicians triage patients quickly and assist medical professionals in their studies by summarizing their thought processes. “The key word, though, is “assist.” They cannot replace conventional medical training yet,” he told Medscape Medical News.
The researchers found no obvious hallucinations — seemingly made-up information — with any of the three LLMs. Still, Narayanan said, “We need to be aware of this possibility and build mitigations.”
Hilary Pinnock, ERS education council chair and professor of primary care respiratory medicine at The University of Edinburgh who was not involved in the research, said seeing how widely available AI tools can provide solutions to complex cases of respiratory illness in children is exciting and worrying at the same time. “It certainly points the way to a brave new world of AI-supported care.”
“However, before we start to use AI in routine clinical practice, we need to be confident that it will not create errors either through ‘hallucinating’ fake information or because it has been trained on data that does not equitably represent the population we serve,” she said.
AI Predicts Lung Disease in Premature Babies
Identifying bronchopulmonary dysplasia (BPD) in premature babies remains a challenge. Lung function tests usually require blowing out on request, which is a task babies cannot perform. Current techniques require sophisticated equipment to measure an infant’s lung ventilation characteristics, so doctors usually diagnose BPD by the presence of its leading causes, prematurity and the need for respiratory support.
Researchers at the University of Basel in Switzerland trained an ANN model to predict BPD in premature babies.
The team studied a group of 139 full-term and 190 premature infants who had been assessed for BPD, recording their breathing for 10 minutes while they slept. For each baby, 100 consecutive regular breaths, carefully inspected to exclude sighs or other artifacts, were used to train, validate, and test an ANN called a Long Short-Term Memory model (LSTM), which is particularly effective at classifying sequential data such as tidal breathing.
Researchers used 60% of the data to teach the network how to recognize BPD, 20% to validate the model, and then fed the remaining 20% of the data to the model to see if it could correctly identify those babies with BPD.
The LSTM model classified a series of flow values in the unseen test data set as belonging to a patient diagnosed with BPD or not with 96% accuracy.
“Until recently, this need for large amounts of data has hindered efforts to create accurate models for lung disease in infants because it is so difficult to assess their lung function,” Delgado-Eckert said. “Our research delivers, for the first time, a comprehensive way of analyzing infants’ breathing and allows us to detect which babies have BPD as early as 1 month of corrected age.”
The study presented by Delgado-Eckert received funding from the Swiss National Science Foundation. Narayanan and Pinnock reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — Artificial Intelligence (AI) can assist doctors in assessing and diagnosing respiratory illnesses in infants and children, according to two new studies presented at the European Respiratory Society (ERS) 2024 Congress.
Researchers can train artificial neural networks (ANNs) to detect lung disease in premature babies by analyzing their breathing patterns while they sleep. “Our noninvasive test is less distressing for the baby and their parents, meaning they can access treatment more quickly, and may also be relevant for their long-term prognosis,” said Edgar Delgado-Eckert, PhD, adjunct professor in the Department of Biomedical Engineering at The University of Basel, Switzerland, and a research group leader at the University Children’s Hospital, Switzerland.
Manjith Narayanan, MD, a consultant in pediatric pulmonology at the Royal Hospital for Children and Young People, Edinburgh, and honorary senior clinical lecturer at The University of Edinburgh, United Kingdom, said chatbots such as ChatGPT, Bard, and Bing can perform as well as or better than trainee doctors when assessing children with respiratory issues. He said chatbots could triage patients more quickly and ease pressure on health services.
Chatbots Show Promise in Triage of Pediatric Respiratory Illnesses
Researchers at The University of Edinburgh provided 10 trainee doctors with less than 4 months of clinical experience in pediatrics with clinical scenarios that covered topics such as cystic fibrosis, asthma, sleep-disordered breathing, breathlessness, chest infections, or no obvious diagnosis.
The trainee doctors had 1 hour to use the internet, although they were not allowed to use chatbots to solve each scenario with a descriptive answer.
Each scenario was also presented to the three large language models (LLMs): OpenAI’s ChatGPT, Google’s Bard, and Microsoft’s Bing.
Six pediatric respiratory experts assessed all responses, scoring correctness, comprehensiveness, usefulness, plausibility, and coherence on a scale of 0-9. They were also asked to say whether they thought a human or a chatbot generated each response.
ChatGPT scored an average of 7 out of 9 overall and was believed to be more human-like than responses from the other chatbots. Bard scored an average of 6 out of 9 and was more “coherent” than trainee doctors, but in other respects, it was no better or worse than trainee doctors. Bing and trainee doctors scored an average of 4 out of 9.
“Our study is the first, to our knowledge, to test LLMs against trainee doctors in situations that reflect real-life clinical practice,” Narayanan said. “We did this by allowing the trainee doctors to have full access to resources available on the internet, as they would in real life. This moves the focus away from testing memory, where LLMs have a clear advantage.”
Narayanan said that these models could help nurses, trainee doctors, and primary care physicians triage patients quickly and assist medical professionals in their studies by summarizing their thought processes. “The key word, though, is “assist.” They cannot replace conventional medical training yet,” he told Medscape Medical News.
The researchers found no obvious hallucinations — seemingly made-up information — with any of the three LLMs. Still, Narayanan said, “We need to be aware of this possibility and build mitigations.”
Hilary Pinnock, ERS education council chair and professor of primary care respiratory medicine at The University of Edinburgh who was not involved in the research, said seeing how widely available AI tools can provide solutions to complex cases of respiratory illness in children is exciting and worrying at the same time. “It certainly points the way to a brave new world of AI-supported care.”
“However, before we start to use AI in routine clinical practice, we need to be confident that it will not create errors either through ‘hallucinating’ fake information or because it has been trained on data that does not equitably represent the population we serve,” she said.
AI Predicts Lung Disease in Premature Babies
Identifying bronchopulmonary dysplasia (BPD) in premature babies remains a challenge. Lung function tests usually require blowing out on request, which is a task babies cannot perform. Current techniques require sophisticated equipment to measure an infant’s lung ventilation characteristics, so doctors usually diagnose BPD by the presence of its leading causes, prematurity and the need for respiratory support.
Researchers at the University of Basel in Switzerland trained an ANN model to predict BPD in premature babies.
The team studied a group of 139 full-term and 190 premature infants who had been assessed for BPD, recording their breathing for 10 minutes while they slept. For each baby, 100 consecutive regular breaths, carefully inspected to exclude sighs or other artifacts, were used to train, validate, and test an ANN called a Long Short-Term Memory model (LSTM), which is particularly effective at classifying sequential data such as tidal breathing.
Researchers used 60% of the data to teach the network how to recognize BPD, 20% to validate the model, and then fed the remaining 20% of the data to the model to see if it could correctly identify those babies with BPD.
The LSTM model classified a series of flow values in the unseen test data set as belonging to a patient diagnosed with BPD or not with 96% accuracy.
“Until recently, this need for large amounts of data has hindered efforts to create accurate models for lung disease in infants because it is so difficult to assess their lung function,” Delgado-Eckert said. “Our research delivers, for the first time, a comprehensive way of analyzing infants’ breathing and allows us to detect which babies have BPD as early as 1 month of corrected age.”
The study presented by Delgado-Eckert received funding from the Swiss National Science Foundation. Narayanan and Pinnock reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
FROM ERS 2024
Is Wildfire Smoke More Toxic Than General Air Pollution?
Wildfire-related air pollution in Europe kills more than non-wildfire air pollution. As climate change exacerbates the frequency and violence of wildfires, researchers are studying the health implications of mitigation methods such as prescribed fires.
Presenting at the annual congress of the European Respiratory Society (ERS), Cathryn Tonne, PhD, an environmental epidemiologist at the Instituto de Salud Global de Barcelona, Spain, said wildfire-related PM2.5 is more toxic than general PM2.5, leading to significantly higher mortality rates.
Prescribed, controlled fires have been employed worldwide to reduce the chance of uncontrolled, catastrophic fires. However, researchers wonder whether the techniques reduce the overall fire-related PM2.5 or add up to it. “Prescribed fire increases ecosystem resilience and can reduce the risk of catastrophic wildfire,” said Jason Sacks, MPH, an epidemiologist in the Center for Public Health and Environmental Assessment in the Office of Research and Development at the Environmental Protection Agency (EPA), at the congress. “But it also leads to poorer air quality and health impacts, and we still don’t know what this means at a regional scale.”
Wildfire Pollution Kills More Than Other Air Pollution
Researchers at the Instituto de Salud Global de Barcelona used a large dataset of daily mortality data from 32 European countries collected through the EARLY-ADAPT project. They utilized the SILAM model to derive daily average concentrations of wildfire-related PM2.5, non-fire PM2.5, and total PM2.5 levels. They also employed GEOSTAT population grids at a 1-km resolution to calculate the attributable number of deaths across different regions, specifically focusing on data from 2006, 2011, and 2018.
The data analysis indicated that the relative risk per unit of PM2.5 is substantially larger for wildfire-related PM2.5, compared with non-fire PM2.5. “We essentially assume that wildfire smoke PM2.5 has the same toxicity as total PM2.5, but it’s increasingly clear that’s likely not the case,” Dr. Tonne said, presenting the study.
When employing exposure-response functions (ERFs) specific to wildfire smoke, researchers found that the attributable deaths from all causes of wildfire PM2.5 were approximately 10 times larger than those calculated using total PM2.5 exposure estimates. Dr. Tonne explained that this stark difference highlights the critical need for tailored ERFs that accurately reflect the unique health risks posed by wildfire smoke.
“Respiratory mortality usually has the strongest relative risks, and we’re seeing that in this study as well,” Dr. Tonne said. “Wildfire smoke seems to operate through quite immediate mechanisms, likely through inflammation and oxidative stress.”
One significant challenge of the study was the lack of uniform spatial resolution across all countries involved in the analysis. This inconsistency may affect how accurately mortality estimates can be attributed to specific PM2.5 sources. Additionally, the study had limited statistical power for generating age- and sex-specific mortality estimates, which could obscure important demographic differences in vulnerability to wildfire smoke exposure. The analysis was also constrained to data available only up to 2020, thereby excluding critical wildfire events from subsequent years, such as those in 2022 and 2023, which may have further elucidated the health impacts of wildfire smoke in Europe.
Fires Prescription
Prescribed fires or controlled burns are intentional fires set by land managers under carefully managed conditions.
Historically, many forested areas have been subjected to fire suppression practices, which allow combustible materials like dry leaves, twigs, and shrubs to accumulate over time. This buildup leads to a higher likelihood of severe, uncontrollable wildfires. Prescribed fires can reduce these fuel loads and improve the health and resilience of ecosystems.
They release fewer pollutants and emissions than the large-scale, unmanageable wildfires they help prevent because they happen at lower temperatures. But they still introduce pollutants in the air that can negatively affect nearby communities’ health.
People with preexisting respiratory conditions, such as asthma or chronic obstructive pulmonary disease (COPD), are particularly vulnerable to smoke, which can trigger health issues like breathing difficulties, coughing, and eye irritation. The cumulative impact of increased burns raises concerns about long-term air quality, especially in densely populated areas. “We need to understand if we’re actually tipping the scale to having less wildfire smoke or just increasing the total amount of smoke.”
Mitigation strategies include accurately picking the right timing and weather conditions to determine when and where to conduct controlled burns and effective and timely communication to inform local communities about upcoming burns, the potential for smoke exposure, and how to protect themselves.
There is a growing need to improve public messaging around prescribed fires, Mr. Sacks said, because often the message communicated is oversimplified, such as “there will be smoke, but don’t worry. But that’s not the message we want to convey, especially for people with asthma or COPD.”
Instead, he said public health agencies should provide clearer, science-based guidance on the risks for smoke exposure and practical steps people can take to reduce their risk.
What Can Doctors Do?
Chris Carlsten, MD, director of the Centre for Lung Health and professor and head of the Respiratory Medicine Division at the University of British Columbia, Vancouver, Canada, told this news organization that determining whether an exacerbation of a respiratory condition is caused by fire exposure or other factors, such as viral infections, is complex because both can trigger similar responses and may complement each other. “It’s very difficult for any individual to know whether, when they’re having an exacerbation of asthma or COPD, that’s due to the fire,” he said. Fire smoke also increases infection risks, further complicating diagnosis.
Dr. Carlsten suggested that physicians could recommend preventative use of inhalers for at-risk patients when wildfires occur rather than waiting for symptoms to worsen. “That is a really interesting idea that could be practical.” Still, he advises caution, stressing that patients should consult their providers because not all may react well to increased inhaler use.
He also highlighted a significant shift in the healthcare landscape, noting that traditionally, the focus has been on the cardiovascular impacts of pollution, particularly traffic-related pollution. However, as wildfire smoke becomes a growing issue, the focus is shifting back to respiratory problems, with profound implications for healthcare resources, budgets, and drug approvals based on the burden of respiratory disease. “Fire smoke is becoming more of a problem. This swing back to respiratory has huge implications for healthcare systems and respiratory disease burden.”
Mr. Sacks and Dr. Carlsten reported no relevant financial relationships. The study presented by Dr. Tonne received funding from the European Union’s Horizon Europe research and innovation programme under Grant Agreement No. 101057131.
A version of this article first appeared on Medscape.com.
Wildfire-related air pollution in Europe kills more than non-wildfire air pollution. As climate change exacerbates the frequency and violence of wildfires, researchers are studying the health implications of mitigation methods such as prescribed fires.
Presenting at the annual congress of the European Respiratory Society (ERS), Cathryn Tonne, PhD, an environmental epidemiologist at the Instituto de Salud Global de Barcelona, Spain, said wildfire-related PM2.5 is more toxic than general PM2.5, leading to significantly higher mortality rates.
Prescribed, controlled fires have been employed worldwide to reduce the chance of uncontrolled, catastrophic fires. However, researchers wonder whether the techniques reduce the overall fire-related PM2.5 or add up to it. “Prescribed fire increases ecosystem resilience and can reduce the risk of catastrophic wildfire,” said Jason Sacks, MPH, an epidemiologist in the Center for Public Health and Environmental Assessment in the Office of Research and Development at the Environmental Protection Agency (EPA), at the congress. “But it also leads to poorer air quality and health impacts, and we still don’t know what this means at a regional scale.”
Wildfire Pollution Kills More Than Other Air Pollution
Researchers at the Instituto de Salud Global de Barcelona used a large dataset of daily mortality data from 32 European countries collected through the EARLY-ADAPT project. They utilized the SILAM model to derive daily average concentrations of wildfire-related PM2.5, non-fire PM2.5, and total PM2.5 levels. They also employed GEOSTAT population grids at a 1-km resolution to calculate the attributable number of deaths across different regions, specifically focusing on data from 2006, 2011, and 2018.
The data analysis indicated that the relative risk per unit of PM2.5 is substantially larger for wildfire-related PM2.5, compared with non-fire PM2.5. “We essentially assume that wildfire smoke PM2.5 has the same toxicity as total PM2.5, but it’s increasingly clear that’s likely not the case,” Dr. Tonne said, presenting the study.
When employing exposure-response functions (ERFs) specific to wildfire smoke, researchers found that the attributable deaths from all causes of wildfire PM2.5 were approximately 10 times larger than those calculated using total PM2.5 exposure estimates. Dr. Tonne explained that this stark difference highlights the critical need for tailored ERFs that accurately reflect the unique health risks posed by wildfire smoke.
“Respiratory mortality usually has the strongest relative risks, and we’re seeing that in this study as well,” Dr. Tonne said. “Wildfire smoke seems to operate through quite immediate mechanisms, likely through inflammation and oxidative stress.”
One significant challenge of the study was the lack of uniform spatial resolution across all countries involved in the analysis. This inconsistency may affect how accurately mortality estimates can be attributed to specific PM2.5 sources. Additionally, the study had limited statistical power for generating age- and sex-specific mortality estimates, which could obscure important demographic differences in vulnerability to wildfire smoke exposure. The analysis was also constrained to data available only up to 2020, thereby excluding critical wildfire events from subsequent years, such as those in 2022 and 2023, which may have further elucidated the health impacts of wildfire smoke in Europe.
Fires Prescription
Prescribed fires or controlled burns are intentional fires set by land managers under carefully managed conditions.
Historically, many forested areas have been subjected to fire suppression practices, which allow combustible materials like dry leaves, twigs, and shrubs to accumulate over time. This buildup leads to a higher likelihood of severe, uncontrollable wildfires. Prescribed fires can reduce these fuel loads and improve the health and resilience of ecosystems.
They release fewer pollutants and emissions than the large-scale, unmanageable wildfires they help prevent because they happen at lower temperatures. But they still introduce pollutants in the air that can negatively affect nearby communities’ health.
People with preexisting respiratory conditions, such as asthma or chronic obstructive pulmonary disease (COPD), are particularly vulnerable to smoke, which can trigger health issues like breathing difficulties, coughing, and eye irritation. The cumulative impact of increased burns raises concerns about long-term air quality, especially in densely populated areas. “We need to understand if we’re actually tipping the scale to having less wildfire smoke or just increasing the total amount of smoke.”
Mitigation strategies include accurately picking the right timing and weather conditions to determine when and where to conduct controlled burns and effective and timely communication to inform local communities about upcoming burns, the potential for smoke exposure, and how to protect themselves.
There is a growing need to improve public messaging around prescribed fires, Mr. Sacks said, because often the message communicated is oversimplified, such as “there will be smoke, but don’t worry. But that’s not the message we want to convey, especially for people with asthma or COPD.”
Instead, he said public health agencies should provide clearer, science-based guidance on the risks for smoke exposure and practical steps people can take to reduce their risk.
What Can Doctors Do?
Chris Carlsten, MD, director of the Centre for Lung Health and professor and head of the Respiratory Medicine Division at the University of British Columbia, Vancouver, Canada, told this news organization that determining whether an exacerbation of a respiratory condition is caused by fire exposure or other factors, such as viral infections, is complex because both can trigger similar responses and may complement each other. “It’s very difficult for any individual to know whether, when they’re having an exacerbation of asthma or COPD, that’s due to the fire,” he said. Fire smoke also increases infection risks, further complicating diagnosis.
Dr. Carlsten suggested that physicians could recommend preventative use of inhalers for at-risk patients when wildfires occur rather than waiting for symptoms to worsen. “That is a really interesting idea that could be practical.” Still, he advises caution, stressing that patients should consult their providers because not all may react well to increased inhaler use.
He also highlighted a significant shift in the healthcare landscape, noting that traditionally, the focus has been on the cardiovascular impacts of pollution, particularly traffic-related pollution. However, as wildfire smoke becomes a growing issue, the focus is shifting back to respiratory problems, with profound implications for healthcare resources, budgets, and drug approvals based on the burden of respiratory disease. “Fire smoke is becoming more of a problem. This swing back to respiratory has huge implications for healthcare systems and respiratory disease burden.”
Mr. Sacks and Dr. Carlsten reported no relevant financial relationships. The study presented by Dr. Tonne received funding from the European Union’s Horizon Europe research and innovation programme under Grant Agreement No. 101057131.
A version of this article first appeared on Medscape.com.
Wildfire-related air pollution in Europe kills more than non-wildfire air pollution. As climate change exacerbates the frequency and violence of wildfires, researchers are studying the health implications of mitigation methods such as prescribed fires.
Presenting at the annual congress of the European Respiratory Society (ERS), Cathryn Tonne, PhD, an environmental epidemiologist at the Instituto de Salud Global de Barcelona, Spain, said wildfire-related PM2.5 is more toxic than general PM2.5, leading to significantly higher mortality rates.
Prescribed, controlled fires have been employed worldwide to reduce the chance of uncontrolled, catastrophic fires. However, researchers wonder whether the techniques reduce the overall fire-related PM2.5 or add up to it. “Prescribed fire increases ecosystem resilience and can reduce the risk of catastrophic wildfire,” said Jason Sacks, MPH, an epidemiologist in the Center for Public Health and Environmental Assessment in the Office of Research and Development at the Environmental Protection Agency (EPA), at the congress. “But it also leads to poorer air quality and health impacts, and we still don’t know what this means at a regional scale.”
Wildfire Pollution Kills More Than Other Air Pollution
Researchers at the Instituto de Salud Global de Barcelona used a large dataset of daily mortality data from 32 European countries collected through the EARLY-ADAPT project. They utilized the SILAM model to derive daily average concentrations of wildfire-related PM2.5, non-fire PM2.5, and total PM2.5 levels. They also employed GEOSTAT population grids at a 1-km resolution to calculate the attributable number of deaths across different regions, specifically focusing on data from 2006, 2011, and 2018.
The data analysis indicated that the relative risk per unit of PM2.5 is substantially larger for wildfire-related PM2.5, compared with non-fire PM2.5. “We essentially assume that wildfire smoke PM2.5 has the same toxicity as total PM2.5, but it’s increasingly clear that’s likely not the case,” Dr. Tonne said, presenting the study.
When employing exposure-response functions (ERFs) specific to wildfire smoke, researchers found that the attributable deaths from all causes of wildfire PM2.5 were approximately 10 times larger than those calculated using total PM2.5 exposure estimates. Dr. Tonne explained that this stark difference highlights the critical need for tailored ERFs that accurately reflect the unique health risks posed by wildfire smoke.
“Respiratory mortality usually has the strongest relative risks, and we’re seeing that in this study as well,” Dr. Tonne said. “Wildfire smoke seems to operate through quite immediate mechanisms, likely through inflammation and oxidative stress.”
One significant challenge of the study was the lack of uniform spatial resolution across all countries involved in the analysis. This inconsistency may affect how accurately mortality estimates can be attributed to specific PM2.5 sources. Additionally, the study had limited statistical power for generating age- and sex-specific mortality estimates, which could obscure important demographic differences in vulnerability to wildfire smoke exposure. The analysis was also constrained to data available only up to 2020, thereby excluding critical wildfire events from subsequent years, such as those in 2022 and 2023, which may have further elucidated the health impacts of wildfire smoke in Europe.
Fires Prescription
Prescribed fires or controlled burns are intentional fires set by land managers under carefully managed conditions.
Historically, many forested areas have been subjected to fire suppression practices, which allow combustible materials like dry leaves, twigs, and shrubs to accumulate over time. This buildup leads to a higher likelihood of severe, uncontrollable wildfires. Prescribed fires can reduce these fuel loads and improve the health and resilience of ecosystems.
They release fewer pollutants and emissions than the large-scale, unmanageable wildfires they help prevent because they happen at lower temperatures. But they still introduce pollutants in the air that can negatively affect nearby communities’ health.
People with preexisting respiratory conditions, such as asthma or chronic obstructive pulmonary disease (COPD), are particularly vulnerable to smoke, which can trigger health issues like breathing difficulties, coughing, and eye irritation. The cumulative impact of increased burns raises concerns about long-term air quality, especially in densely populated areas. “We need to understand if we’re actually tipping the scale to having less wildfire smoke or just increasing the total amount of smoke.”
Mitigation strategies include accurately picking the right timing and weather conditions to determine when and where to conduct controlled burns and effective and timely communication to inform local communities about upcoming burns, the potential for smoke exposure, and how to protect themselves.
There is a growing need to improve public messaging around prescribed fires, Mr. Sacks said, because often the message communicated is oversimplified, such as “there will be smoke, but don’t worry. But that’s not the message we want to convey, especially for people with asthma or COPD.”
Instead, he said public health agencies should provide clearer, science-based guidance on the risks for smoke exposure and practical steps people can take to reduce their risk.
What Can Doctors Do?
Chris Carlsten, MD, director of the Centre for Lung Health and professor and head of the Respiratory Medicine Division at the University of British Columbia, Vancouver, Canada, told this news organization that determining whether an exacerbation of a respiratory condition is caused by fire exposure or other factors, such as viral infections, is complex because both can trigger similar responses and may complement each other. “It’s very difficult for any individual to know whether, when they’re having an exacerbation of asthma or COPD, that’s due to the fire,” he said. Fire smoke also increases infection risks, further complicating diagnosis.
Dr. Carlsten suggested that physicians could recommend preventative use of inhalers for at-risk patients when wildfires occur rather than waiting for symptoms to worsen. “That is a really interesting idea that could be practical.” Still, he advises caution, stressing that patients should consult their providers because not all may react well to increased inhaler use.
He also highlighted a significant shift in the healthcare landscape, noting that traditionally, the focus has been on the cardiovascular impacts of pollution, particularly traffic-related pollution. However, as wildfire smoke becomes a growing issue, the focus is shifting back to respiratory problems, with profound implications for healthcare resources, budgets, and drug approvals based on the burden of respiratory disease. “Fire smoke is becoming more of a problem. This swing back to respiratory has huge implications for healthcare systems and respiratory disease burden.”
Mr. Sacks and Dr. Carlsten reported no relevant financial relationships. The study presented by Dr. Tonne received funding from the European Union’s Horizon Europe research and innovation programme under Grant Agreement No. 101057131.
A version of this article first appeared on Medscape.com.
FROM ERS 2024
How Doctors Can Overcome Vaccine Hesitancy Through Empathy, Storytelling, and Patient-Centered Communication
When Kimberly Fisher, MD, was a junior doctor, she got fired up when patients showed hesitancy about vaccines. She responded by providing numbers, data, and facts that proved vaccines were safe and effective in preventing life-threatening diseases. But she soon realized that regurgitating scientific evidence wasn’t a winning strategy. “I’ve made the mistake of launching into a let me tell you all the things that I know that you don’t know kind of lecture,” Dr. Fisher, now an associate professor of medicine at UMass Chan Medical School, Worcester, Massachusetts, a pulmonary physician, and a researcher interested in patient-provider communication, told this news organization. “Through experience and research, I have learned that when you do that, they stop listening.”
She said when patients give reasons for not getting vaccinated that are factually wrong and rooted in misinformation, the most common reaction is to correct that information and not let it stand. “That is important; it just can’t be the first thing you do,” she said.
Diane Arnaout, MD, a pediatrician at Cook Children’s Pediatrics in Fort Worth, Texas, said listening to some patients explaining why vaccine injections are poisonous or a conspiracy can be exhausting and frustrating, but she agrees that presenting scientific facts alone won’t change people’s minds. “Even in my worst days, I take the time to stop talking for a moment and let the parents talk about what concerns them because if you just get mad and put a wall up, then that trust is gone, possibly forever, not just about vaccines.”
The Default Option
Since the start of the COVID-19 pandemic, Dr. Fisher has dedicated much of her time researching vaccine hesitancy. One of the most “fascinating and unexpected” findings of her work was that people are more likely to get vaccinated if a healthcare provider recommends that they get vaccinated in a “presumptive style,” which means that the provider uses language that presupposes that the person’s going to get vaccinated. “Rather than asking whether they wanted to get the vaccine conveying that the option of not getting it is just as valid, you make vaccination the default option,” she suggested.
The strategy wins many undecided, but it might not work on the most reluctant. “The presumptive recommendation is very directive, and if that works, great, but if it doesn’t, you need to shift to almost the opposite strategy, showing empathy and understanding about the person’s reasons for not wanting to be vaccinated,” Dr. Fisher said.
Find One Thing to Agree On
During a focus group on COVID-19 vaccine hesitancy that Dr. Fisher conducted in December 2021, most physicians expressed frustration that some patients remained resistant despite their best efforts. However, one participant shared an approach she found effective with even the most hesitant patients. The physician would listen carefully and express understanding, and even if what the patient said wasn’t accurate, she would find a kernel of truth to agree with and align herself with the patient. By doing this, she made patients feel like they were a team.
The example she gave was if a patient said, “I don’t know. I’ve heard different things and don’t feel comfortable taking the vaccine,” she might respond with something like, “I think it’s great that you’re thinking critically about this before making a decision. I was the same way — I wanted to fully understand the data before getting vaccinated. I also wouldn’t want to take something if I thought it wasn’t safe. It’s good that you’re being thorough.” Acknowledging their careful thought process, the physician helped patients feel seen and understood only after she introduced additional information to guide them toward understanding why the vaccine might be beneficial.
Focus on the Disease
Dr. Arnaout’s frustration grows when at the end of an appointment some parents object to vaccines with irrational and misguided concerns. “You’ve trusted me with everything else we’ve discussed today — whether it’s a diaper rash or an ear infection — so why wouldn’t you trust me on this? Sometimes it feels almost offensive — why trust my medical expertise on everything else but not vaccines?” she said.
The answer, she believes, is that vaccines are preventive, and when the threat of disease feels distant, it’s hard to see the necessity of a painful shot for your healthy child. “But if your baby were dying from meningitis, the needles we use to deliver life-saving medications in the hospital would feel absolutely necessary. It’s hard as a parent to inflict pain for something you’ve never personally seen.”
Dr. Arnaout thinks it is important to bring the focus on the disease the vaccine prevents. “Let’s talk about measles — how if a baby in my waiting room has measles and coughs, the virus can stay suspended in the air for 2 hours, and 100% of unvaccinated people in that room will get measles.”
She said sharing personal stories can also help physicians connect with their patients. “I talk to parents every day about their vaccine concerns, and I’ve found that if I take the time to explain why we vaccinate, they start to understand. I also tell them, ‘I vaccinated my children for everything on time and give them the flu shot every year. Why would I offer your child something I wouldn’t give my own?’ That personal decision, made without hesitation, resonates with parents.”
Wired for Stories
Medical professionals have a professional necessity to think and speak with precision. Their training is based on analyzing studies and data, and they develop a specialized vocabulary to describe their findings accurately.
But the human brain is naturally inclined to process and make sense of information through the structure and narrative of stories. We instinctively organize reality into a “shape of a story” rather than just isolated facts, explained Ben Riggs, senior communications specialist at Kettering Health, Dayton, Ohio, a nonfiction writing coach and author. Storytelling also taps into the emotional, rather than just the rational, parts of the brain. This emotional connection helps make the information more memorable and impactful for the listener.
Mr. Riggs said that moving from this world of precision and accuracy to one that also requires effective communication with those who haven’t had that same training is much like learning a new language. “If they can’t speak in a way that non-scientists understand, it’s like the old saying: If a tree falls in the woods and no one hears it, does it make a sound?”
Metaphors can help doctors translate scientific facts into language that meets people where they are, allowing patients to make informed decisions about their health. They can help physicians transform abstract concepts into vivid, tangible mental images that are easier for people to understand and relate to, Mr. Riggs explained. “We are predominantly concrete thinkers. Metaphors can create concrete scenes and do much of the heavy lifting when communicating complex ideas.”
“It’s important to align yourself with the other person by showing that you care, that you’re truly listening, and understand their perspective,” concluded Dr. Fisher. “Acknowledge their point of view and emphasize that they have autonomy in the decision-making process. This can open people up to hearing your perspective. You also need to know when to let go don’t cause a rift in the relationship.”
Dr. Fisher, Dr. Arnaout, and Mr. Riggs reported no relevant financial relationships.
A version of this article first appeared on Medscape.com.
When Kimberly Fisher, MD, was a junior doctor, she got fired up when patients showed hesitancy about vaccines. She responded by providing numbers, data, and facts that proved vaccines were safe and effective in preventing life-threatening diseases. But she soon realized that regurgitating scientific evidence wasn’t a winning strategy. “I’ve made the mistake of launching into a let me tell you all the things that I know that you don’t know kind of lecture,” Dr. Fisher, now an associate professor of medicine at UMass Chan Medical School, Worcester, Massachusetts, a pulmonary physician, and a researcher interested in patient-provider communication, told this news organization. “Through experience and research, I have learned that when you do that, they stop listening.”
She said when patients give reasons for not getting vaccinated that are factually wrong and rooted in misinformation, the most common reaction is to correct that information and not let it stand. “That is important; it just can’t be the first thing you do,” she said.
Diane Arnaout, MD, a pediatrician at Cook Children’s Pediatrics in Fort Worth, Texas, said listening to some patients explaining why vaccine injections are poisonous or a conspiracy can be exhausting and frustrating, but she agrees that presenting scientific facts alone won’t change people’s minds. “Even in my worst days, I take the time to stop talking for a moment and let the parents talk about what concerns them because if you just get mad and put a wall up, then that trust is gone, possibly forever, not just about vaccines.”
The Default Option
Since the start of the COVID-19 pandemic, Dr. Fisher has dedicated much of her time researching vaccine hesitancy. One of the most “fascinating and unexpected” findings of her work was that people are more likely to get vaccinated if a healthcare provider recommends that they get vaccinated in a “presumptive style,” which means that the provider uses language that presupposes that the person’s going to get vaccinated. “Rather than asking whether they wanted to get the vaccine conveying that the option of not getting it is just as valid, you make vaccination the default option,” she suggested.
The strategy wins many undecided, but it might not work on the most reluctant. “The presumptive recommendation is very directive, and if that works, great, but if it doesn’t, you need to shift to almost the opposite strategy, showing empathy and understanding about the person’s reasons for not wanting to be vaccinated,” Dr. Fisher said.
Find One Thing to Agree On
During a focus group on COVID-19 vaccine hesitancy that Dr. Fisher conducted in December 2021, most physicians expressed frustration that some patients remained resistant despite their best efforts. However, one participant shared an approach she found effective with even the most hesitant patients. The physician would listen carefully and express understanding, and even if what the patient said wasn’t accurate, she would find a kernel of truth to agree with and align herself with the patient. By doing this, she made patients feel like they were a team.
The example she gave was if a patient said, “I don’t know. I’ve heard different things and don’t feel comfortable taking the vaccine,” she might respond with something like, “I think it’s great that you’re thinking critically about this before making a decision. I was the same way — I wanted to fully understand the data before getting vaccinated. I also wouldn’t want to take something if I thought it wasn’t safe. It’s good that you’re being thorough.” Acknowledging their careful thought process, the physician helped patients feel seen and understood only after she introduced additional information to guide them toward understanding why the vaccine might be beneficial.
Focus on the Disease
Dr. Arnaout’s frustration grows when at the end of an appointment some parents object to vaccines with irrational and misguided concerns. “You’ve trusted me with everything else we’ve discussed today — whether it’s a diaper rash or an ear infection — so why wouldn’t you trust me on this? Sometimes it feels almost offensive — why trust my medical expertise on everything else but not vaccines?” she said.
The answer, she believes, is that vaccines are preventive, and when the threat of disease feels distant, it’s hard to see the necessity of a painful shot for your healthy child. “But if your baby were dying from meningitis, the needles we use to deliver life-saving medications in the hospital would feel absolutely necessary. It’s hard as a parent to inflict pain for something you’ve never personally seen.”
Dr. Arnaout thinks it is important to bring the focus on the disease the vaccine prevents. “Let’s talk about measles — how if a baby in my waiting room has measles and coughs, the virus can stay suspended in the air for 2 hours, and 100% of unvaccinated people in that room will get measles.”
She said sharing personal stories can also help physicians connect with their patients. “I talk to parents every day about their vaccine concerns, and I’ve found that if I take the time to explain why we vaccinate, they start to understand. I also tell them, ‘I vaccinated my children for everything on time and give them the flu shot every year. Why would I offer your child something I wouldn’t give my own?’ That personal decision, made without hesitation, resonates with parents.”
Wired for Stories
Medical professionals have a professional necessity to think and speak with precision. Their training is based on analyzing studies and data, and they develop a specialized vocabulary to describe their findings accurately.
But the human brain is naturally inclined to process and make sense of information through the structure and narrative of stories. We instinctively organize reality into a “shape of a story” rather than just isolated facts, explained Ben Riggs, senior communications specialist at Kettering Health, Dayton, Ohio, a nonfiction writing coach and author. Storytelling also taps into the emotional, rather than just the rational, parts of the brain. This emotional connection helps make the information more memorable and impactful for the listener.
Mr. Riggs said that moving from this world of precision and accuracy to one that also requires effective communication with those who haven’t had that same training is much like learning a new language. “If they can’t speak in a way that non-scientists understand, it’s like the old saying: If a tree falls in the woods and no one hears it, does it make a sound?”
Metaphors can help doctors translate scientific facts into language that meets people where they are, allowing patients to make informed decisions about their health. They can help physicians transform abstract concepts into vivid, tangible mental images that are easier for people to understand and relate to, Mr. Riggs explained. “We are predominantly concrete thinkers. Metaphors can create concrete scenes and do much of the heavy lifting when communicating complex ideas.”
“It’s important to align yourself with the other person by showing that you care, that you’re truly listening, and understand their perspective,” concluded Dr. Fisher. “Acknowledge their point of view and emphasize that they have autonomy in the decision-making process. This can open people up to hearing your perspective. You also need to know when to let go don’t cause a rift in the relationship.”
Dr. Fisher, Dr. Arnaout, and Mr. Riggs reported no relevant financial relationships.
A version of this article first appeared on Medscape.com.
When Kimberly Fisher, MD, was a junior doctor, she got fired up when patients showed hesitancy about vaccines. She responded by providing numbers, data, and facts that proved vaccines were safe and effective in preventing life-threatening diseases. But she soon realized that regurgitating scientific evidence wasn’t a winning strategy. “I’ve made the mistake of launching into a let me tell you all the things that I know that you don’t know kind of lecture,” Dr. Fisher, now an associate professor of medicine at UMass Chan Medical School, Worcester, Massachusetts, a pulmonary physician, and a researcher interested in patient-provider communication, told this news organization. “Through experience and research, I have learned that when you do that, they stop listening.”
She said when patients give reasons for not getting vaccinated that are factually wrong and rooted in misinformation, the most common reaction is to correct that information and not let it stand. “That is important; it just can’t be the first thing you do,” she said.
Diane Arnaout, MD, a pediatrician at Cook Children’s Pediatrics in Fort Worth, Texas, said listening to some patients explaining why vaccine injections are poisonous or a conspiracy can be exhausting and frustrating, but she agrees that presenting scientific facts alone won’t change people’s minds. “Even in my worst days, I take the time to stop talking for a moment and let the parents talk about what concerns them because if you just get mad and put a wall up, then that trust is gone, possibly forever, not just about vaccines.”
The Default Option
Since the start of the COVID-19 pandemic, Dr. Fisher has dedicated much of her time researching vaccine hesitancy. One of the most “fascinating and unexpected” findings of her work was that people are more likely to get vaccinated if a healthcare provider recommends that they get vaccinated in a “presumptive style,” which means that the provider uses language that presupposes that the person’s going to get vaccinated. “Rather than asking whether they wanted to get the vaccine conveying that the option of not getting it is just as valid, you make vaccination the default option,” she suggested.
The strategy wins many undecided, but it might not work on the most reluctant. “The presumptive recommendation is very directive, and if that works, great, but if it doesn’t, you need to shift to almost the opposite strategy, showing empathy and understanding about the person’s reasons for not wanting to be vaccinated,” Dr. Fisher said.
Find One Thing to Agree On
During a focus group on COVID-19 vaccine hesitancy that Dr. Fisher conducted in December 2021, most physicians expressed frustration that some patients remained resistant despite their best efforts. However, one participant shared an approach she found effective with even the most hesitant patients. The physician would listen carefully and express understanding, and even if what the patient said wasn’t accurate, she would find a kernel of truth to agree with and align herself with the patient. By doing this, she made patients feel like they were a team.
The example she gave was if a patient said, “I don’t know. I’ve heard different things and don’t feel comfortable taking the vaccine,” she might respond with something like, “I think it’s great that you’re thinking critically about this before making a decision. I was the same way — I wanted to fully understand the data before getting vaccinated. I also wouldn’t want to take something if I thought it wasn’t safe. It’s good that you’re being thorough.” Acknowledging their careful thought process, the physician helped patients feel seen and understood only after she introduced additional information to guide them toward understanding why the vaccine might be beneficial.
Focus on the Disease
Dr. Arnaout’s frustration grows when at the end of an appointment some parents object to vaccines with irrational and misguided concerns. “You’ve trusted me with everything else we’ve discussed today — whether it’s a diaper rash or an ear infection — so why wouldn’t you trust me on this? Sometimes it feels almost offensive — why trust my medical expertise on everything else but not vaccines?” she said.
The answer, she believes, is that vaccines are preventive, and when the threat of disease feels distant, it’s hard to see the necessity of a painful shot for your healthy child. “But if your baby were dying from meningitis, the needles we use to deliver life-saving medications in the hospital would feel absolutely necessary. It’s hard as a parent to inflict pain for something you’ve never personally seen.”
Dr. Arnaout thinks it is important to bring the focus on the disease the vaccine prevents. “Let’s talk about measles — how if a baby in my waiting room has measles and coughs, the virus can stay suspended in the air for 2 hours, and 100% of unvaccinated people in that room will get measles.”
She said sharing personal stories can also help physicians connect with their patients. “I talk to parents every day about their vaccine concerns, and I’ve found that if I take the time to explain why we vaccinate, they start to understand. I also tell them, ‘I vaccinated my children for everything on time and give them the flu shot every year. Why would I offer your child something I wouldn’t give my own?’ That personal decision, made without hesitation, resonates with parents.”
Wired for Stories
Medical professionals have a professional necessity to think and speak with precision. Their training is based on analyzing studies and data, and they develop a specialized vocabulary to describe their findings accurately.
But the human brain is naturally inclined to process and make sense of information through the structure and narrative of stories. We instinctively organize reality into a “shape of a story” rather than just isolated facts, explained Ben Riggs, senior communications specialist at Kettering Health, Dayton, Ohio, a nonfiction writing coach and author. Storytelling also taps into the emotional, rather than just the rational, parts of the brain. This emotional connection helps make the information more memorable and impactful for the listener.
Mr. Riggs said that moving from this world of precision and accuracy to one that also requires effective communication with those who haven’t had that same training is much like learning a new language. “If they can’t speak in a way that non-scientists understand, it’s like the old saying: If a tree falls in the woods and no one hears it, does it make a sound?”
Metaphors can help doctors translate scientific facts into language that meets people where they are, allowing patients to make informed decisions about their health. They can help physicians transform abstract concepts into vivid, tangible mental images that are easier for people to understand and relate to, Mr. Riggs explained. “We are predominantly concrete thinkers. Metaphors can create concrete scenes and do much of the heavy lifting when communicating complex ideas.”
“It’s important to align yourself with the other person by showing that you care, that you’re truly listening, and understand their perspective,” concluded Dr. Fisher. “Acknowledge their point of view and emphasize that they have autonomy in the decision-making process. This can open people up to hearing your perspective. You also need to know when to let go don’t cause a rift in the relationship.”
Dr. Fisher, Dr. Arnaout, and Mr. Riggs reported no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Monitor Asthma Patients on Biologics for Remission, Potential EGPA Symptoms During Steroid Tapering
VIENNA — , according to pulmonary experts presenting at the European Respiratory Society (ERS) 2024 International Congress.
Biologics have revolutionized the treatment of severe asthma, significantly improving patient outcomes. However, the focus has recently shifted toward achieving more comprehensive disease control. Remission, already a well-established goal in conditions like rheumatoid arthritis and inflammatory bowel disease, is now being explored in patients with asthma receiving biologics.
Peter Howarth, medical director at Global Medical, Specialty Medicine, GSK, in Brentford, England, said that new clinical remission criteria in asthma may be overly rigid and of little use. He said that more attainable limits must be created. Meanwhile, clinicians should collect clinical data more thoroughly.
In parallel, studies have also raised questions about the role of biologics in the emergence of EGPA.
Defining Clinical Remission in Asthma
Last year, a working group, including members from the American Thoracic Society and the American College and Academy of Allergy, Asthma, and Immunology, proposed new guidelines to define clinical remission in asthma. These guidelines extended beyond the typical outcomes of no severe exacerbations, no maintenance oral corticosteroid use, good asthma control, and stable lung function. The additional recommendations included no missed work or school due to asthma, limited use of rescue medication (no more than once a month), and reduced inhaled corticosteroid use to low or medium doses.
To explore the feasibility of achieving these clinical remission outcomes, GSK partnered with the Mayo Clinic for a retrospective analysis of the medical records of 700 patients with asthma undergoing various biologic therapies. The study revealed that essential data for determining clinical remission, such as asthma control and exacerbation records, were inconsistently documented. While some data were recorded, such as maintenance corticosteroid use in 50%-60% of cases, other key measures, like asthma control, were recorded in less than a quarter of the patients.
GSK researchers analyzed available data and found that around 30% of patients on any biologic therapy met three components of remission. Mepolizumab performed better than other corticosteroids, with over 40% of those receiving the drug meeting these criteria. However, when stricter definitions were applied, such as requiring four or more remission components, fewer patients achieved remission — less than 10% for four components, with no patients meeting the full seven-point criteria proposed by the working group.
An ongoing ERS Task Force is now exploring what clinical remission outcomes are practical to achieve, as the current definitions may be too aspirational, said Mr. Howarth. “It’s a matter of defying what is practical to achieve because if you can’t achieve it, then it won’t be valuable.”
He also pointed out that biologics are often used for the most severe cases of asthma after other treatments have failed. Evidence suggests that introducing biologics earlier in the disease, before chronic damage occurs, may result in better patient outcomes.
Biologics and EGPA
In a retrospective study, clinical details of 27 patients with adult-onset asthma from 28 countries, all on biologic therapy, were analyzed. The study, a multicounty collaboration, was led by ERS Severe Heterogeneous Asthma Research Collaboration, Patient-centred (SHARP), and aimed to understand the role of biologics in the emergence of EGPA.
The most significant finding presented at the ERS 2024 International Congress was that EGPA was not associated with maintenance corticosteroids; instead, it often emerged when corticosteroid doses were reduced or tapered off. “This might suggest that steroid withdrawal may unmask the underlying disease,” said Hitasha Rupani, MD, a consultant respiratory physician at the University Hospital Southampton, in Southampton, England. Importantly, the rate at which steroids were tapered did not influence the onset of EGPA, indicating that the tapering process, rather than its speed, may be the critical factor. However, due to the small sample size, this remains a hypothesis, Dr. Rupani explained.
The study also found that when clinicians had a clinical suspicion of EGPA before starting biologic therapy, the diagnosis was made earlier than in cases without such suspicion. Dr. Rupani concluded that this underscores the importance of clinical vigilance and the need to monitor patients closely for EGPA symptoms, especially during corticosteroid tapering.
The study was funded by GSK. Mr. Howarth is an employee at GSK. Dr. Rupani reports no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — , according to pulmonary experts presenting at the European Respiratory Society (ERS) 2024 International Congress.
Biologics have revolutionized the treatment of severe asthma, significantly improving patient outcomes. However, the focus has recently shifted toward achieving more comprehensive disease control. Remission, already a well-established goal in conditions like rheumatoid arthritis and inflammatory bowel disease, is now being explored in patients with asthma receiving biologics.
Peter Howarth, medical director at Global Medical, Specialty Medicine, GSK, in Brentford, England, said that new clinical remission criteria in asthma may be overly rigid and of little use. He said that more attainable limits must be created. Meanwhile, clinicians should collect clinical data more thoroughly.
In parallel, studies have also raised questions about the role of biologics in the emergence of EGPA.
Defining Clinical Remission in Asthma
Last year, a working group, including members from the American Thoracic Society and the American College and Academy of Allergy, Asthma, and Immunology, proposed new guidelines to define clinical remission in asthma. These guidelines extended beyond the typical outcomes of no severe exacerbations, no maintenance oral corticosteroid use, good asthma control, and stable lung function. The additional recommendations included no missed work or school due to asthma, limited use of rescue medication (no more than once a month), and reduced inhaled corticosteroid use to low or medium doses.
To explore the feasibility of achieving these clinical remission outcomes, GSK partnered with the Mayo Clinic for a retrospective analysis of the medical records of 700 patients with asthma undergoing various biologic therapies. The study revealed that essential data for determining clinical remission, such as asthma control and exacerbation records, were inconsistently documented. While some data were recorded, such as maintenance corticosteroid use in 50%-60% of cases, other key measures, like asthma control, were recorded in less than a quarter of the patients.
GSK researchers analyzed available data and found that around 30% of patients on any biologic therapy met three components of remission. Mepolizumab performed better than other corticosteroids, with over 40% of those receiving the drug meeting these criteria. However, when stricter definitions were applied, such as requiring four or more remission components, fewer patients achieved remission — less than 10% for four components, with no patients meeting the full seven-point criteria proposed by the working group.
An ongoing ERS Task Force is now exploring what clinical remission outcomes are practical to achieve, as the current definitions may be too aspirational, said Mr. Howarth. “It’s a matter of defying what is practical to achieve because if you can’t achieve it, then it won’t be valuable.”
He also pointed out that biologics are often used for the most severe cases of asthma after other treatments have failed. Evidence suggests that introducing biologics earlier in the disease, before chronic damage occurs, may result in better patient outcomes.
Biologics and EGPA
In a retrospective study, clinical details of 27 patients with adult-onset asthma from 28 countries, all on biologic therapy, were analyzed. The study, a multicounty collaboration, was led by ERS Severe Heterogeneous Asthma Research Collaboration, Patient-centred (SHARP), and aimed to understand the role of biologics in the emergence of EGPA.
The most significant finding presented at the ERS 2024 International Congress was that EGPA was not associated with maintenance corticosteroids; instead, it often emerged when corticosteroid doses were reduced or tapered off. “This might suggest that steroid withdrawal may unmask the underlying disease,” said Hitasha Rupani, MD, a consultant respiratory physician at the University Hospital Southampton, in Southampton, England. Importantly, the rate at which steroids were tapered did not influence the onset of EGPA, indicating that the tapering process, rather than its speed, may be the critical factor. However, due to the small sample size, this remains a hypothesis, Dr. Rupani explained.
The study also found that when clinicians had a clinical suspicion of EGPA before starting biologic therapy, the diagnosis was made earlier than in cases without such suspicion. Dr. Rupani concluded that this underscores the importance of clinical vigilance and the need to monitor patients closely for EGPA symptoms, especially during corticosteroid tapering.
The study was funded by GSK. Mr. Howarth is an employee at GSK. Dr. Rupani reports no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — , according to pulmonary experts presenting at the European Respiratory Society (ERS) 2024 International Congress.
Biologics have revolutionized the treatment of severe asthma, significantly improving patient outcomes. However, the focus has recently shifted toward achieving more comprehensive disease control. Remission, already a well-established goal in conditions like rheumatoid arthritis and inflammatory bowel disease, is now being explored in patients with asthma receiving biologics.
Peter Howarth, medical director at Global Medical, Specialty Medicine, GSK, in Brentford, England, said that new clinical remission criteria in asthma may be overly rigid and of little use. He said that more attainable limits must be created. Meanwhile, clinicians should collect clinical data more thoroughly.
In parallel, studies have also raised questions about the role of biologics in the emergence of EGPA.
Defining Clinical Remission in Asthma
Last year, a working group, including members from the American Thoracic Society and the American College and Academy of Allergy, Asthma, and Immunology, proposed new guidelines to define clinical remission in asthma. These guidelines extended beyond the typical outcomes of no severe exacerbations, no maintenance oral corticosteroid use, good asthma control, and stable lung function. The additional recommendations included no missed work or school due to asthma, limited use of rescue medication (no more than once a month), and reduced inhaled corticosteroid use to low or medium doses.
To explore the feasibility of achieving these clinical remission outcomes, GSK partnered with the Mayo Clinic for a retrospective analysis of the medical records of 700 patients with asthma undergoing various biologic therapies. The study revealed that essential data for determining clinical remission, such as asthma control and exacerbation records, were inconsistently documented. While some data were recorded, such as maintenance corticosteroid use in 50%-60% of cases, other key measures, like asthma control, were recorded in less than a quarter of the patients.
GSK researchers analyzed available data and found that around 30% of patients on any biologic therapy met three components of remission. Mepolizumab performed better than other corticosteroids, with over 40% of those receiving the drug meeting these criteria. However, when stricter definitions were applied, such as requiring four or more remission components, fewer patients achieved remission — less than 10% for four components, with no patients meeting the full seven-point criteria proposed by the working group.
An ongoing ERS Task Force is now exploring what clinical remission outcomes are practical to achieve, as the current definitions may be too aspirational, said Mr. Howarth. “It’s a matter of defying what is practical to achieve because if you can’t achieve it, then it won’t be valuable.”
He also pointed out that biologics are often used for the most severe cases of asthma after other treatments have failed. Evidence suggests that introducing biologics earlier in the disease, before chronic damage occurs, may result in better patient outcomes.
Biologics and EGPA
In a retrospective study, clinical details of 27 patients with adult-onset asthma from 28 countries, all on biologic therapy, were analyzed. The study, a multicounty collaboration, was led by ERS Severe Heterogeneous Asthma Research Collaboration, Patient-centred (SHARP), and aimed to understand the role of biologics in the emergence of EGPA.
The most significant finding presented at the ERS 2024 International Congress was that EGPA was not associated with maintenance corticosteroids; instead, it often emerged when corticosteroid doses were reduced or tapered off. “This might suggest that steroid withdrawal may unmask the underlying disease,” said Hitasha Rupani, MD, a consultant respiratory physician at the University Hospital Southampton, in Southampton, England. Importantly, the rate at which steroids were tapered did not influence the onset of EGPA, indicating that the tapering process, rather than its speed, may be the critical factor. However, due to the small sample size, this remains a hypothesis, Dr. Rupani explained.
The study also found that when clinicians had a clinical suspicion of EGPA before starting biologic therapy, the diagnosis was made earlier than in cases without such suspicion. Dr. Rupani concluded that this underscores the importance of clinical vigilance and the need to monitor patients closely for EGPA symptoms, especially during corticosteroid tapering.
The study was funded by GSK. Mr. Howarth is an employee at GSK. Dr. Rupani reports no relevant financial relationships.
A version of this article appeared on Medscape.com.
Genetic Testing and Novel Biomarkers Important in Cystic Fibrosis Diagnosis and Monitoring
VIENNA — Advances in genetic testing and newly discovered biomarkers can help screen newborns and monitor inflammation and pulmonary exacerbations in patients diagnosed with cystic fibrosis.
At the European Respiratory Society (ERS) 2024 International Congress, clinical researchers presented results from the Turkish context.
Cystic fibrosis is the most common genetic disorder among Caucasians. The average prevalence at birth in Europe is 1 in 5000, whereas the overall population averages 1 in 9000. Both rates vary significantly based on geographic area. In the central Anatolia region, one study found that the incidence of cystic fibrosis is 1 in 3400 live births.
Çigdem Korkmaz, a researcher at the Department of Pediatric Pulmonology at the Istanbul University-Cerrahpasa in Istanbul, Turkey, said that diagnosis in Turkey is especially challenging because of the genetic diversity of cystic fibrosis within the population. She said genetic testing might be necessary to catch missed cases by traditional screening methods.
Genetic Testing Picks Up Missed Cases
In 2022, 30 European countries run newborn bloodspot screening for cystic fibrosis, with 26 national programs. Screening protocols vary between countries but generally involve initial screening using an immunoreactive trypsinogen (IRT) blood test. Follow-up testing may include a second IRT test, DNA analysis for common CFTR mutations, and sweat chloride test (SCT).
Turkey introduced newborn screening for cystic fibrosis in 2015. Newborns with an elevated IRT and confirmatory SCT undergo genetic testing. However, in a retrospective study, researchers found that IRT tests turn many false-positive results, and some patients who turn a normal SCT are diagnosed with the disease through genetic testing.
The study included 205 infants referred to a tertiary care center in Istanbul between January 2015 and January 2023 following an elevator IRT result. The researchers analyzed the clinical and sociodemographic data, IRT and SCT values, and genetic analysis results.
“The high false-positive rate of the current screening strategy suggests that the IRT thresholds used in Turkey may be too low,” said Ms. Korkmaz, who presented the study at the ERS Congress. She added that genetic testing might be important, especially in patients with normal SCT results. “Early diagnosis means these patients avoid missing or delaying treatments.”
Biomarkers for Monitoring Cystic Fibrosis Exacerbations
C-reactive protein (CRP) blood testing is typically used in monitoring inflammation and pulmonary exacerbations in patients who have already been diagnosed with cystic fibrosis. CRP is an inflammatory biomarker that increases in patients with cystic fibrosis during pulmonary exacerbations and settles with treatment.
Researchers at Gazi University in Ankara, Turkey, found other biomarkers to identify inflammation and pulmonary exacerbations with great sensitivity and specificity in patients with cystic fibrosis.
Over 3 years, from 2021 to 2024, the researchers analyzed blood samples from 54 children aged 1-18 years during exacerbation and non-exacerbation periods. Besides CRP, they tested CRP/albumin (ALB) ratio, neutrophil-to-lymphocyte ratio (NLR), delivered NLR (dNLR), and systemic immune inflammation (SII).
All biomarkers increased during exacerbation episodes. All showed high specificity and sensitivity:
- CPR/ALB had a specificity of 81% and a sensitivity of 90% at a cutoff of 1.7 mg/dL.
- SII had a specificity of 86% and a sensitivity of 67% at a cutoff of 426 mg/dL.
- NLR had a specificity of 62% and a sensitivity of 79% at a cutoff of 2.2 mg/dL.
- SII had a specificity of 86% and a sensitivity of 67% at a cutoff of 426 mg/dL.
- dNLR had a specificity of 71% and a sensitivity of 66% at a cutoff of 1.15 mg/dL.
- In comparison, CPR had a specificity of 85% and a sensitivity of 84% at a cutoff of 6.2 mg/dL.
Ayse Tana Aslan, a professor at the Department of Pediatric Pulmonology, Faculty of Medicine, at Gazi University in Ankara, Turkey, who presented the results at the ERS Congress, said that these biomarkers can be easily and quickly identified with a blood test while waiting on phlegm culture results, which can take days. “It is important to predict inflammation and exacerbation quickly so that patients can start a course of antibiotics as soon as possible,” she said.
Ms. Korkmaz and Ms. Aslan reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — Advances in genetic testing and newly discovered biomarkers can help screen newborns and monitor inflammation and pulmonary exacerbations in patients diagnosed with cystic fibrosis.
At the European Respiratory Society (ERS) 2024 International Congress, clinical researchers presented results from the Turkish context.
Cystic fibrosis is the most common genetic disorder among Caucasians. The average prevalence at birth in Europe is 1 in 5000, whereas the overall population averages 1 in 9000. Both rates vary significantly based on geographic area. In the central Anatolia region, one study found that the incidence of cystic fibrosis is 1 in 3400 live births.
Çigdem Korkmaz, a researcher at the Department of Pediatric Pulmonology at the Istanbul University-Cerrahpasa in Istanbul, Turkey, said that diagnosis in Turkey is especially challenging because of the genetic diversity of cystic fibrosis within the population. She said genetic testing might be necessary to catch missed cases by traditional screening methods.
Genetic Testing Picks Up Missed Cases
In 2022, 30 European countries run newborn bloodspot screening for cystic fibrosis, with 26 national programs. Screening protocols vary between countries but generally involve initial screening using an immunoreactive trypsinogen (IRT) blood test. Follow-up testing may include a second IRT test, DNA analysis for common CFTR mutations, and sweat chloride test (SCT).
Turkey introduced newborn screening for cystic fibrosis in 2015. Newborns with an elevated IRT and confirmatory SCT undergo genetic testing. However, in a retrospective study, researchers found that IRT tests turn many false-positive results, and some patients who turn a normal SCT are diagnosed with the disease through genetic testing.
The study included 205 infants referred to a tertiary care center in Istanbul between January 2015 and January 2023 following an elevator IRT result. The researchers analyzed the clinical and sociodemographic data, IRT and SCT values, and genetic analysis results.
“The high false-positive rate of the current screening strategy suggests that the IRT thresholds used in Turkey may be too low,” said Ms. Korkmaz, who presented the study at the ERS Congress. She added that genetic testing might be important, especially in patients with normal SCT results. “Early diagnosis means these patients avoid missing or delaying treatments.”
Biomarkers for Monitoring Cystic Fibrosis Exacerbations
C-reactive protein (CRP) blood testing is typically used in monitoring inflammation and pulmonary exacerbations in patients who have already been diagnosed with cystic fibrosis. CRP is an inflammatory biomarker that increases in patients with cystic fibrosis during pulmonary exacerbations and settles with treatment.
Researchers at Gazi University in Ankara, Turkey, found other biomarkers to identify inflammation and pulmonary exacerbations with great sensitivity and specificity in patients with cystic fibrosis.
Over 3 years, from 2021 to 2024, the researchers analyzed blood samples from 54 children aged 1-18 years during exacerbation and non-exacerbation periods. Besides CRP, they tested CRP/albumin (ALB) ratio, neutrophil-to-lymphocyte ratio (NLR), delivered NLR (dNLR), and systemic immune inflammation (SII).
All biomarkers increased during exacerbation episodes. All showed high specificity and sensitivity:
- CPR/ALB had a specificity of 81% and a sensitivity of 90% at a cutoff of 1.7 mg/dL.
- SII had a specificity of 86% and a sensitivity of 67% at a cutoff of 426 mg/dL.
- NLR had a specificity of 62% and a sensitivity of 79% at a cutoff of 2.2 mg/dL.
- SII had a specificity of 86% and a sensitivity of 67% at a cutoff of 426 mg/dL.
- dNLR had a specificity of 71% and a sensitivity of 66% at a cutoff of 1.15 mg/dL.
- In comparison, CPR had a specificity of 85% and a sensitivity of 84% at a cutoff of 6.2 mg/dL.
Ayse Tana Aslan, a professor at the Department of Pediatric Pulmonology, Faculty of Medicine, at Gazi University in Ankara, Turkey, who presented the results at the ERS Congress, said that these biomarkers can be easily and quickly identified with a blood test while waiting on phlegm culture results, which can take days. “It is important to predict inflammation and exacerbation quickly so that patients can start a course of antibiotics as soon as possible,” she said.
Ms. Korkmaz and Ms. Aslan reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — Advances in genetic testing and newly discovered biomarkers can help screen newborns and monitor inflammation and pulmonary exacerbations in patients diagnosed with cystic fibrosis.
At the European Respiratory Society (ERS) 2024 International Congress, clinical researchers presented results from the Turkish context.
Cystic fibrosis is the most common genetic disorder among Caucasians. The average prevalence at birth in Europe is 1 in 5000, whereas the overall population averages 1 in 9000. Both rates vary significantly based on geographic area. In the central Anatolia region, one study found that the incidence of cystic fibrosis is 1 in 3400 live births.
Çigdem Korkmaz, a researcher at the Department of Pediatric Pulmonology at the Istanbul University-Cerrahpasa in Istanbul, Turkey, said that diagnosis in Turkey is especially challenging because of the genetic diversity of cystic fibrosis within the population. She said genetic testing might be necessary to catch missed cases by traditional screening methods.
Genetic Testing Picks Up Missed Cases
In 2022, 30 European countries run newborn bloodspot screening for cystic fibrosis, with 26 national programs. Screening protocols vary between countries but generally involve initial screening using an immunoreactive trypsinogen (IRT) blood test. Follow-up testing may include a second IRT test, DNA analysis for common CFTR mutations, and sweat chloride test (SCT).
Turkey introduced newborn screening for cystic fibrosis in 2015. Newborns with an elevated IRT and confirmatory SCT undergo genetic testing. However, in a retrospective study, researchers found that IRT tests turn many false-positive results, and some patients who turn a normal SCT are diagnosed with the disease through genetic testing.
The study included 205 infants referred to a tertiary care center in Istanbul between January 2015 and January 2023 following an elevator IRT result. The researchers analyzed the clinical and sociodemographic data, IRT and SCT values, and genetic analysis results.
“The high false-positive rate of the current screening strategy suggests that the IRT thresholds used in Turkey may be too low,” said Ms. Korkmaz, who presented the study at the ERS Congress. She added that genetic testing might be important, especially in patients with normal SCT results. “Early diagnosis means these patients avoid missing or delaying treatments.”
Biomarkers for Monitoring Cystic Fibrosis Exacerbations
C-reactive protein (CRP) blood testing is typically used in monitoring inflammation and pulmonary exacerbations in patients who have already been diagnosed with cystic fibrosis. CRP is an inflammatory biomarker that increases in patients with cystic fibrosis during pulmonary exacerbations and settles with treatment.
Researchers at Gazi University in Ankara, Turkey, found other biomarkers to identify inflammation and pulmonary exacerbations with great sensitivity and specificity in patients with cystic fibrosis.
Over 3 years, from 2021 to 2024, the researchers analyzed blood samples from 54 children aged 1-18 years during exacerbation and non-exacerbation periods. Besides CRP, they tested CRP/albumin (ALB) ratio, neutrophil-to-lymphocyte ratio (NLR), delivered NLR (dNLR), and systemic immune inflammation (SII).
All biomarkers increased during exacerbation episodes. All showed high specificity and sensitivity:
- CPR/ALB had a specificity of 81% and a sensitivity of 90% at a cutoff of 1.7 mg/dL.
- SII had a specificity of 86% and a sensitivity of 67% at a cutoff of 426 mg/dL.
- NLR had a specificity of 62% and a sensitivity of 79% at a cutoff of 2.2 mg/dL.
- SII had a specificity of 86% and a sensitivity of 67% at a cutoff of 426 mg/dL.
- dNLR had a specificity of 71% and a sensitivity of 66% at a cutoff of 1.15 mg/dL.
- In comparison, CPR had a specificity of 85% and a sensitivity of 84% at a cutoff of 6.2 mg/dL.
Ayse Tana Aslan, a professor at the Department of Pediatric Pulmonology, Faculty of Medicine, at Gazi University in Ankara, Turkey, who presented the results at the ERS Congress, said that these biomarkers can be easily and quickly identified with a blood test while waiting on phlegm culture results, which can take days. “It is important to predict inflammation and exacerbation quickly so that patients can start a course of antibiotics as soon as possible,” she said.
Ms. Korkmaz and Ms. Aslan reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
Blood Eosinophil Counts Might Predict Childhood Asthma, Treatment Response
VIENNA — Simply relying on clinical symptoms is insufficient to predict which children with wheezing will develop asthma and respond to treatments.
Sejal Saglani, MD, PhD, a professor of pediatric respiratory medicine at the National Heart and Lung Institute, Imperial College, London, England, said that preschool wheezing has long-term adverse consequences through to adulthood. “We need to prevent that downward trajectory of low lung function,” she said, presenting the latest research in the field at the annual European Respiratory Society International Congress.
Wheezing affects up to one third of all infants and preschool children, with one third developing asthma later in life. “It’s important to identify those kids because then we can treat them with the right medication,” said Mariëlle W.H. Pijnenburg, MD, PhD, a pulmonary specialist at Erasmus University Rotterdam in the Netherlands.
“We cannot just use clinical phenotype to decide what treatment a child should get. We need to run tests to identify the endotype of preschool wheeze and intervene appropriately,” Dr. Saglani added.
Eosinophilia as a Biomarker for Predicting Exacerbations and Steroid Responsiveness
In a cluster analysis, Dr. Saglani and colleagues classified preschool children with wheezing into two main subgroups: Those who experience frequent exacerbations and those who experience sporadic attacks. Frequent exacerbators were more likely to develop asthma, use asthma medications, and show signs of reduced lung function and airway inflammation, such as higher fractional exhaled nitric oxide and allergic sensitization. “Severe and frequent exacerbators are the kids that get in trouble,” she said. “They’re the ones we must identify at preschool age and really try to minimize their exacerbations.”
Research has shown that eosinophilia is a valuable biomarker in predicting both asthma exacerbations and responsiveness to inhaled corticosteroids. Children with elevated blood eosinophils are more likely to experience frequent and severe exacerbations. These children often demonstrate an inflammatory profile more responsive to corticosteroids, making eosinophilia a predictor of treatment success. Children with eosinophilia are also more likely to have underlying allergic sensitizations, which further supports the use of corticosteroids as part of their management strategy.
Dr. Saglani said a simple blood test can provide a window into the child’s inflammatory status, allowing physicians to make more targeted and personalized treatment plans.
Traditionally, identifying eosinophilia required venipuncture and laboratory analysis, which can be time consuming and impractical in a busy clinical setting. Dr. Saglani’s research group is developing a point-of-care test designed to quickly and efficiently measure blood eosinophil levels in children with asthma or wheezing symptoms from a finger-prick test. Preliminary data presented at the congress show that children with higher eosinophil counts in the clinic were more likely to experience an asthma attack within 3 months.
“The problem is the majority of the children we see are either not atopic or do not have high blood eosinophils. What are we going to do with those?”
How to Treat Those Who Don’t Have Eosinophilia
Most children with wheezing are not atopic and do not exhibit eosinophilic inflammation, and these children may not respond as effectively to corticosteroids. How to treat them remains the “1-billion-dollar question,” Dr. Saglani said.
Respiratory syncytial virus and rhinovirus play a crucial role in triggering wheezing episodes in these children. Research has shown that viral-induced wheezing is a common feature in this phenotype, and repeated viral infections can lead to an increased severity and frequency of exacerbations. However, there are currently no effective antiviral therapies or vaccines for rhinovirus, which limits the ability to address the viral component of the disease directly.
Up to 50% of children with severe, recurrent wheezing also have bacterial pathogens like Moraxella catarrhalis and Haemophilus influenzae in their lower airways. For these children, addressing the bacterial infection is the best treatment option to mitigate the wheezing. “We now have something that we can target with antibiotics for those who don’t respond to corticosteroids,” Dr. Saglani said.
Dr. Pijnenburg said that this body of research is helping pulmonary specialists and general pediatricians navigate the complexity of childhood wheezing beyond phenotyping and symptoms. “We need to dive more deeply into those kids with preschool wheezing to see what’s happening in their lungs.”
Dr. Pijnenburg and Dr. Saglani reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — Simply relying on clinical symptoms is insufficient to predict which children with wheezing will develop asthma and respond to treatments.
Sejal Saglani, MD, PhD, a professor of pediatric respiratory medicine at the National Heart and Lung Institute, Imperial College, London, England, said that preschool wheezing has long-term adverse consequences through to adulthood. “We need to prevent that downward trajectory of low lung function,” she said, presenting the latest research in the field at the annual European Respiratory Society International Congress.
Wheezing affects up to one third of all infants and preschool children, with one third developing asthma later in life. “It’s important to identify those kids because then we can treat them with the right medication,” said Mariëlle W.H. Pijnenburg, MD, PhD, a pulmonary specialist at Erasmus University Rotterdam in the Netherlands.
“We cannot just use clinical phenotype to decide what treatment a child should get. We need to run tests to identify the endotype of preschool wheeze and intervene appropriately,” Dr. Saglani added.
Eosinophilia as a Biomarker for Predicting Exacerbations and Steroid Responsiveness
In a cluster analysis, Dr. Saglani and colleagues classified preschool children with wheezing into two main subgroups: Those who experience frequent exacerbations and those who experience sporadic attacks. Frequent exacerbators were more likely to develop asthma, use asthma medications, and show signs of reduced lung function and airway inflammation, such as higher fractional exhaled nitric oxide and allergic sensitization. “Severe and frequent exacerbators are the kids that get in trouble,” she said. “They’re the ones we must identify at preschool age and really try to minimize their exacerbations.”
Research has shown that eosinophilia is a valuable biomarker in predicting both asthma exacerbations and responsiveness to inhaled corticosteroids. Children with elevated blood eosinophils are more likely to experience frequent and severe exacerbations. These children often demonstrate an inflammatory profile more responsive to corticosteroids, making eosinophilia a predictor of treatment success. Children with eosinophilia are also more likely to have underlying allergic sensitizations, which further supports the use of corticosteroids as part of their management strategy.
Dr. Saglani said a simple blood test can provide a window into the child’s inflammatory status, allowing physicians to make more targeted and personalized treatment plans.
Traditionally, identifying eosinophilia required venipuncture and laboratory analysis, which can be time consuming and impractical in a busy clinical setting. Dr. Saglani’s research group is developing a point-of-care test designed to quickly and efficiently measure blood eosinophil levels in children with asthma or wheezing symptoms from a finger-prick test. Preliminary data presented at the congress show that children with higher eosinophil counts in the clinic were more likely to experience an asthma attack within 3 months.
“The problem is the majority of the children we see are either not atopic or do not have high blood eosinophils. What are we going to do with those?”
How to Treat Those Who Don’t Have Eosinophilia
Most children with wheezing are not atopic and do not exhibit eosinophilic inflammation, and these children may not respond as effectively to corticosteroids. How to treat them remains the “1-billion-dollar question,” Dr. Saglani said.
Respiratory syncytial virus and rhinovirus play a crucial role in triggering wheezing episodes in these children. Research has shown that viral-induced wheezing is a common feature in this phenotype, and repeated viral infections can lead to an increased severity and frequency of exacerbations. However, there are currently no effective antiviral therapies or vaccines for rhinovirus, which limits the ability to address the viral component of the disease directly.
Up to 50% of children with severe, recurrent wheezing also have bacterial pathogens like Moraxella catarrhalis and Haemophilus influenzae in their lower airways. For these children, addressing the bacterial infection is the best treatment option to mitigate the wheezing. “We now have something that we can target with antibiotics for those who don’t respond to corticosteroids,” Dr. Saglani said.
Dr. Pijnenburg said that this body of research is helping pulmonary specialists and general pediatricians navigate the complexity of childhood wheezing beyond phenotyping and symptoms. “We need to dive more deeply into those kids with preschool wheezing to see what’s happening in their lungs.”
Dr. Pijnenburg and Dr. Saglani reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — Simply relying on clinical symptoms is insufficient to predict which children with wheezing will develop asthma and respond to treatments.
Sejal Saglani, MD, PhD, a professor of pediatric respiratory medicine at the National Heart and Lung Institute, Imperial College, London, England, said that preschool wheezing has long-term adverse consequences through to adulthood. “We need to prevent that downward trajectory of low lung function,” she said, presenting the latest research in the field at the annual European Respiratory Society International Congress.
Wheezing affects up to one third of all infants and preschool children, with one third developing asthma later in life. “It’s important to identify those kids because then we can treat them with the right medication,” said Mariëlle W.H. Pijnenburg, MD, PhD, a pulmonary specialist at Erasmus University Rotterdam in the Netherlands.
“We cannot just use clinical phenotype to decide what treatment a child should get. We need to run tests to identify the endotype of preschool wheeze and intervene appropriately,” Dr. Saglani added.
Eosinophilia as a Biomarker for Predicting Exacerbations and Steroid Responsiveness
In a cluster analysis, Dr. Saglani and colleagues classified preschool children with wheezing into two main subgroups: Those who experience frequent exacerbations and those who experience sporadic attacks. Frequent exacerbators were more likely to develop asthma, use asthma medications, and show signs of reduced lung function and airway inflammation, such as higher fractional exhaled nitric oxide and allergic sensitization. “Severe and frequent exacerbators are the kids that get in trouble,” she said. “They’re the ones we must identify at preschool age and really try to minimize their exacerbations.”
Research has shown that eosinophilia is a valuable biomarker in predicting both asthma exacerbations and responsiveness to inhaled corticosteroids. Children with elevated blood eosinophils are more likely to experience frequent and severe exacerbations. These children often demonstrate an inflammatory profile more responsive to corticosteroids, making eosinophilia a predictor of treatment success. Children with eosinophilia are also more likely to have underlying allergic sensitizations, which further supports the use of corticosteroids as part of their management strategy.
Dr. Saglani said a simple blood test can provide a window into the child’s inflammatory status, allowing physicians to make more targeted and personalized treatment plans.
Traditionally, identifying eosinophilia required venipuncture and laboratory analysis, which can be time consuming and impractical in a busy clinical setting. Dr. Saglani’s research group is developing a point-of-care test designed to quickly and efficiently measure blood eosinophil levels in children with asthma or wheezing symptoms from a finger-prick test. Preliminary data presented at the congress show that children with higher eosinophil counts in the clinic were more likely to experience an asthma attack within 3 months.
“The problem is the majority of the children we see are either not atopic or do not have high blood eosinophils. What are we going to do with those?”
How to Treat Those Who Don’t Have Eosinophilia
Most children with wheezing are not atopic and do not exhibit eosinophilic inflammation, and these children may not respond as effectively to corticosteroids. How to treat them remains the “1-billion-dollar question,” Dr. Saglani said.
Respiratory syncytial virus and rhinovirus play a crucial role in triggering wheezing episodes in these children. Research has shown that viral-induced wheezing is a common feature in this phenotype, and repeated viral infections can lead to an increased severity and frequency of exacerbations. However, there are currently no effective antiviral therapies or vaccines for rhinovirus, which limits the ability to address the viral component of the disease directly.
Up to 50% of children with severe, recurrent wheezing also have bacterial pathogens like Moraxella catarrhalis and Haemophilus influenzae in their lower airways. For these children, addressing the bacterial infection is the best treatment option to mitigate the wheezing. “We now have something that we can target with antibiotics for those who don’t respond to corticosteroids,” Dr. Saglani said.
Dr. Pijnenburg said that this body of research is helping pulmonary specialists and general pediatricians navigate the complexity of childhood wheezing beyond phenotyping and symptoms. “We need to dive more deeply into those kids with preschool wheezing to see what’s happening in their lungs.”
Dr. Pijnenburg and Dr. Saglani reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
AI in Medicine Sparks Excitement, Concerns From Experts
VIENNA — At the European Respiratory Society (ERS) 2024 Congress, experts discussed the benefits and risks of artificial intelligence (AI) in medicine and explored ethical implications and practical challenges.
Joshua Hatherley, PhD, a postdoctoral fellow at the School of Philosophy and History of Ideas at Aarhus University in Denmark, said the traditional bioethical principles — autonomy, beneficence, nonmaleficence, and justice — remain a crucial framework for assessing ethics regarding the use of AI tools in medicine. However, he said the emerging fifth principle of “explainability” has gained attention due to the unique characteristics of AI systems.
“Everyone is excited about AI right now, but there are many open questions about how much we can trust it and to what extent we can use it,” Ana Catalina Hernandez Padilla, a clinical researcher at the Université de Limoges, France, told this news organization.
Joseph Alderman, MBChB, an AI and digital health clinical research fellow at the Institute of Inflammation and Ageing at the University of Birmingham, UK, said these are undoubtedly exciting times to work in AI and health, but he believes clinicians should be “part of the story” and advocate for AI that is safe, effective, and equitable.
The Pros
Dr. Alderman said AI has huge potential to improve healthcare and patients’ experiences.
One interesting area in which AI is being applied is the informed consent process. Conversational AI models, like large language models, can provide patients with a time-unlimited platform to discuss risks, benefits, and recommendations, potentially improving understanding and patient engagement. AI systems can also predict the preferences of noncommunicative patients by analyzing their social media and medical data, which may improve surrogate decision-making and ensure treatment aligns with patient preferences, Dr. Hatherley explained.
Another significant benefit is AI’s capacity to improve patient outcomes through better resource allocation. For example, AI can help optimize the allocation of hospital beds, leading to more efficient use of resources and improved patient health outcomes.
AI systems can reduce medical errors and enhance diagnosis or treatment plans through large-scale data analysis, leading to faster and more accurate decision-making. It can handle administrative tasks, reducing clinician burnout and allowing healthcare professionals to focus more on patient care.
AI also promises to advance health equity by improving access to quality care in underserved areas. In rural hospitals or developing countries, AI can help fill gaps in clinical expertise, potentially leveling the playing field in access to healthcare.
The Cons
Despite its potential, AI in medicine presents several risks that require careful ethical considerations. One primary concern is the possibility of embedded bias in AI systems.
For example, AI-driven advice from an AI agent may prioritize certain outcomes, such as survival, based on broad standards rather than unique patient values, potentially misaligning with the preferences of patients who value quality of life over longevity. “That may interfere with patients’ autonomous decisions,” Dr. Hatherley said.
AI systems also have limited generalizability. Models trained on a specific patient population may perform poorly when applied to different groups due to changes in demographic or clinical characteristics. This can result in less accurate or inappropriate recommendations in real-world settings. “These technologies work on the very narrow population on which the tool was developed but might not necessarily work in the real world,” said Dr. Alderman.
Another significant risk is algorithmic bias, which can worsen health disparities. AI models trained on biased datasets may perpetuate or exacerbate existing inequities in healthcare delivery, leading to suboptimal care for marginalized populations. “We have evidence of algorithms directly discriminating against people with certain characteristics,” Dr. Alderman said.
AI’s Black Box
AI systems, particularly those utilizing deep learning, often function as “black boxes,” meaning their internal decision-making processes are opaque and difficult to interpret. Dr. Hatherley said this lack of transparency raises significant concerns about trust and accountability in clinical decision-making.
While Explainable AI methods have been developed to offer insights into how these systems generate their recommendations, these explanations frequently fail to capture the reasoning process entirely. Dr. Hatherley explained that this is similar to using a pharmaceutical medicine without a clear understanding of the mechanisms for which it works.
This opacity in AI decision-making can lead to mistrust among clinicians and patients, limiting its effective use in healthcare. “We don’t really know how to interpret the information it provides,” Ms. Hernandez said.
She said while younger clinicians might be more open to testing the waters with AI tools, older practitioners still prefer to trust their own senses while looking at a patient as a whole and observing the evolution of their disease. “They are not just ticking boxes. They interpret all these variables together to make a medical decision,” she said.
“I am really optimistic about the future of AI,” Dr. Hatherley concluded. “There are still many challenges to overcome, but, ultimately, it’s not enough to talk about how AI should be adapted to human beings. We also need to talk about how humans should adapt to AI.”
Dr. Hatherley, Dr. Alderman, and Ms. Hernandez have reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — At the European Respiratory Society (ERS) 2024 Congress, experts discussed the benefits and risks of artificial intelligence (AI) in medicine and explored ethical implications and practical challenges.
Joshua Hatherley, PhD, a postdoctoral fellow at the School of Philosophy and History of Ideas at Aarhus University in Denmark, said the traditional bioethical principles — autonomy, beneficence, nonmaleficence, and justice — remain a crucial framework for assessing ethics regarding the use of AI tools in medicine. However, he said the emerging fifth principle of “explainability” has gained attention due to the unique characteristics of AI systems.
“Everyone is excited about AI right now, but there are many open questions about how much we can trust it and to what extent we can use it,” Ana Catalina Hernandez Padilla, a clinical researcher at the Université de Limoges, France, told this news organization.
Joseph Alderman, MBChB, an AI and digital health clinical research fellow at the Institute of Inflammation and Ageing at the University of Birmingham, UK, said these are undoubtedly exciting times to work in AI and health, but he believes clinicians should be “part of the story” and advocate for AI that is safe, effective, and equitable.
The Pros
Dr. Alderman said AI has huge potential to improve healthcare and patients’ experiences.
One interesting area in which AI is being applied is the informed consent process. Conversational AI models, like large language models, can provide patients with a time-unlimited platform to discuss risks, benefits, and recommendations, potentially improving understanding and patient engagement. AI systems can also predict the preferences of noncommunicative patients by analyzing their social media and medical data, which may improve surrogate decision-making and ensure treatment aligns with patient preferences, Dr. Hatherley explained.
Another significant benefit is AI’s capacity to improve patient outcomes through better resource allocation. For example, AI can help optimize the allocation of hospital beds, leading to more efficient use of resources and improved patient health outcomes.
AI systems can reduce medical errors and enhance diagnosis or treatment plans through large-scale data analysis, leading to faster and more accurate decision-making. It can handle administrative tasks, reducing clinician burnout and allowing healthcare professionals to focus more on patient care.
AI also promises to advance health equity by improving access to quality care in underserved areas. In rural hospitals or developing countries, AI can help fill gaps in clinical expertise, potentially leveling the playing field in access to healthcare.
The Cons
Despite its potential, AI in medicine presents several risks that require careful ethical considerations. One primary concern is the possibility of embedded bias in AI systems.
For example, AI-driven advice from an AI agent may prioritize certain outcomes, such as survival, based on broad standards rather than unique patient values, potentially misaligning with the preferences of patients who value quality of life over longevity. “That may interfere with patients’ autonomous decisions,” Dr. Hatherley said.
AI systems also have limited generalizability. Models trained on a specific patient population may perform poorly when applied to different groups due to changes in demographic or clinical characteristics. This can result in less accurate or inappropriate recommendations in real-world settings. “These technologies work on the very narrow population on which the tool was developed but might not necessarily work in the real world,” said Dr. Alderman.
Another significant risk is algorithmic bias, which can worsen health disparities. AI models trained on biased datasets may perpetuate or exacerbate existing inequities in healthcare delivery, leading to suboptimal care for marginalized populations. “We have evidence of algorithms directly discriminating against people with certain characteristics,” Dr. Alderman said.
AI’s Black Box
AI systems, particularly those utilizing deep learning, often function as “black boxes,” meaning their internal decision-making processes are opaque and difficult to interpret. Dr. Hatherley said this lack of transparency raises significant concerns about trust and accountability in clinical decision-making.
While Explainable AI methods have been developed to offer insights into how these systems generate their recommendations, these explanations frequently fail to capture the reasoning process entirely. Dr. Hatherley explained that this is similar to using a pharmaceutical medicine without a clear understanding of the mechanisms for which it works.
This opacity in AI decision-making can lead to mistrust among clinicians and patients, limiting its effective use in healthcare. “We don’t really know how to interpret the information it provides,” Ms. Hernandez said.
She said while younger clinicians might be more open to testing the waters with AI tools, older practitioners still prefer to trust their own senses while looking at a patient as a whole and observing the evolution of their disease. “They are not just ticking boxes. They interpret all these variables together to make a medical decision,” she said.
“I am really optimistic about the future of AI,” Dr. Hatherley concluded. “There are still many challenges to overcome, but, ultimately, it’s not enough to talk about how AI should be adapted to human beings. We also need to talk about how humans should adapt to AI.”
Dr. Hatherley, Dr. Alderman, and Ms. Hernandez have reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — At the European Respiratory Society (ERS) 2024 Congress, experts discussed the benefits and risks of artificial intelligence (AI) in medicine and explored ethical implications and practical challenges.
Joshua Hatherley, PhD, a postdoctoral fellow at the School of Philosophy and History of Ideas at Aarhus University in Denmark, said the traditional bioethical principles — autonomy, beneficence, nonmaleficence, and justice — remain a crucial framework for assessing ethics regarding the use of AI tools in medicine. However, he said the emerging fifth principle of “explainability” has gained attention due to the unique characteristics of AI systems.
“Everyone is excited about AI right now, but there are many open questions about how much we can trust it and to what extent we can use it,” Ana Catalina Hernandez Padilla, a clinical researcher at the Université de Limoges, France, told this news organization.
Joseph Alderman, MBChB, an AI and digital health clinical research fellow at the Institute of Inflammation and Ageing at the University of Birmingham, UK, said these are undoubtedly exciting times to work in AI and health, but he believes clinicians should be “part of the story” and advocate for AI that is safe, effective, and equitable.
The Pros
Dr. Alderman said AI has huge potential to improve healthcare and patients’ experiences.
One interesting area in which AI is being applied is the informed consent process. Conversational AI models, like large language models, can provide patients with a time-unlimited platform to discuss risks, benefits, and recommendations, potentially improving understanding and patient engagement. AI systems can also predict the preferences of noncommunicative patients by analyzing their social media and medical data, which may improve surrogate decision-making and ensure treatment aligns with patient preferences, Dr. Hatherley explained.
Another significant benefit is AI’s capacity to improve patient outcomes through better resource allocation. For example, AI can help optimize the allocation of hospital beds, leading to more efficient use of resources and improved patient health outcomes.
AI systems can reduce medical errors and enhance diagnosis or treatment plans through large-scale data analysis, leading to faster and more accurate decision-making. It can handle administrative tasks, reducing clinician burnout and allowing healthcare professionals to focus more on patient care.
AI also promises to advance health equity by improving access to quality care in underserved areas. In rural hospitals or developing countries, AI can help fill gaps in clinical expertise, potentially leveling the playing field in access to healthcare.
The Cons
Despite its potential, AI in medicine presents several risks that require careful ethical considerations. One primary concern is the possibility of embedded bias in AI systems.
For example, AI-driven advice from an AI agent may prioritize certain outcomes, such as survival, based on broad standards rather than unique patient values, potentially misaligning with the preferences of patients who value quality of life over longevity. “That may interfere with patients’ autonomous decisions,” Dr. Hatherley said.
AI systems also have limited generalizability. Models trained on a specific patient population may perform poorly when applied to different groups due to changes in demographic or clinical characteristics. This can result in less accurate or inappropriate recommendations in real-world settings. “These technologies work on the very narrow population on which the tool was developed but might not necessarily work in the real world,” said Dr. Alderman.
Another significant risk is algorithmic bias, which can worsen health disparities. AI models trained on biased datasets may perpetuate or exacerbate existing inequities in healthcare delivery, leading to suboptimal care for marginalized populations. “We have evidence of algorithms directly discriminating against people with certain characteristics,” Dr. Alderman said.
AI’s Black Box
AI systems, particularly those utilizing deep learning, often function as “black boxes,” meaning their internal decision-making processes are opaque and difficult to interpret. Dr. Hatherley said this lack of transparency raises significant concerns about trust and accountability in clinical decision-making.
While Explainable AI methods have been developed to offer insights into how these systems generate their recommendations, these explanations frequently fail to capture the reasoning process entirely. Dr. Hatherley explained that this is similar to using a pharmaceutical medicine without a clear understanding of the mechanisms for which it works.
This opacity in AI decision-making can lead to mistrust among clinicians and patients, limiting its effective use in healthcare. “We don’t really know how to interpret the information it provides,” Ms. Hernandez said.
She said while younger clinicians might be more open to testing the waters with AI tools, older practitioners still prefer to trust their own senses while looking at a patient as a whole and observing the evolution of their disease. “They are not just ticking boxes. They interpret all these variables together to make a medical decision,” she said.
“I am really optimistic about the future of AI,” Dr. Hatherley concluded. “There are still many challenges to overcome, but, ultimately, it’s not enough to talk about how AI should be adapted to human beings. We also need to talk about how humans should adapt to AI.”
Dr. Hatherley, Dr. Alderman, and Ms. Hernandez have reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
Survival Rate in Bilateral Lung and Heart-Lung Transplants Comparable
VIENNA — , said researchers presenting at the European Respiratory Society (ERS) 2024 International Congress.
Transplant for end-stage PAH remains an important treatment option. Heart-lung transplantation plummeted from 91.7% to 21.4% between 1991 and 2014. Yet in the United States and Europe, PAH is the second most common reason to perform a heart-lung transplant, said Baharan Zarrabian, DO, a pulmonologist at the Mayo Clinic in Rochester, Minnesota.
Over the past decades, physicians have debated whether to opt for a bilateral lung or a heart-lung transplant. However, there is currently a lack of definitive cardiac indicators to guide the decision between the two procedures in patients with PAH.
While the lung condition has cardiac ramifications, some experts suggest that the heart can repair itself over time after a bilateral lung transplant.
No Survival Difference Between Bilateral Lung and Heart-Lung Transplants
Researchers compared the outcomes of bilateral lung transplantation with those of combined heart-lung transplantation in patients with PAH. They used data from the Organ Procurement and Transplantation Network, focusing on adult patients with PAH without congenital or structural cardiac abnormalities who underwent transplantation between June 2004 and September 2022.
The study included 918 patients, with the majority (84.6%) receiving bilateral lung transplants and 15.4% receiving heart-lung transplants. Pretransplant mean pulmonary arterial pressure and pulmonary vascular resistance were similar between the two groups. However, those who received bilateral lung transplants had higher cardiac output and lower pulmonary capillary wedge pressure than those who received heart-lung transplants. A higher percentage of heart-lung transplant recipients required extracorporeal membrane oxygenation (ECMO) before transplantation, while bilateral lung transplant recipients had longer median ischemic times.
Despite these differences in pretransplant characteristics and surgical factors, researchers found no significant difference in survival outcomes between the two groups at the 1-year and 5-year marks. Similarly, graft survival rates at 1 and 5 years posttransplant did not differ significantly between the two groups.
A higher proportion of patients who received bilateral lung transplant were on ECMO and remained intubated at 72 hours. “That did not translate into a worse outcome later on,” Dr. Zarrabian said.
Cardiac Recovery Post-Bilateral Lung Transplant
Saskia Bos, MD, PhD, a respiratory consultant, lung transplant physician, and transplant pulmonologist at University Hospitals Leuven, Belgium, who was not involved in the study, told this news organization that doctors have historically preferred combined heart-lung transplantation. The decision was motivated by a lack of understanding regarding whether the heart could remodel on its own. “Now we know that the right ventricle, which is the most affected part of the heart in pulmonary hypertension, has a huge ability to readapt to the new situation after just bilateral lung transplantation,” she said.
Bos suggested that in cases where a patient has pulmonary hypertension without any structural heart defects and where the left side of the heart is functioning normally, doctors can opt for a bilateral lung transplant rather than a combined heart-lung transplant. The right ventricle, typically the only part of the heart affected by the condition, can recover once the pulmonary hypertension is addressed through lung transplantation, she explained.
The advantage of bilateral lung transplantation over a heart-lung transplant is that the donor’s heart remains available for someone else.
“The recommendation is that physicians opt for a bilateral lung transplant,” Dr. Zarrabian concluded. “But we need to make that decision on a case-by-case basis because we still don’t know what are the cardiac parameters that we need to look for before the transplant to decide whether or not they should receive a bilateral lung or a heart-lung.”
Dr. Zarrabian and Dr. Bos reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — , said researchers presenting at the European Respiratory Society (ERS) 2024 International Congress.
Transplant for end-stage PAH remains an important treatment option. Heart-lung transplantation plummeted from 91.7% to 21.4% between 1991 and 2014. Yet in the United States and Europe, PAH is the second most common reason to perform a heart-lung transplant, said Baharan Zarrabian, DO, a pulmonologist at the Mayo Clinic in Rochester, Minnesota.
Over the past decades, physicians have debated whether to opt for a bilateral lung or a heart-lung transplant. However, there is currently a lack of definitive cardiac indicators to guide the decision between the two procedures in patients with PAH.
While the lung condition has cardiac ramifications, some experts suggest that the heart can repair itself over time after a bilateral lung transplant.
No Survival Difference Between Bilateral Lung and Heart-Lung Transplants
Researchers compared the outcomes of bilateral lung transplantation with those of combined heart-lung transplantation in patients with PAH. They used data from the Organ Procurement and Transplantation Network, focusing on adult patients with PAH without congenital or structural cardiac abnormalities who underwent transplantation between June 2004 and September 2022.
The study included 918 patients, with the majority (84.6%) receiving bilateral lung transplants and 15.4% receiving heart-lung transplants. Pretransplant mean pulmonary arterial pressure and pulmonary vascular resistance were similar between the two groups. However, those who received bilateral lung transplants had higher cardiac output and lower pulmonary capillary wedge pressure than those who received heart-lung transplants. A higher percentage of heart-lung transplant recipients required extracorporeal membrane oxygenation (ECMO) before transplantation, while bilateral lung transplant recipients had longer median ischemic times.
Despite these differences in pretransplant characteristics and surgical factors, researchers found no significant difference in survival outcomes between the two groups at the 1-year and 5-year marks. Similarly, graft survival rates at 1 and 5 years posttransplant did not differ significantly between the two groups.
A higher proportion of patients who received bilateral lung transplant were on ECMO and remained intubated at 72 hours. “That did not translate into a worse outcome later on,” Dr. Zarrabian said.
Cardiac Recovery Post-Bilateral Lung Transplant
Saskia Bos, MD, PhD, a respiratory consultant, lung transplant physician, and transplant pulmonologist at University Hospitals Leuven, Belgium, who was not involved in the study, told this news organization that doctors have historically preferred combined heart-lung transplantation. The decision was motivated by a lack of understanding regarding whether the heart could remodel on its own. “Now we know that the right ventricle, which is the most affected part of the heart in pulmonary hypertension, has a huge ability to readapt to the new situation after just bilateral lung transplantation,” she said.
Bos suggested that in cases where a patient has pulmonary hypertension without any structural heart defects and where the left side of the heart is functioning normally, doctors can opt for a bilateral lung transplant rather than a combined heart-lung transplant. The right ventricle, typically the only part of the heart affected by the condition, can recover once the pulmonary hypertension is addressed through lung transplantation, she explained.
The advantage of bilateral lung transplantation over a heart-lung transplant is that the donor’s heart remains available for someone else.
“The recommendation is that physicians opt for a bilateral lung transplant,” Dr. Zarrabian concluded. “But we need to make that decision on a case-by-case basis because we still don’t know what are the cardiac parameters that we need to look for before the transplant to decide whether or not they should receive a bilateral lung or a heart-lung.”
Dr. Zarrabian and Dr. Bos reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — , said researchers presenting at the European Respiratory Society (ERS) 2024 International Congress.
Transplant for end-stage PAH remains an important treatment option. Heart-lung transplantation plummeted from 91.7% to 21.4% between 1991 and 2014. Yet in the United States and Europe, PAH is the second most common reason to perform a heart-lung transplant, said Baharan Zarrabian, DO, a pulmonologist at the Mayo Clinic in Rochester, Minnesota.
Over the past decades, physicians have debated whether to opt for a bilateral lung or a heart-lung transplant. However, there is currently a lack of definitive cardiac indicators to guide the decision between the two procedures in patients with PAH.
While the lung condition has cardiac ramifications, some experts suggest that the heart can repair itself over time after a bilateral lung transplant.
No Survival Difference Between Bilateral Lung and Heart-Lung Transplants
Researchers compared the outcomes of bilateral lung transplantation with those of combined heart-lung transplantation in patients with PAH. They used data from the Organ Procurement and Transplantation Network, focusing on adult patients with PAH without congenital or structural cardiac abnormalities who underwent transplantation between June 2004 and September 2022.
The study included 918 patients, with the majority (84.6%) receiving bilateral lung transplants and 15.4% receiving heart-lung transplants. Pretransplant mean pulmonary arterial pressure and pulmonary vascular resistance were similar between the two groups. However, those who received bilateral lung transplants had higher cardiac output and lower pulmonary capillary wedge pressure than those who received heart-lung transplants. A higher percentage of heart-lung transplant recipients required extracorporeal membrane oxygenation (ECMO) before transplantation, while bilateral lung transplant recipients had longer median ischemic times.
Despite these differences in pretransplant characteristics and surgical factors, researchers found no significant difference in survival outcomes between the two groups at the 1-year and 5-year marks. Similarly, graft survival rates at 1 and 5 years posttransplant did not differ significantly between the two groups.
A higher proportion of patients who received bilateral lung transplant were on ECMO and remained intubated at 72 hours. “That did not translate into a worse outcome later on,” Dr. Zarrabian said.
Cardiac Recovery Post-Bilateral Lung Transplant
Saskia Bos, MD, PhD, a respiratory consultant, lung transplant physician, and transplant pulmonologist at University Hospitals Leuven, Belgium, who was not involved in the study, told this news organization that doctors have historically preferred combined heart-lung transplantation. The decision was motivated by a lack of understanding regarding whether the heart could remodel on its own. “Now we know that the right ventricle, which is the most affected part of the heart in pulmonary hypertension, has a huge ability to readapt to the new situation after just bilateral lung transplantation,” she said.
Bos suggested that in cases where a patient has pulmonary hypertension without any structural heart defects and where the left side of the heart is functioning normally, doctors can opt for a bilateral lung transplant rather than a combined heart-lung transplant. The right ventricle, typically the only part of the heart affected by the condition, can recover once the pulmonary hypertension is addressed through lung transplantation, she explained.
The advantage of bilateral lung transplantation over a heart-lung transplant is that the donor’s heart remains available for someone else.
“The recommendation is that physicians opt for a bilateral lung transplant,” Dr. Zarrabian concluded. “But we need to make that decision on a case-by-case basis because we still don’t know what are the cardiac parameters that we need to look for before the transplant to decide whether or not they should receive a bilateral lung or a heart-lung.”
Dr. Zarrabian and Dr. Bos reported no relevant financial relationships.
A version of this article appeared on Medscape.com.
Controversy Surrounds Optimal Treatment for High-Risk Pulmonary Embolism
VIENNA — The optimal course of treatment when managing acute, high-risk pulmonary embolism (PE) remains a contentious topic among respiratory specialists.
Systemic thrombolysis, specifically using recombinant tissue plasminogen activator (rtPA), is the current gold standard treatment for high-risk PE. However, the real-world application is less straightforward due to patient complexities.
Here at the European Respiratory Society (ERS) 2024 Congress, respiratory specialists presented contrasting viewpoints and the latest evidence on each side of the issue to provide a comprehensive framework for navigating the complex decision-making process required for effective treatment.
“High-risk PE is a mechanical problem and thus needs a mechanical solution,” said Parth M. Rali, MD, an associate professor in thoracic medicine and surgery at the Lewis Katz School of Medicine at Temple University, Philadelphia.
“The marketing on some of the mechanical techniques is very impressive,” said Olivier Sanchez, MD, a pulmonologist in the Department of Pneumology and Intensive Care at the Georges Pompidou European Hospital in France. “But what is the evidence of such treatment in the setting of pulmonary embolism?”
The Case Against rtPA as the Standard of Care
High-risk PE typically involves hemodynamically unstable patients presenting with conditions such as low blood pressure, cardiac arrest, or the need for mechanical circulatory support. There is a spectrum of severity within high-risk PE, making it a complex condition to manage, especially since many patients have comorbidities like anemia or active cancer, complicating treatment. “It’s a very dynamic and fluid condition, and we can’t take for granted that rtPA is a standard of care,” Dr. Rali said.
Alternative treatments such as catheter-directed therapies, extracorporeal membrane oxygenation (ECMO), and surgical embolectomy are emerging as promising options, especially for patients who do not respond to or cannot receive rtPA. Mechanical treatments offer benefits in reducing clot burden and stabilizing patients, but they come with their own challenges.
ECMO can stabilize patients who are in shock or cardiac arrest, buying time for the clot to resolve or for further interventions like surgery or catheter-based treatments, said Dr. Rali. However, it is an invasive procedure requiring cannulation of large blood vessels, often involving significant resources and expertise.
Catheter-directed thrombolysis is a minimally invasive technique where a catheter is inserted directly into the pulmonary artery to deliver thrombolytic drugs at lower doses. This method allows for more targeted treatment of the clot, reducing the risk for systemic bleeding that comes with higher doses of thrombolytic agents used in systemic therapy, Dr. Rali explained.
Dr. Rali reported results from the FLAME study, which investigated the effectiveness of FlowTriever mechanical thrombectomy compared with conventional therapies for high-risk PE. This prospective, multicenter observational study enrolled 53 patients in the FlowTriever arm and 61 in the context arm, which included patients treated with systemic thrombolysis or anticoagulation. The primary endpoint, a composite of adverse in-hospital outcomes, was reached in 17% of FlowTriever patients, significantly lower than the 32% performance goal and the 63.9% rate in the context arm. In-hospital mortality was dramatically lower in the FlowTriever arm (1.9%) compared to the context arm (29.5%).
When catheter-based treatment fails, surgical pulmonary embolectomy is a last-resort option. “Only a minority of the high-risk PE [patients] would qualify for rtPA without harmful side effects,” Dr. Rali concluded. “So think wise before you pull your trigger.”
rtPA Not a Matter of the Past
In high-risk PE, the therapeutic priority is rapid hemodynamic stabilization and restoration of pulmonary blood flow to prevent cardiovascular collapse. Systemic thrombolysis acts quickly, reducing pulmonary vascular resistance and obstruction within hours, said Dr. Sanchez.
Presenting at the ERS Congress, he reported numerous studies, including 15 randomized controlled trials that demonstrated its effectiveness in high-risk PE. The PEITHO trial, in particular, demonstrated the ability of systemic thrombolysis to reduce all-cause mortality and hemodynamic collapse within 7 days.
However, this benefit comes at the cost of increased bleeding risk, including a 10% rate of major bleeding and a 2% risk for intracranial hemorrhage. “These data come from old studies using invasive diagnostic procedures, and with current diagnostic procedures, the rate of bleeding is probably lower,” Dr. Sanchez said. The risk of bleeding is also related to the type of thrombolytic agent, with tenecteplase being strongly associated with a higher risk of bleeding, while alteplase shows no increase in the risk of major bleeding, he added. New strategies like reduced-dose thrombolysis offer comparable efficacy and improved safety, as demonstrated in ongoing trials like PEITHO-3, which aim to optimize the balance between efficacy and bleeding risk. Dr. Sanchez is the lead investigator of the PEITHO-3 study.
While rtPA might not be optimal for all patients, Dr. Sanchez thinks there is not enough evidence to replace it as a first-line treatment.
Existing studies on catheter-directed therapies often focus on surrogate endpoints, such as right-to-left ventricular ratio changes, rather than clinical outcomes like mortality, he said. Retrospective data suggest that catheter-directed therapies may reduce in-hospital mortality compared with systemic therapies, but they also increase the risk of intracranial bleeding, post-procedure complications, and device-related events.
Sanchez mentioned the same FLAME study described by Dr. Rali, which reported a 23% rate of device-related complications and 11% major bleeding in patients treated with catheter-directed therapies.
“Systemic thrombolysis remains the first treatment of choice,” Dr. Sanchez concluded. “The use of catheter-directed treatment should be discussed as an alternative in case of contraindications.”
The Debate Continues
Numerous ongoing clinical studies, such as the FLARE trial, will address gaps in evidence and refine treatment protocols, potentially reshaping the standard of care in high-risk PE in the near future by providing new data on the efficacy and safety of existing and emerging therapies.
“The coming data will make it clearer what the best option is,” said Thamer Al Khouzaie, MD, a pulmonary medicine consultant at Johns Hopkins Aramco Healthcare in Dhahran, Saudi Arabia. For now, he said, systemic thrombolysis remains the best option for most patients because it is widely available, easily administered with intravenous infusion, and at a limited cost. Catheter-directed treatment and surgical options are only available in specialized centers, require expertise and training, and are also very expensive.
Dr. Rali, Dr. Sanchez, and Dr. Khouzaie report no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — The optimal course of treatment when managing acute, high-risk pulmonary embolism (PE) remains a contentious topic among respiratory specialists.
Systemic thrombolysis, specifically using recombinant tissue plasminogen activator (rtPA), is the current gold standard treatment for high-risk PE. However, the real-world application is less straightforward due to patient complexities.
Here at the European Respiratory Society (ERS) 2024 Congress, respiratory specialists presented contrasting viewpoints and the latest evidence on each side of the issue to provide a comprehensive framework for navigating the complex decision-making process required for effective treatment.
“High-risk PE is a mechanical problem and thus needs a mechanical solution,” said Parth M. Rali, MD, an associate professor in thoracic medicine and surgery at the Lewis Katz School of Medicine at Temple University, Philadelphia.
“The marketing on some of the mechanical techniques is very impressive,” said Olivier Sanchez, MD, a pulmonologist in the Department of Pneumology and Intensive Care at the Georges Pompidou European Hospital in France. “But what is the evidence of such treatment in the setting of pulmonary embolism?”
The Case Against rtPA as the Standard of Care
High-risk PE typically involves hemodynamically unstable patients presenting with conditions such as low blood pressure, cardiac arrest, or the need for mechanical circulatory support. There is a spectrum of severity within high-risk PE, making it a complex condition to manage, especially since many patients have comorbidities like anemia or active cancer, complicating treatment. “It’s a very dynamic and fluid condition, and we can’t take for granted that rtPA is a standard of care,” Dr. Rali said.
Alternative treatments such as catheter-directed therapies, extracorporeal membrane oxygenation (ECMO), and surgical embolectomy are emerging as promising options, especially for patients who do not respond to or cannot receive rtPA. Mechanical treatments offer benefits in reducing clot burden and stabilizing patients, but they come with their own challenges.
ECMO can stabilize patients who are in shock or cardiac arrest, buying time for the clot to resolve or for further interventions like surgery or catheter-based treatments, said Dr. Rali. However, it is an invasive procedure requiring cannulation of large blood vessels, often involving significant resources and expertise.
Catheter-directed thrombolysis is a minimally invasive technique where a catheter is inserted directly into the pulmonary artery to deliver thrombolytic drugs at lower doses. This method allows for more targeted treatment of the clot, reducing the risk for systemic bleeding that comes with higher doses of thrombolytic agents used in systemic therapy, Dr. Rali explained.
Dr. Rali reported results from the FLAME study, which investigated the effectiveness of FlowTriever mechanical thrombectomy compared with conventional therapies for high-risk PE. This prospective, multicenter observational study enrolled 53 patients in the FlowTriever arm and 61 in the context arm, which included patients treated with systemic thrombolysis or anticoagulation. The primary endpoint, a composite of adverse in-hospital outcomes, was reached in 17% of FlowTriever patients, significantly lower than the 32% performance goal and the 63.9% rate in the context arm. In-hospital mortality was dramatically lower in the FlowTriever arm (1.9%) compared to the context arm (29.5%).
When catheter-based treatment fails, surgical pulmonary embolectomy is a last-resort option. “Only a minority of the high-risk PE [patients] would qualify for rtPA without harmful side effects,” Dr. Rali concluded. “So think wise before you pull your trigger.”
rtPA Not a Matter of the Past
In high-risk PE, the therapeutic priority is rapid hemodynamic stabilization and restoration of pulmonary blood flow to prevent cardiovascular collapse. Systemic thrombolysis acts quickly, reducing pulmonary vascular resistance and obstruction within hours, said Dr. Sanchez.
Presenting at the ERS Congress, he reported numerous studies, including 15 randomized controlled trials that demonstrated its effectiveness in high-risk PE. The PEITHO trial, in particular, demonstrated the ability of systemic thrombolysis to reduce all-cause mortality and hemodynamic collapse within 7 days.
However, this benefit comes at the cost of increased bleeding risk, including a 10% rate of major bleeding and a 2% risk for intracranial hemorrhage. “These data come from old studies using invasive diagnostic procedures, and with current diagnostic procedures, the rate of bleeding is probably lower,” Dr. Sanchez said. The risk of bleeding is also related to the type of thrombolytic agent, with tenecteplase being strongly associated with a higher risk of bleeding, while alteplase shows no increase in the risk of major bleeding, he added. New strategies like reduced-dose thrombolysis offer comparable efficacy and improved safety, as demonstrated in ongoing trials like PEITHO-3, which aim to optimize the balance between efficacy and bleeding risk. Dr. Sanchez is the lead investigator of the PEITHO-3 study.
While rtPA might not be optimal for all patients, Dr. Sanchez thinks there is not enough evidence to replace it as a first-line treatment.
Existing studies on catheter-directed therapies often focus on surrogate endpoints, such as right-to-left ventricular ratio changes, rather than clinical outcomes like mortality, he said. Retrospective data suggest that catheter-directed therapies may reduce in-hospital mortality compared with systemic therapies, but they also increase the risk of intracranial bleeding, post-procedure complications, and device-related events.
Sanchez mentioned the same FLAME study described by Dr. Rali, which reported a 23% rate of device-related complications and 11% major bleeding in patients treated with catheter-directed therapies.
“Systemic thrombolysis remains the first treatment of choice,” Dr. Sanchez concluded. “The use of catheter-directed treatment should be discussed as an alternative in case of contraindications.”
The Debate Continues
Numerous ongoing clinical studies, such as the FLARE trial, will address gaps in evidence and refine treatment protocols, potentially reshaping the standard of care in high-risk PE in the near future by providing new data on the efficacy and safety of existing and emerging therapies.
“The coming data will make it clearer what the best option is,” said Thamer Al Khouzaie, MD, a pulmonary medicine consultant at Johns Hopkins Aramco Healthcare in Dhahran, Saudi Arabia. For now, he said, systemic thrombolysis remains the best option for most patients because it is widely available, easily administered with intravenous infusion, and at a limited cost. Catheter-directed treatment and surgical options are only available in specialized centers, require expertise and training, and are also very expensive.
Dr. Rali, Dr. Sanchez, and Dr. Khouzaie report no relevant financial relationships.
A version of this article appeared on Medscape.com.
VIENNA — The optimal course of treatment when managing acute, high-risk pulmonary embolism (PE) remains a contentious topic among respiratory specialists.
Systemic thrombolysis, specifically using recombinant tissue plasminogen activator (rtPA), is the current gold standard treatment for high-risk PE. However, the real-world application is less straightforward due to patient complexities.
Here at the European Respiratory Society (ERS) 2024 Congress, respiratory specialists presented contrasting viewpoints and the latest evidence on each side of the issue to provide a comprehensive framework for navigating the complex decision-making process required for effective treatment.
“High-risk PE is a mechanical problem and thus needs a mechanical solution,” said Parth M. Rali, MD, an associate professor in thoracic medicine and surgery at the Lewis Katz School of Medicine at Temple University, Philadelphia.
“The marketing on some of the mechanical techniques is very impressive,” said Olivier Sanchez, MD, a pulmonologist in the Department of Pneumology and Intensive Care at the Georges Pompidou European Hospital in France. “But what is the evidence of such treatment in the setting of pulmonary embolism?”
The Case Against rtPA as the Standard of Care
High-risk PE typically involves hemodynamically unstable patients presenting with conditions such as low blood pressure, cardiac arrest, or the need for mechanical circulatory support. There is a spectrum of severity within high-risk PE, making it a complex condition to manage, especially since many patients have comorbidities like anemia or active cancer, complicating treatment. “It’s a very dynamic and fluid condition, and we can’t take for granted that rtPA is a standard of care,” Dr. Rali said.
Alternative treatments such as catheter-directed therapies, extracorporeal membrane oxygenation (ECMO), and surgical embolectomy are emerging as promising options, especially for patients who do not respond to or cannot receive rtPA. Mechanical treatments offer benefits in reducing clot burden and stabilizing patients, but they come with their own challenges.
ECMO can stabilize patients who are in shock or cardiac arrest, buying time for the clot to resolve or for further interventions like surgery or catheter-based treatments, said Dr. Rali. However, it is an invasive procedure requiring cannulation of large blood vessels, often involving significant resources and expertise.
Catheter-directed thrombolysis is a minimally invasive technique where a catheter is inserted directly into the pulmonary artery to deliver thrombolytic drugs at lower doses. This method allows for more targeted treatment of the clot, reducing the risk for systemic bleeding that comes with higher doses of thrombolytic agents used in systemic therapy, Dr. Rali explained.
Dr. Rali reported results from the FLAME study, which investigated the effectiveness of FlowTriever mechanical thrombectomy compared with conventional therapies for high-risk PE. This prospective, multicenter observational study enrolled 53 patients in the FlowTriever arm and 61 in the context arm, which included patients treated with systemic thrombolysis or anticoagulation. The primary endpoint, a composite of adverse in-hospital outcomes, was reached in 17% of FlowTriever patients, significantly lower than the 32% performance goal and the 63.9% rate in the context arm. In-hospital mortality was dramatically lower in the FlowTriever arm (1.9%) compared to the context arm (29.5%).
When catheter-based treatment fails, surgical pulmonary embolectomy is a last-resort option. “Only a minority of the high-risk PE [patients] would qualify for rtPA without harmful side effects,” Dr. Rali concluded. “So think wise before you pull your trigger.”
rtPA Not a Matter of the Past
In high-risk PE, the therapeutic priority is rapid hemodynamic stabilization and restoration of pulmonary blood flow to prevent cardiovascular collapse. Systemic thrombolysis acts quickly, reducing pulmonary vascular resistance and obstruction within hours, said Dr. Sanchez.
Presenting at the ERS Congress, he reported numerous studies, including 15 randomized controlled trials that demonstrated its effectiveness in high-risk PE. The PEITHO trial, in particular, demonstrated the ability of systemic thrombolysis to reduce all-cause mortality and hemodynamic collapse within 7 days.
However, this benefit comes at the cost of increased bleeding risk, including a 10% rate of major bleeding and a 2% risk for intracranial hemorrhage. “These data come from old studies using invasive diagnostic procedures, and with current diagnostic procedures, the rate of bleeding is probably lower,” Dr. Sanchez said. The risk of bleeding is also related to the type of thrombolytic agent, with tenecteplase being strongly associated with a higher risk of bleeding, while alteplase shows no increase in the risk of major bleeding, he added. New strategies like reduced-dose thrombolysis offer comparable efficacy and improved safety, as demonstrated in ongoing trials like PEITHO-3, which aim to optimize the balance between efficacy and bleeding risk. Dr. Sanchez is the lead investigator of the PEITHO-3 study.
While rtPA might not be optimal for all patients, Dr. Sanchez thinks there is not enough evidence to replace it as a first-line treatment.
Existing studies on catheter-directed therapies often focus on surrogate endpoints, such as right-to-left ventricular ratio changes, rather than clinical outcomes like mortality, he said. Retrospective data suggest that catheter-directed therapies may reduce in-hospital mortality compared with systemic therapies, but they also increase the risk of intracranial bleeding, post-procedure complications, and device-related events.
Sanchez mentioned the same FLAME study described by Dr. Rali, which reported a 23% rate of device-related complications and 11% major bleeding in patients treated with catheter-directed therapies.
“Systemic thrombolysis remains the first treatment of choice,” Dr. Sanchez concluded. “The use of catheter-directed treatment should be discussed as an alternative in case of contraindications.”
The Debate Continues
Numerous ongoing clinical studies, such as the FLARE trial, will address gaps in evidence and refine treatment protocols, potentially reshaping the standard of care in high-risk PE in the near future by providing new data on the efficacy and safety of existing and emerging therapies.
“The coming data will make it clearer what the best option is,” said Thamer Al Khouzaie, MD, a pulmonary medicine consultant at Johns Hopkins Aramco Healthcare in Dhahran, Saudi Arabia. For now, he said, systemic thrombolysis remains the best option for most patients because it is widely available, easily administered with intravenous infusion, and at a limited cost. Catheter-directed treatment and surgical options are only available in specialized centers, require expertise and training, and are also very expensive.
Dr. Rali, Dr. Sanchez, and Dr. Khouzaie report no relevant financial relationships.
A version of this article appeared on Medscape.com.
Research Promises Better Diabetic Retinopathy Management
STOCKHOLM — At the American Society of Retina Specialists (ASRS) 2024 Annual Meeting, researchers discussed how insights into potential risk factors and new treatments could improve outcomes for patients with diabetic retinopathy.
Jennifer Lim, MD, an ophthalmologist and director of the Retina Service at the University of Illinois Hospital & Health Sciences System in Chicago, told this news organization that emerging approaches to treating diabetic retinopathy offer hope because they address the root causes of the disease beyond just targeting vascular endothelial growth factor (VEGF). She said innovative methods and add-on treatments could lead to more durable and effective drugs.
Exploration of risk factors and treatment options for diabetic retinopathy could lead to more effective management strategies for the condition, agreed David Boyer, MD, an ophthalmologist at Retina Vitreous Associates Medical Group in Los Angeles, speaking with this news organization.
Risk Factors for Diabetic Retinopathy
Sodium-glucose cotransporter 2 (SGLT2) inhibitors and glucagon-like peptide 1 receptor agonists (GLP-1 RAs) have gained popularity because of their benefits beyond glycemic control, including weight loss, and cardiovascular and kidney protection. However, the impact of these medications on vision-threatening retinal complications is not fully understood. “There has always been a question about whether these newer diabetes medications might exacerbate diabetic eye disease,” said Dr. Boyer.
In a retrospective observational study, researchers included adults with type 2 diabetes and moderate cardiovascular disease risk who had no history of advanced diabetic retinal complications. These patients initiated treatment with GLP-1 RA, SGLT2 inhibitors, DPP-4 inhibitors, or sulfonylureas. The study used inverse probability of treatment weighting to mimic randomization and compared the time to the first treatment for diabetic macular edema or proliferative diabetic retinopathy across the treatment groups.
Results, presented by Andrew J. Barkmeier, MD, an associate professor of ophthalmology at the Mayo Clinic, showed that among 371,698 patients, those who initiated therapy with SGLT2 inhibitors had a lower risk of requiring treatment for sight-threatening retinopathy compared with those using other medication classes. GLP-1 RA did not increase retinopathy risk relative to dipeptidyl peptidase 4 inhibitors and sulfonylurea medications.
“[This study] told us that we do have to keep an eye on patients’ retinopathy when they start on these new inhibitors. But the progression is minimal and, overall, I think most people today favor keeping blood sugar levels as good as possible,” said Dr. Boyer, who was not involved in the study.
Another factor that might increase diabetic retinopathy progression is obstructive sleep apnea. This underdiagnosed condition is linked to several health issues, including dementia, stroke, and myocardial infarctions. Although not easily treated, obstructive sleep apnea is manageable, Dr. Boyer explained.
Researchers utilized the TriNetX electronic health records research network to identify patients with nonproliferative diabetic retinopathy, both with and without obstructive sleep apnea.
The results, presented by Ehsan Rahimy, MD, a retinal specialist at Palo Alto Medical Foundation and a professor at Stanford University, showed that patients with obstructive sleep apnea had a significantly higher risk of progressing to proliferative diabetic retinopathy and developing new-onset diabetic macular edema. These patients were more likely to require ocular interventions, such as intravitreal injections and laser photocoagulation. They also had greater risks for stroke, myocardial infarction, and death compared with those who did not have obstructive sleep apnea.
“It was good to bring this to everybody’s attention,” said Dr. Boyer, who was not involved in the study. “It’s an easy question to ask someone if they snore.”
New Treatments on the Horizon
In another presentation, Nathan C. Steinle, MD, of California Retina Consultants, presented a study that assessed the durability of response to sozinibercept in patients with retinal vascular diseases. This novel therapeutic agent is designed to inhibit VEGF-C and VEGF-D in conditions where VEGF-A suppression alone is insufficient.
Sozinibercept was combined with standard anti–VEGF-A therapies such as ranibizumab or aflibercept. It involved a prospective, post hoc analysis of two phase 1b, open-label, dose-escalation studies, including 40 patients with neovascular age-related macular degeneration (nAMD; 31 patients) or diabetic macular edema (nine patients). These patients, either treatment-naive or previously treated, received three intravitreal injections of ranibizumab or aflibercept in combination with sozinibercept at various doses.
Results indicated that sozinibercept combination therapy was well tolerated, with no dose-limiting toxicities. In treatment-naive nAMD patients, the mean best-corrected visual acuity (BCVA) improved significantly from baseline at months 3 and 6. Previously treated nAMD patients also showed BCVA improvements, although to a lesser extent. For patients with persistent diabetic macular edema, switching to sozinibercept plus aflibercept resulted in notable BCVA gains. The mean time to requiring retreatment was longer in treatment-naive patients than in those previously treated, indicating a durable response.
“Combination therapy with sozinibercept is going to be really important,” said Dr. Lim, who was not involved in the study, “because it attacks with a dual mechanism of action.”
Oral agents promise a potentially easier alternative for patients compared with frequent injections. CU06-1004 is a novel orally administered endothelial dysfunction blocker that has shown promise in stabilizing damaged capillaries, reducing abnormal angiogenesis, and inhibiting inflammatory activation in preclinical studies. “CU06 is very interesting to me because by preventing endothelial loss, it gets to the pathophysiology of why the blood vessels break down,” Dr. Lim said.
In a proof-of-concept phase 2a, multicenter, open-label, parallel-group trial, investigators randomly assigned 67 patients with diabetic macular edema to receive 100 mg, 200 mg, or 300 mg of CU06-1004 once daily for 12 weeks, followed by a 4-week follow-up.
Results presented by Victor Gonzalez, MD, of Valley Retina Institute in Texas, indicated that the oral agent improved BCVA, stabilized central subfield thickness, and showed positive anatomical changes in optical coherence tomography images. CU06-1004 was well tolerated, with no drug-related serious adverse events.
“The number [of patients] was very small, and we will need a much longer, larger trial to see if [CU06-1004] has benefits long term,” said Dr. Boyer, who was not involved in the study. “But I think we’re all very excited if we can find an oral agent for treating diabetic retinopathy. It would be easier for the patient to take a pill than having to come in for injections.”
The sustained-release axitinib implant, OTX-TKI, is also generating significant interest, particularly for nonproliferative diabetic retinopathy. Axitinib, a tyrosine kinase inhibitor (TKI), targets signaling pathways crucial in cellular processes, providing a novel approach to managing diseases where traditional therapies might fall short. Unlike traditional anti-VEGF treatments that focus solely on cytokine levels, TKIs block the activation of signaling pathways, preventing downstream signaling regardless of cytokine levels. This mechanism is particularly important because it effectively inhibits disease progression even if levels of VEGF are high, Dr. Lim explained.
In the phase 1 HELIOS trial, OTX-TKI was assessed in patients with nonproliferative diabetic retinopathy. This multicenter, double-masked, parallel-group clinical study included 21 patients who had not received anti-VEGF treatment, dexamethasone intravitreal implants in the previous 12 months, or intraocular steroid injections in the prior 4 months. Patients were randomly assigned to receive either OTX-TKI or sham treatment.
Results presented by Dilsher S. Dhoot, MD, of California Retina Consultants, indicated that OTX-TKI was generally well tolerated, with no serious ocular adverse events. At 48 weeks, 46.2% of eyes treated with OTX-TKI showed a 1- or 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) compared with none in the sham arm. Additionally, no eyes treated with OTX-TKI experienced a worsening on the DRSS, whereas 25% of eyes in the sham arm did. Vision-threatening complications, such as proliferative diabetic retinopathy or diabetic macular edema, developed in 37.5% of the sham group but in none of the OTX-TKI treated eyes. A single injection of OTX-TKI provided durable DRSS improvement for up to 48 weeks, with no patients in either arm requiring rescue therapy.
“This is a really exciting add-on treatment,” Dr. Lim said, who was not involved in the study. She explained that it is initially necessary to control the disease with standard treatments, because TKIs may take longer to exhibit their effects. Once the disease is stabilized, TKIs can be used alongside other therapies, potentially reducing the reliance on frequent anti-VEGF injections. “These are preliminary results, but that’s the hope going forward.”
Dr. Lim and Dr. Boyer report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
STOCKHOLM — At the American Society of Retina Specialists (ASRS) 2024 Annual Meeting, researchers discussed how insights into potential risk factors and new treatments could improve outcomes for patients with diabetic retinopathy.
Jennifer Lim, MD, an ophthalmologist and director of the Retina Service at the University of Illinois Hospital & Health Sciences System in Chicago, told this news organization that emerging approaches to treating diabetic retinopathy offer hope because they address the root causes of the disease beyond just targeting vascular endothelial growth factor (VEGF). She said innovative methods and add-on treatments could lead to more durable and effective drugs.
Exploration of risk factors and treatment options for diabetic retinopathy could lead to more effective management strategies for the condition, agreed David Boyer, MD, an ophthalmologist at Retina Vitreous Associates Medical Group in Los Angeles, speaking with this news organization.
Risk Factors for Diabetic Retinopathy
Sodium-glucose cotransporter 2 (SGLT2) inhibitors and glucagon-like peptide 1 receptor agonists (GLP-1 RAs) have gained popularity because of their benefits beyond glycemic control, including weight loss, and cardiovascular and kidney protection. However, the impact of these medications on vision-threatening retinal complications is not fully understood. “There has always been a question about whether these newer diabetes medications might exacerbate diabetic eye disease,” said Dr. Boyer.
In a retrospective observational study, researchers included adults with type 2 diabetes and moderate cardiovascular disease risk who had no history of advanced diabetic retinal complications. These patients initiated treatment with GLP-1 RA, SGLT2 inhibitors, DPP-4 inhibitors, or sulfonylureas. The study used inverse probability of treatment weighting to mimic randomization and compared the time to the first treatment for diabetic macular edema or proliferative diabetic retinopathy across the treatment groups.
Results, presented by Andrew J. Barkmeier, MD, an associate professor of ophthalmology at the Mayo Clinic, showed that among 371,698 patients, those who initiated therapy with SGLT2 inhibitors had a lower risk of requiring treatment for sight-threatening retinopathy compared with those using other medication classes. GLP-1 RA did not increase retinopathy risk relative to dipeptidyl peptidase 4 inhibitors and sulfonylurea medications.
“[This study] told us that we do have to keep an eye on patients’ retinopathy when they start on these new inhibitors. But the progression is minimal and, overall, I think most people today favor keeping blood sugar levels as good as possible,” said Dr. Boyer, who was not involved in the study.
Another factor that might increase diabetic retinopathy progression is obstructive sleep apnea. This underdiagnosed condition is linked to several health issues, including dementia, stroke, and myocardial infarctions. Although not easily treated, obstructive sleep apnea is manageable, Dr. Boyer explained.
Researchers utilized the TriNetX electronic health records research network to identify patients with nonproliferative diabetic retinopathy, both with and without obstructive sleep apnea.
The results, presented by Ehsan Rahimy, MD, a retinal specialist at Palo Alto Medical Foundation and a professor at Stanford University, showed that patients with obstructive sleep apnea had a significantly higher risk of progressing to proliferative diabetic retinopathy and developing new-onset diabetic macular edema. These patients were more likely to require ocular interventions, such as intravitreal injections and laser photocoagulation. They also had greater risks for stroke, myocardial infarction, and death compared with those who did not have obstructive sleep apnea.
“It was good to bring this to everybody’s attention,” said Dr. Boyer, who was not involved in the study. “It’s an easy question to ask someone if they snore.”
New Treatments on the Horizon
In another presentation, Nathan C. Steinle, MD, of California Retina Consultants, presented a study that assessed the durability of response to sozinibercept in patients with retinal vascular diseases. This novel therapeutic agent is designed to inhibit VEGF-C and VEGF-D in conditions where VEGF-A suppression alone is insufficient.
Sozinibercept was combined with standard anti–VEGF-A therapies such as ranibizumab or aflibercept. It involved a prospective, post hoc analysis of two phase 1b, open-label, dose-escalation studies, including 40 patients with neovascular age-related macular degeneration (nAMD; 31 patients) or diabetic macular edema (nine patients). These patients, either treatment-naive or previously treated, received three intravitreal injections of ranibizumab or aflibercept in combination with sozinibercept at various doses.
Results indicated that sozinibercept combination therapy was well tolerated, with no dose-limiting toxicities. In treatment-naive nAMD patients, the mean best-corrected visual acuity (BCVA) improved significantly from baseline at months 3 and 6. Previously treated nAMD patients also showed BCVA improvements, although to a lesser extent. For patients with persistent diabetic macular edema, switching to sozinibercept plus aflibercept resulted in notable BCVA gains. The mean time to requiring retreatment was longer in treatment-naive patients than in those previously treated, indicating a durable response.
“Combination therapy with sozinibercept is going to be really important,” said Dr. Lim, who was not involved in the study, “because it attacks with a dual mechanism of action.”
Oral agents promise a potentially easier alternative for patients compared with frequent injections. CU06-1004 is a novel orally administered endothelial dysfunction blocker that has shown promise in stabilizing damaged capillaries, reducing abnormal angiogenesis, and inhibiting inflammatory activation in preclinical studies. “CU06 is very interesting to me because by preventing endothelial loss, it gets to the pathophysiology of why the blood vessels break down,” Dr. Lim said.
In a proof-of-concept phase 2a, multicenter, open-label, parallel-group trial, investigators randomly assigned 67 patients with diabetic macular edema to receive 100 mg, 200 mg, or 300 mg of CU06-1004 once daily for 12 weeks, followed by a 4-week follow-up.
Results presented by Victor Gonzalez, MD, of Valley Retina Institute in Texas, indicated that the oral agent improved BCVA, stabilized central subfield thickness, and showed positive anatomical changes in optical coherence tomography images. CU06-1004 was well tolerated, with no drug-related serious adverse events.
“The number [of patients] was very small, and we will need a much longer, larger trial to see if [CU06-1004] has benefits long term,” said Dr. Boyer, who was not involved in the study. “But I think we’re all very excited if we can find an oral agent for treating diabetic retinopathy. It would be easier for the patient to take a pill than having to come in for injections.”
The sustained-release axitinib implant, OTX-TKI, is also generating significant interest, particularly for nonproliferative diabetic retinopathy. Axitinib, a tyrosine kinase inhibitor (TKI), targets signaling pathways crucial in cellular processes, providing a novel approach to managing diseases where traditional therapies might fall short. Unlike traditional anti-VEGF treatments that focus solely on cytokine levels, TKIs block the activation of signaling pathways, preventing downstream signaling regardless of cytokine levels. This mechanism is particularly important because it effectively inhibits disease progression even if levels of VEGF are high, Dr. Lim explained.
In the phase 1 HELIOS trial, OTX-TKI was assessed in patients with nonproliferative diabetic retinopathy. This multicenter, double-masked, parallel-group clinical study included 21 patients who had not received anti-VEGF treatment, dexamethasone intravitreal implants in the previous 12 months, or intraocular steroid injections in the prior 4 months. Patients were randomly assigned to receive either OTX-TKI or sham treatment.
Results presented by Dilsher S. Dhoot, MD, of California Retina Consultants, indicated that OTX-TKI was generally well tolerated, with no serious ocular adverse events. At 48 weeks, 46.2% of eyes treated with OTX-TKI showed a 1- or 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) compared with none in the sham arm. Additionally, no eyes treated with OTX-TKI experienced a worsening on the DRSS, whereas 25% of eyes in the sham arm did. Vision-threatening complications, such as proliferative diabetic retinopathy or diabetic macular edema, developed in 37.5% of the sham group but in none of the OTX-TKI treated eyes. A single injection of OTX-TKI provided durable DRSS improvement for up to 48 weeks, with no patients in either arm requiring rescue therapy.
“This is a really exciting add-on treatment,” Dr. Lim said, who was not involved in the study. She explained that it is initially necessary to control the disease with standard treatments, because TKIs may take longer to exhibit their effects. Once the disease is stabilized, TKIs can be used alongside other therapies, potentially reducing the reliance on frequent anti-VEGF injections. “These are preliminary results, but that’s the hope going forward.”
Dr. Lim and Dr. Boyer report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
STOCKHOLM — At the American Society of Retina Specialists (ASRS) 2024 Annual Meeting, researchers discussed how insights into potential risk factors and new treatments could improve outcomes for patients with diabetic retinopathy.
Jennifer Lim, MD, an ophthalmologist and director of the Retina Service at the University of Illinois Hospital & Health Sciences System in Chicago, told this news organization that emerging approaches to treating diabetic retinopathy offer hope because they address the root causes of the disease beyond just targeting vascular endothelial growth factor (VEGF). She said innovative methods and add-on treatments could lead to more durable and effective drugs.
Exploration of risk factors and treatment options for diabetic retinopathy could lead to more effective management strategies for the condition, agreed David Boyer, MD, an ophthalmologist at Retina Vitreous Associates Medical Group in Los Angeles, speaking with this news organization.
Risk Factors for Diabetic Retinopathy
Sodium-glucose cotransporter 2 (SGLT2) inhibitors and glucagon-like peptide 1 receptor agonists (GLP-1 RAs) have gained popularity because of their benefits beyond glycemic control, including weight loss, and cardiovascular and kidney protection. However, the impact of these medications on vision-threatening retinal complications is not fully understood. “There has always been a question about whether these newer diabetes medications might exacerbate diabetic eye disease,” said Dr. Boyer.
In a retrospective observational study, researchers included adults with type 2 diabetes and moderate cardiovascular disease risk who had no history of advanced diabetic retinal complications. These patients initiated treatment with GLP-1 RA, SGLT2 inhibitors, DPP-4 inhibitors, or sulfonylureas. The study used inverse probability of treatment weighting to mimic randomization and compared the time to the first treatment for diabetic macular edema or proliferative diabetic retinopathy across the treatment groups.
Results, presented by Andrew J. Barkmeier, MD, an associate professor of ophthalmology at the Mayo Clinic, showed that among 371,698 patients, those who initiated therapy with SGLT2 inhibitors had a lower risk of requiring treatment for sight-threatening retinopathy compared with those using other medication classes. GLP-1 RA did not increase retinopathy risk relative to dipeptidyl peptidase 4 inhibitors and sulfonylurea medications.
“[This study] told us that we do have to keep an eye on patients’ retinopathy when they start on these new inhibitors. But the progression is minimal and, overall, I think most people today favor keeping blood sugar levels as good as possible,” said Dr. Boyer, who was not involved in the study.
Another factor that might increase diabetic retinopathy progression is obstructive sleep apnea. This underdiagnosed condition is linked to several health issues, including dementia, stroke, and myocardial infarctions. Although not easily treated, obstructive sleep apnea is manageable, Dr. Boyer explained.
Researchers utilized the TriNetX electronic health records research network to identify patients with nonproliferative diabetic retinopathy, both with and without obstructive sleep apnea.
The results, presented by Ehsan Rahimy, MD, a retinal specialist at Palo Alto Medical Foundation and a professor at Stanford University, showed that patients with obstructive sleep apnea had a significantly higher risk of progressing to proliferative diabetic retinopathy and developing new-onset diabetic macular edema. These patients were more likely to require ocular interventions, such as intravitreal injections and laser photocoagulation. They also had greater risks for stroke, myocardial infarction, and death compared with those who did not have obstructive sleep apnea.
“It was good to bring this to everybody’s attention,” said Dr. Boyer, who was not involved in the study. “It’s an easy question to ask someone if they snore.”
New Treatments on the Horizon
In another presentation, Nathan C. Steinle, MD, of California Retina Consultants, presented a study that assessed the durability of response to sozinibercept in patients with retinal vascular diseases. This novel therapeutic agent is designed to inhibit VEGF-C and VEGF-D in conditions where VEGF-A suppression alone is insufficient.
Sozinibercept was combined with standard anti–VEGF-A therapies such as ranibizumab or aflibercept. It involved a prospective, post hoc analysis of two phase 1b, open-label, dose-escalation studies, including 40 patients with neovascular age-related macular degeneration (nAMD; 31 patients) or diabetic macular edema (nine patients). These patients, either treatment-naive or previously treated, received three intravitreal injections of ranibizumab or aflibercept in combination with sozinibercept at various doses.
Results indicated that sozinibercept combination therapy was well tolerated, with no dose-limiting toxicities. In treatment-naive nAMD patients, the mean best-corrected visual acuity (BCVA) improved significantly from baseline at months 3 and 6. Previously treated nAMD patients also showed BCVA improvements, although to a lesser extent. For patients with persistent diabetic macular edema, switching to sozinibercept plus aflibercept resulted in notable BCVA gains. The mean time to requiring retreatment was longer in treatment-naive patients than in those previously treated, indicating a durable response.
“Combination therapy with sozinibercept is going to be really important,” said Dr. Lim, who was not involved in the study, “because it attacks with a dual mechanism of action.”
Oral agents promise a potentially easier alternative for patients compared with frequent injections. CU06-1004 is a novel orally administered endothelial dysfunction blocker that has shown promise in stabilizing damaged capillaries, reducing abnormal angiogenesis, and inhibiting inflammatory activation in preclinical studies. “CU06 is very interesting to me because by preventing endothelial loss, it gets to the pathophysiology of why the blood vessels break down,” Dr. Lim said.
In a proof-of-concept phase 2a, multicenter, open-label, parallel-group trial, investigators randomly assigned 67 patients with diabetic macular edema to receive 100 mg, 200 mg, or 300 mg of CU06-1004 once daily for 12 weeks, followed by a 4-week follow-up.
Results presented by Victor Gonzalez, MD, of Valley Retina Institute in Texas, indicated that the oral agent improved BCVA, stabilized central subfield thickness, and showed positive anatomical changes in optical coherence tomography images. CU06-1004 was well tolerated, with no drug-related serious adverse events.
“The number [of patients] was very small, and we will need a much longer, larger trial to see if [CU06-1004] has benefits long term,” said Dr. Boyer, who was not involved in the study. “But I think we’re all very excited if we can find an oral agent for treating diabetic retinopathy. It would be easier for the patient to take a pill than having to come in for injections.”
The sustained-release axitinib implant, OTX-TKI, is also generating significant interest, particularly for nonproliferative diabetic retinopathy. Axitinib, a tyrosine kinase inhibitor (TKI), targets signaling pathways crucial in cellular processes, providing a novel approach to managing diseases where traditional therapies might fall short. Unlike traditional anti-VEGF treatments that focus solely on cytokine levels, TKIs block the activation of signaling pathways, preventing downstream signaling regardless of cytokine levels. This mechanism is particularly important because it effectively inhibits disease progression even if levels of VEGF are high, Dr. Lim explained.
In the phase 1 HELIOS trial, OTX-TKI was assessed in patients with nonproliferative diabetic retinopathy. This multicenter, double-masked, parallel-group clinical study included 21 patients who had not received anti-VEGF treatment, dexamethasone intravitreal implants in the previous 12 months, or intraocular steroid injections in the prior 4 months. Patients were randomly assigned to receive either OTX-TKI or sham treatment.
Results presented by Dilsher S. Dhoot, MD, of California Retina Consultants, indicated that OTX-TKI was generally well tolerated, with no serious ocular adverse events. At 48 weeks, 46.2% of eyes treated with OTX-TKI showed a 1- or 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) compared with none in the sham arm. Additionally, no eyes treated with OTX-TKI experienced a worsening on the DRSS, whereas 25% of eyes in the sham arm did. Vision-threatening complications, such as proliferative diabetic retinopathy or diabetic macular edema, developed in 37.5% of the sham group but in none of the OTX-TKI treated eyes. A single injection of OTX-TKI provided durable DRSS improvement for up to 48 weeks, with no patients in either arm requiring rescue therapy.
“This is a really exciting add-on treatment,” Dr. Lim said, who was not involved in the study. She explained that it is initially necessary to control the disease with standard treatments, because TKIs may take longer to exhibit their effects. Once the disease is stabilized, TKIs can be used alongside other therapies, potentially reducing the reliance on frequent anti-VEGF injections. “These are preliminary results, but that’s the hope going forward.”
Dr. Lim and Dr. Boyer report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
FROM ASRS 2024