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SB-525 gene therapy looks viable for hemophilia A
Interim data suggest SB-525, an investigational gene therapy, may be safe and effective for patients with severe hemophilia A.
In the phase 1/2 Alta trial, SB-525 produced dose-dependent increases in factor VIII activity, with two of eight patients achieving normal factor VIII levels.
In addition, SB-525 was considered well tolerated. One patient did experience treatment-related serious adverse events – hypotension and fever – but these resolved within 24 hours.
Sangamo Therapeutics and Pfizer recently released these data in a press release and conference call.
The data include eight patients with severe hemophilia A who received SB-525 at 9e11 vg/kg, 2e12 vg/kg, 1e13 vg/kg, and 3e13 vg/kg.
Patient 1 (9e11 vg/kg), Patient 2 (9e11 vg/kg), and Patient 3 (2e12 vg/kg) did not experience clinically relevant increases in factor VIII levels and still require prophylactic recombinant factor VIII therapy.
Patient 4 (2e12 vg/kg) and Patient 5 (1e13 vg/kg) discontinued factor VIII therapy but have experienced spontaneous bleeds. Patient 4 had three bleeds in 48 weeks of follow-up, and Patient 5 had two bleeds in 40 weeks of follow-up.
Patient 6 (1e13 vg/kg), Patient 7 (3e13 vg/kg), and Patient 8 (3e13 vg/kg) have stopped factor VIII therapy and remain free of spontaneous bleeds at 28 weeks, 12 weeks, and 6 weeks of follow-up, respectively.
Patients 7 and 8 have achieved normal factor VIII levels. At 6 weeks, their factor VIII levels reached 140% and 94% of normal, respectively, according to a one-stage clotting assay, and 93% and 65%, respectively, according to a chromogenic assay.
“It appears the patients achieve their peak factor level at week 5 to 7 and maintain that level,” Edward Conner, MD, chief medical officer of Sangamo, said during the conference call.
The adverse events observed in this trial include grade 1 tachycardia (n = 1), grade 1 fatigue (n = 1), grade 1 alanine aminotransferase increase (n = 3), grade 1 myalgia (n = 1), grade 2 pyrexia (n = 2), and grade 3 hypotension (n = 1).
“These interim results indicate that SB-525 has the potential to comprise a well-tolerated, reliable, and predictable treatment, features that we believe will be a hallmark of the future gene therapy treatment of hemophilia A,” Dr. Conner said during the call.
Sangamo and Pfizer are enrolling additional patients in this trial, with the goal of expanding the 3e13 vg/kg cohort by up to five patients. The companies plan to present longer-term follow-up data at an upcoming scientific meeting.
The Alta trial is sponsored by Sangamo Therapeutics in collaboration with Pfizer.
Interim data suggest SB-525, an investigational gene therapy, may be safe and effective for patients with severe hemophilia A.
In the phase 1/2 Alta trial, SB-525 produced dose-dependent increases in factor VIII activity, with two of eight patients achieving normal factor VIII levels.
In addition, SB-525 was considered well tolerated. One patient did experience treatment-related serious adverse events – hypotension and fever – but these resolved within 24 hours.
Sangamo Therapeutics and Pfizer recently released these data in a press release and conference call.
The data include eight patients with severe hemophilia A who received SB-525 at 9e11 vg/kg, 2e12 vg/kg, 1e13 vg/kg, and 3e13 vg/kg.
Patient 1 (9e11 vg/kg), Patient 2 (9e11 vg/kg), and Patient 3 (2e12 vg/kg) did not experience clinically relevant increases in factor VIII levels and still require prophylactic recombinant factor VIII therapy.
Patient 4 (2e12 vg/kg) and Patient 5 (1e13 vg/kg) discontinued factor VIII therapy but have experienced spontaneous bleeds. Patient 4 had three bleeds in 48 weeks of follow-up, and Patient 5 had two bleeds in 40 weeks of follow-up.
Patient 6 (1e13 vg/kg), Patient 7 (3e13 vg/kg), and Patient 8 (3e13 vg/kg) have stopped factor VIII therapy and remain free of spontaneous bleeds at 28 weeks, 12 weeks, and 6 weeks of follow-up, respectively.
Patients 7 and 8 have achieved normal factor VIII levels. At 6 weeks, their factor VIII levels reached 140% and 94% of normal, respectively, according to a one-stage clotting assay, and 93% and 65%, respectively, according to a chromogenic assay.
“It appears the patients achieve their peak factor level at week 5 to 7 and maintain that level,” Edward Conner, MD, chief medical officer of Sangamo, said during the conference call.
The adverse events observed in this trial include grade 1 tachycardia (n = 1), grade 1 fatigue (n = 1), grade 1 alanine aminotransferase increase (n = 3), grade 1 myalgia (n = 1), grade 2 pyrexia (n = 2), and grade 3 hypotension (n = 1).
“These interim results indicate that SB-525 has the potential to comprise a well-tolerated, reliable, and predictable treatment, features that we believe will be a hallmark of the future gene therapy treatment of hemophilia A,” Dr. Conner said during the call.
Sangamo and Pfizer are enrolling additional patients in this trial, with the goal of expanding the 3e13 vg/kg cohort by up to five patients. The companies plan to present longer-term follow-up data at an upcoming scientific meeting.
The Alta trial is sponsored by Sangamo Therapeutics in collaboration with Pfizer.
Interim data suggest SB-525, an investigational gene therapy, may be safe and effective for patients with severe hemophilia A.
In the phase 1/2 Alta trial, SB-525 produced dose-dependent increases in factor VIII activity, with two of eight patients achieving normal factor VIII levels.
In addition, SB-525 was considered well tolerated. One patient did experience treatment-related serious adverse events – hypotension and fever – but these resolved within 24 hours.
Sangamo Therapeutics and Pfizer recently released these data in a press release and conference call.
The data include eight patients with severe hemophilia A who received SB-525 at 9e11 vg/kg, 2e12 vg/kg, 1e13 vg/kg, and 3e13 vg/kg.
Patient 1 (9e11 vg/kg), Patient 2 (9e11 vg/kg), and Patient 3 (2e12 vg/kg) did not experience clinically relevant increases in factor VIII levels and still require prophylactic recombinant factor VIII therapy.
Patient 4 (2e12 vg/kg) and Patient 5 (1e13 vg/kg) discontinued factor VIII therapy but have experienced spontaneous bleeds. Patient 4 had three bleeds in 48 weeks of follow-up, and Patient 5 had two bleeds in 40 weeks of follow-up.
Patient 6 (1e13 vg/kg), Patient 7 (3e13 vg/kg), and Patient 8 (3e13 vg/kg) have stopped factor VIII therapy and remain free of spontaneous bleeds at 28 weeks, 12 weeks, and 6 weeks of follow-up, respectively.
Patients 7 and 8 have achieved normal factor VIII levels. At 6 weeks, their factor VIII levels reached 140% and 94% of normal, respectively, according to a one-stage clotting assay, and 93% and 65%, respectively, according to a chromogenic assay.
“It appears the patients achieve their peak factor level at week 5 to 7 and maintain that level,” Edward Conner, MD, chief medical officer of Sangamo, said during the conference call.
The adverse events observed in this trial include grade 1 tachycardia (n = 1), grade 1 fatigue (n = 1), grade 1 alanine aminotransferase increase (n = 3), grade 1 myalgia (n = 1), grade 2 pyrexia (n = 2), and grade 3 hypotension (n = 1).
“These interim results indicate that SB-525 has the potential to comprise a well-tolerated, reliable, and predictable treatment, features that we believe will be a hallmark of the future gene therapy treatment of hemophilia A,” Dr. Conner said during the call.
Sangamo and Pfizer are enrolling additional patients in this trial, with the goal of expanding the 3e13 vg/kg cohort by up to five patients. The companies plan to present longer-term follow-up data at an upcoming scientific meeting.
The Alta trial is sponsored by Sangamo Therapeutics in collaboration with Pfizer.
My patient will die
“You are the best doctor I ever had.” These were the words of my patient at our final session. I kissed Rosa on the cheek and embraced her as she left my office. And I said, “It is important to show the love.”
“This may be the last time I come to your office,” she said. When Rosa came through the door at the beginning of the session, I was taken aback. She had lost weight and was using a walker; her face was drawn and sallow. I knew she had a recent diagnosis of liver cancer and had been hospitalized. She told me: “I have 3 months to live.” The cancer was inoperable.
She sat in a chair close to me, and we reminisced about 20 years as doctor and patient. She also talked about the stents in her liver; when they blocked, the pain resulted in a revisit to the emergency department. She had help from home health aides for several hours a day. Rosa’s sister arrived from Puerto Rico to be here “for as long as it takes.”
When she started therapy, Rosa was a single mother who lived in the projects with her adolescent son, Wesley. Her husband had died of AIDS. Unemployed and depressed, she told me that an uncle had sexually abused her when she was a child. Over the years, she looked to me for support: When her son, Wesley, got shot in the leg on a basketball court; when Wesley married a woman who shunned her; when her nephew who stayed with her got arrested for selling drugs and she lost her apartment as a result. Rosa remained in New York, displaced and struggling to find a reasonable home. After Wesley married, he moved with his family to rural Pennsylvania.
Whenever Rosa called to set up a therapy session, we talked about her problems. I prescribed medication for her, and I directed her to proper medical care. Often, I encouraged her to improve her diet, lose weight, and exercise – but to no avail. Her health deteriorated. She had cardiac surgery, heart failure, diabetes, hypertension, and chronic obesity. All these illnesses became her concern. She attended clinics at the hospital.
Now she told me that she would miss her son and would not see her two young grandchildren grow up. Rosa took Wesley to a funeral home to select a coffin and a headstone. It was tough for both of them, but she wanted to spare Wesley the trouble of doing it alone. She reflected, “It was like hitting a concrete wall” when she discovered her terminal diagnosis.
Rosa is facing pain, saying goodbye, and death. During her meeting, her ordeal made me cry, but I tried to contain it. I have been her doctor for so long, not a member of her family, not a friend. Yet I love her.
“Just to be is a blessing. Just to live is holy.”
– Abraham Joshua Heschel
Dr. Cohen is in private practice and is a clinical assistant professor of psychiatry at Weill Cornell Medical Center of New York-Presbyterian Hospital, and psychiatric consultant at the Hospital for Special Surgery, also in New York. She made changes to the patient’s story to protect confidentiality.
“You are the best doctor I ever had.” These were the words of my patient at our final session. I kissed Rosa on the cheek and embraced her as she left my office. And I said, “It is important to show the love.”
“This may be the last time I come to your office,” she said. When Rosa came through the door at the beginning of the session, I was taken aback. She had lost weight and was using a walker; her face was drawn and sallow. I knew she had a recent diagnosis of liver cancer and had been hospitalized. She told me: “I have 3 months to live.” The cancer was inoperable.
She sat in a chair close to me, and we reminisced about 20 years as doctor and patient. She also talked about the stents in her liver; when they blocked, the pain resulted in a revisit to the emergency department. She had help from home health aides for several hours a day. Rosa’s sister arrived from Puerto Rico to be here “for as long as it takes.”
When she started therapy, Rosa was a single mother who lived in the projects with her adolescent son, Wesley. Her husband had died of AIDS. Unemployed and depressed, she told me that an uncle had sexually abused her when she was a child. Over the years, she looked to me for support: When her son, Wesley, got shot in the leg on a basketball court; when Wesley married a woman who shunned her; when her nephew who stayed with her got arrested for selling drugs and she lost her apartment as a result. Rosa remained in New York, displaced and struggling to find a reasonable home. After Wesley married, he moved with his family to rural Pennsylvania.
Whenever Rosa called to set up a therapy session, we talked about her problems. I prescribed medication for her, and I directed her to proper medical care. Often, I encouraged her to improve her diet, lose weight, and exercise – but to no avail. Her health deteriorated. She had cardiac surgery, heart failure, diabetes, hypertension, and chronic obesity. All these illnesses became her concern. She attended clinics at the hospital.
Now she told me that she would miss her son and would not see her two young grandchildren grow up. Rosa took Wesley to a funeral home to select a coffin and a headstone. It was tough for both of them, but she wanted to spare Wesley the trouble of doing it alone. She reflected, “It was like hitting a concrete wall” when she discovered her terminal diagnosis.
Rosa is facing pain, saying goodbye, and death. During her meeting, her ordeal made me cry, but I tried to contain it. I have been her doctor for so long, not a member of her family, not a friend. Yet I love her.
“Just to be is a blessing. Just to live is holy.”
– Abraham Joshua Heschel
Dr. Cohen is in private practice and is a clinical assistant professor of psychiatry at Weill Cornell Medical Center of New York-Presbyterian Hospital, and psychiatric consultant at the Hospital for Special Surgery, also in New York. She made changes to the patient’s story to protect confidentiality.
“You are the best doctor I ever had.” These were the words of my patient at our final session. I kissed Rosa on the cheek and embraced her as she left my office. And I said, “It is important to show the love.”
“This may be the last time I come to your office,” she said. When Rosa came through the door at the beginning of the session, I was taken aback. She had lost weight and was using a walker; her face was drawn and sallow. I knew she had a recent diagnosis of liver cancer and had been hospitalized. She told me: “I have 3 months to live.” The cancer was inoperable.
She sat in a chair close to me, and we reminisced about 20 years as doctor and patient. She also talked about the stents in her liver; when they blocked, the pain resulted in a revisit to the emergency department. She had help from home health aides for several hours a day. Rosa’s sister arrived from Puerto Rico to be here “for as long as it takes.”
When she started therapy, Rosa was a single mother who lived in the projects with her adolescent son, Wesley. Her husband had died of AIDS. Unemployed and depressed, she told me that an uncle had sexually abused her when she was a child. Over the years, she looked to me for support: When her son, Wesley, got shot in the leg on a basketball court; when Wesley married a woman who shunned her; when her nephew who stayed with her got arrested for selling drugs and she lost her apartment as a result. Rosa remained in New York, displaced and struggling to find a reasonable home. After Wesley married, he moved with his family to rural Pennsylvania.
Whenever Rosa called to set up a therapy session, we talked about her problems. I prescribed medication for her, and I directed her to proper medical care. Often, I encouraged her to improve her diet, lose weight, and exercise – but to no avail. Her health deteriorated. She had cardiac surgery, heart failure, diabetes, hypertension, and chronic obesity. All these illnesses became her concern. She attended clinics at the hospital.
Now she told me that she would miss her son and would not see her two young grandchildren grow up. Rosa took Wesley to a funeral home to select a coffin and a headstone. It was tough for both of them, but she wanted to spare Wesley the trouble of doing it alone. She reflected, “It was like hitting a concrete wall” when she discovered her terminal diagnosis.
Rosa is facing pain, saying goodbye, and death. During her meeting, her ordeal made me cry, but I tried to contain it. I have been her doctor for so long, not a member of her family, not a friend. Yet I love her.
“Just to be is a blessing. Just to live is holy.”
– Abraham Joshua Heschel
Dr. Cohen is in private practice and is a clinical assistant professor of psychiatry at Weill Cornell Medical Center of New York-Presbyterian Hospital, and psychiatric consultant at the Hospital for Special Surgery, also in New York. She made changes to the patient’s story to protect confidentiality.
In search of high-value care
Six steps that can help your team
U.S. spending on health care is growing rapidly and expected to reach 19.7% of gross domestic product by 2026.1 In response, the Centers for Medicare and Medicaid Services and national organizations such as the American Board of Internal Medicine (ABIM) and the American College of Physicians (ACP) have launched initiatives to ensure that the value being delivered to patients is on par with the escalating cost of care.
Over the past 10 years, I have led and advised hundreds of small- and large-scale projects that focused on improving patient care quality and cost. Below, I share what I, along with other leaders in high-value care, have observed that it takes to implement successful and lasting improvements – for the benefit of patients and hospitals.
A brief history of high-value care
When compared to other wealthy countries, the United States spends disproportionately more money on health care. In 2016, U.S. health care spending was $3.3 trillion1, or $10,348 per person.2 Hospital care alone was responsible for a third of health spending and amounted to $1.1 trillion in 20161. By 2026, national health spending is projected to reach $5.7 trillion1.
In response to escalating health care costs, CMS and other payers have shifted toward value-based reimbursements that tie payments to health care facilities and clinicians to their performance on selected quality, cost, and efficiency measures. For example, under the CMS Merit-based Incentive Payment System (MIPS), 5% of clinicians’ revenue in 2020 is tied to their 2018 performance in four categories: Quality, Cost, Improvement Activities, and Promoting Interoperability. The percentage of revenue at risk will increase to 9% in 2022, based on 2020 performance.
Rising health care costs put a burden not just on the federal and state budgets, but on individual and family budgets as well. Out-of-pocket spending grew 3.9% in 2016 to $352.5 billion1 and is expected to increase in the future. High health care costs rightfully bring into question the value individual consumers of health care services are getting in return. If value is defined as the level of benefit achieved for a given cost, what is high-value care? The 2013 Institute of Medicine report3 defined high-value care as “the best care for the patient, with the optimal result for the circumstances, delivered at the right price.” It goes beyond a set of quality and cost measures used by payers to affect provider reimbursement and is driven by day-to-day individual providers’ decisions that affect individual patients’ outcomes and their cost of care.
High-value care has been embraced by national organizations. In 2012, the ABIM Foundation launched the Choosing Wisely initiative to support and promote conversations between clinicians and patients in choosing care that is truly necessary, supported by evidence, and free from harm. The result was an evidence-based list of recommendations from 540 specialty societies, including the Society of Hospital Medicine. The SHM – Adult Hospital Medicine list4 features the following “Five things physicians and patients should question”:
- Don’t place, or leave in place, urinary catheters for incontinence or convenience or monitoring of output for non–critically ill patients.
- Don’t prescribe medications for stress ulcer prophylaxis to medical inpatients unless at high risk for GI complications.
- Avoid transfusions of red blood cells for arbitrary hemoglobin or hematocrit thresholds and in the absence of symptoms of active coronary disease, heart failure, or stroke.
- Don’t order continuous telemetry monitoring outside of the ICU without using a protocol that governs continuation.
- Don’t perform repetitive CBC and chemistry testing in the face of clinical and lab stability.
The ACP launched a high-value care initiative that offers learning resources for clinicians and medical educators, clinical guidelines, and best practice advice. In 2012, a workgroup of internists convened by ACP developed a list of 37 clinical situations in which medical tests are commonly used but do not provide high value.5 Seven of those situations are applicable to adult hospital medicine.
High-value care today: What the experts say
More than 5 years later, what progress have hospitalists made in adopting high-value care practices? To answer this and other questions, I reached out to three national experts in high-value care in hospital medicine: Amit Pahwa, MD, assistant professor of medicine and pediatrics at Johns Hopkins University, Baltimore, and a course director of “Topics in interdisciplinary medicine: High-value health care”; Christopher Petrilli, MD, clinical assistant professor in the department of medicine at New York University Langone Health and clinical lead, Manhattan campus, value-based management; and Charlie Wray, DO, MS, assistant professor of medicine at the University of California in San Francisco and a coauthor of an article on high-value care in hospital medicine published recently in the Journal of General Internal Medicine6.
The experts agree that awareness of high-value care among practicing physicians and medical trainees has increased in the last few years. Major professional publications have highlighted the topic, including The Journal of Hospital Medicine’s “Things We Do For No Reason” series, JAMA’s “Teachable Moments,” and the American Journal of Medicine’s recurring column dedicated to high-value care practice. Leading teaching institutions have built high-value care curricula as a part of their medical student and resident training. However, widespread adoption has been slow and sometimes difficult.
The barriers to adoption of high-value practices among hospitalists are numerous and deep rooted in historical practices and culture. As Dr. Petrilli said, the “culture of overordering [diagnostic tests] is hard to break.” Hospitalists may not have well-developed relationships with patients, or time to explain why some tests or treatments are unnecessary. There is a lack of cost transparency, including the cost of the tests themselves and the downstream costs of additional tests and follow-ups. The best intended interventions fail to produce durable change unless they are seamlessly integrated into a hospitalist’s daily workflow.
Six steps to implementing a successful high-value care initiative
What can hospitalists do to improve the value of care they provide to their patients and hospital partners?
1. Identify high-value care opportunities at your hospital.
Dr. Wray pointed out that “all high-value care is local.” Start by looking at the national guidelines and talking to your senior clinical leaders and colleagues. Review your hospital data to identify opportunities and understand the root causes, including variability among providers.
If you choose to analyze and present provider-specific data, first be transparent on why you are doing that. Your goal is not to tell physicians how to practice or to score them, but instead, to promote adoption of evidence-based high-value care by identifying and discussing provider practice variations, and to generate possible solutions. Second, make sure that the data you present is credible and trustworthy by clearly outlining the data source, time frame, sample size per provider, any inclusion and exclusion criteria, attribution logic, and severity adjustment methodology. Third, expect initial pushback as transparency and change can be scary. But most doctors are inherently competitive and will want to be the best at caring for their patients.
2. Assemble the team.
Identify an executive sponsor – a senior clinical executive (for example, the chief medical officer or vice president of medical affairs) whose role is to help engage stakeholders, secure resources, and remove barriers. When assembling the rest of the team, include a representative from each major stakeholder group, but keep the team small enough to be effective. For example, if your project focuses on improving telemetry utilization, seek representation from hospitalists, cardiologists, nurses, utilization managers, and possibly IT. Look for people with the relevant knowledge and experience who are respected by their peers and can influence opinion.
3. Design a sustainable solution.
To be sustainable, a solution must be evidence based, well integrated in provider workflow, and have acceptable impact on daily workload (e.g., additional time per patient). If an estimated impact is significant, you need to discuss adding resources or negotiating trade-offs.
A great example of a sustainable solution, aimed to control overutilization of telemetry and urinary catheters, is the one implemented by Dr. Wray and his team.7 They designed an EHR-based “silent” indicator that clearly signaled an active telemetry or urinary catheter order for each patient. Clicking on the indicator directed a provider to a “manage order” screen where she could cancel the order, if necessary.
4. Engage providers.
You may design the best solution, but it will not succeed unless it is embraced by others. To engage providers, you must clearly communicate why the change is urgently needed for the benefit of their patients, hospital, or community, and appeal to their minds, hearts, and competitive nature.
For example, if you are focusing on overutilization of urinary catheters, you may share your hospital’s urinary catheter device utilization ratio (# of indwelling catheter days/# patient days) against national benchmarks, or the impact on hospital catheter–associated urinary tract infections (CAUTI) rates to appeal to the physicians’ minds. Often, data alone are not enough to move people to action. You must appeal to their hearts by sharing stories of real patients whose lives were affected by preventable CAUTI. Leverage physicians’ competitive nature by using provider-specific data to compare against their peers to spark a discussion.
5. Evaluate impact.
Even before you implement a solution, select metrics to measure impact and set SMART (specific, measurable, achievable, relevant, and time-bound) goals. As your implementation moves forward, do not let up or give up – continue to evaluate impact, remove barriers, refine your solution to get back on track if needed, and constantly communicate to share ongoing project results and lessons learned.
6. Sustain improvements.
Sustainable improvements require well-designed solutions integrated into provider workflow, but that is just the first step. Once you demonstrate the impact, consider including the metric (e.g., telemetry or urinary catheter utilization) in your team and/or individual provider performance dashboard, regularly reviewing and discussing performance during your team meetings to maintain engagement, and if needed, making improvements to get back on track.
Successful adoption of high-value care practices requires a disciplined approach to design and implement solutions that are patient-centric, evidence-based, data-driven and integrated in provider workflow.
Dr. Farah is a hospitalist, Physician Advisor, and Lean Six Sigma Black Belt. She is a performance improvement consultant based in Corvallis, Ore., and a member of The Hospitalist’s editorial advisory board.
References
1. From the Centers for Medicare & Medicaid Services: National Health Expenditure Projections 2018-2027.
2. Peterson-Kaiser Health System Tracker: How does health spending in the U.S. compare to other countries?
3. Creating a new culture of care, in “Best care at lower cost: The path to continuously learning health care in America.” (Washington: National Academies Press, 2013, pp. 255-80).
4. Choosing Wisely: SHM – Adult Hospital Medicine; Five things physicians and patients should question.
5. Qaseem A et al. Appropriate use of screening and diagnostic tests to foster high-value, cost-conscious care. Ann Intern Med. 2012 Jan 17;156(2):147-9.
6. Cho HJ et al. Right care in hospital medicine: Co-creation of ten opportunities in overuse and underuse for improving value in hospital medicine. J Gen Intern Med. 2018 Jun;33(6):804-6.
7. Wray CM et al. Improving value by reducing unnecessary telemetry and urinary catheter utilization in hospitalized patients. Am J Med. 2017 Sep;130(9):1037-41.
Six steps that can help your team
Six steps that can help your team
U.S. spending on health care is growing rapidly and expected to reach 19.7% of gross domestic product by 2026.1 In response, the Centers for Medicare and Medicaid Services and national organizations such as the American Board of Internal Medicine (ABIM) and the American College of Physicians (ACP) have launched initiatives to ensure that the value being delivered to patients is on par with the escalating cost of care.
Over the past 10 years, I have led and advised hundreds of small- and large-scale projects that focused on improving patient care quality and cost. Below, I share what I, along with other leaders in high-value care, have observed that it takes to implement successful and lasting improvements – for the benefit of patients and hospitals.
A brief history of high-value care
When compared to other wealthy countries, the United States spends disproportionately more money on health care. In 2016, U.S. health care spending was $3.3 trillion1, or $10,348 per person.2 Hospital care alone was responsible for a third of health spending and amounted to $1.1 trillion in 20161. By 2026, national health spending is projected to reach $5.7 trillion1.
In response to escalating health care costs, CMS and other payers have shifted toward value-based reimbursements that tie payments to health care facilities and clinicians to their performance on selected quality, cost, and efficiency measures. For example, under the CMS Merit-based Incentive Payment System (MIPS), 5% of clinicians’ revenue in 2020 is tied to their 2018 performance in four categories: Quality, Cost, Improvement Activities, and Promoting Interoperability. The percentage of revenue at risk will increase to 9% in 2022, based on 2020 performance.
Rising health care costs put a burden not just on the federal and state budgets, but on individual and family budgets as well. Out-of-pocket spending grew 3.9% in 2016 to $352.5 billion1 and is expected to increase in the future. High health care costs rightfully bring into question the value individual consumers of health care services are getting in return. If value is defined as the level of benefit achieved for a given cost, what is high-value care? The 2013 Institute of Medicine report3 defined high-value care as “the best care for the patient, with the optimal result for the circumstances, delivered at the right price.” It goes beyond a set of quality and cost measures used by payers to affect provider reimbursement and is driven by day-to-day individual providers’ decisions that affect individual patients’ outcomes and their cost of care.
High-value care has been embraced by national organizations. In 2012, the ABIM Foundation launched the Choosing Wisely initiative to support and promote conversations between clinicians and patients in choosing care that is truly necessary, supported by evidence, and free from harm. The result was an evidence-based list of recommendations from 540 specialty societies, including the Society of Hospital Medicine. The SHM – Adult Hospital Medicine list4 features the following “Five things physicians and patients should question”:
- Don’t place, or leave in place, urinary catheters for incontinence or convenience or monitoring of output for non–critically ill patients.
- Don’t prescribe medications for stress ulcer prophylaxis to medical inpatients unless at high risk for GI complications.
- Avoid transfusions of red blood cells for arbitrary hemoglobin or hematocrit thresholds and in the absence of symptoms of active coronary disease, heart failure, or stroke.
- Don’t order continuous telemetry monitoring outside of the ICU without using a protocol that governs continuation.
- Don’t perform repetitive CBC and chemistry testing in the face of clinical and lab stability.
The ACP launched a high-value care initiative that offers learning resources for clinicians and medical educators, clinical guidelines, and best practice advice. In 2012, a workgroup of internists convened by ACP developed a list of 37 clinical situations in which medical tests are commonly used but do not provide high value.5 Seven of those situations are applicable to adult hospital medicine.
High-value care today: What the experts say
More than 5 years later, what progress have hospitalists made in adopting high-value care practices? To answer this and other questions, I reached out to three national experts in high-value care in hospital medicine: Amit Pahwa, MD, assistant professor of medicine and pediatrics at Johns Hopkins University, Baltimore, and a course director of “Topics in interdisciplinary medicine: High-value health care”; Christopher Petrilli, MD, clinical assistant professor in the department of medicine at New York University Langone Health and clinical lead, Manhattan campus, value-based management; and Charlie Wray, DO, MS, assistant professor of medicine at the University of California in San Francisco and a coauthor of an article on high-value care in hospital medicine published recently in the Journal of General Internal Medicine6.
The experts agree that awareness of high-value care among practicing physicians and medical trainees has increased in the last few years. Major professional publications have highlighted the topic, including The Journal of Hospital Medicine’s “Things We Do For No Reason” series, JAMA’s “Teachable Moments,” and the American Journal of Medicine’s recurring column dedicated to high-value care practice. Leading teaching institutions have built high-value care curricula as a part of their medical student and resident training. However, widespread adoption has been slow and sometimes difficult.
The barriers to adoption of high-value practices among hospitalists are numerous and deep rooted in historical practices and culture. As Dr. Petrilli said, the “culture of overordering [diagnostic tests] is hard to break.” Hospitalists may not have well-developed relationships with patients, or time to explain why some tests or treatments are unnecessary. There is a lack of cost transparency, including the cost of the tests themselves and the downstream costs of additional tests and follow-ups. The best intended interventions fail to produce durable change unless they are seamlessly integrated into a hospitalist’s daily workflow.
Six steps to implementing a successful high-value care initiative
What can hospitalists do to improve the value of care they provide to their patients and hospital partners?
1. Identify high-value care opportunities at your hospital.
Dr. Wray pointed out that “all high-value care is local.” Start by looking at the national guidelines and talking to your senior clinical leaders and colleagues. Review your hospital data to identify opportunities and understand the root causes, including variability among providers.
If you choose to analyze and present provider-specific data, first be transparent on why you are doing that. Your goal is not to tell physicians how to practice or to score them, but instead, to promote adoption of evidence-based high-value care by identifying and discussing provider practice variations, and to generate possible solutions. Second, make sure that the data you present is credible and trustworthy by clearly outlining the data source, time frame, sample size per provider, any inclusion and exclusion criteria, attribution logic, and severity adjustment methodology. Third, expect initial pushback as transparency and change can be scary. But most doctors are inherently competitive and will want to be the best at caring for their patients.
2. Assemble the team.
Identify an executive sponsor – a senior clinical executive (for example, the chief medical officer or vice president of medical affairs) whose role is to help engage stakeholders, secure resources, and remove barriers. When assembling the rest of the team, include a representative from each major stakeholder group, but keep the team small enough to be effective. For example, if your project focuses on improving telemetry utilization, seek representation from hospitalists, cardiologists, nurses, utilization managers, and possibly IT. Look for people with the relevant knowledge and experience who are respected by their peers and can influence opinion.
3. Design a sustainable solution.
To be sustainable, a solution must be evidence based, well integrated in provider workflow, and have acceptable impact on daily workload (e.g., additional time per patient). If an estimated impact is significant, you need to discuss adding resources or negotiating trade-offs.
A great example of a sustainable solution, aimed to control overutilization of telemetry and urinary catheters, is the one implemented by Dr. Wray and his team.7 They designed an EHR-based “silent” indicator that clearly signaled an active telemetry or urinary catheter order for each patient. Clicking on the indicator directed a provider to a “manage order” screen where she could cancel the order, if necessary.
4. Engage providers.
You may design the best solution, but it will not succeed unless it is embraced by others. To engage providers, you must clearly communicate why the change is urgently needed for the benefit of their patients, hospital, or community, and appeal to their minds, hearts, and competitive nature.
For example, if you are focusing on overutilization of urinary catheters, you may share your hospital’s urinary catheter device utilization ratio (# of indwelling catheter days/# patient days) against national benchmarks, or the impact on hospital catheter–associated urinary tract infections (CAUTI) rates to appeal to the physicians’ minds. Often, data alone are not enough to move people to action. You must appeal to their hearts by sharing stories of real patients whose lives were affected by preventable CAUTI. Leverage physicians’ competitive nature by using provider-specific data to compare against their peers to spark a discussion.
5. Evaluate impact.
Even before you implement a solution, select metrics to measure impact and set SMART (specific, measurable, achievable, relevant, and time-bound) goals. As your implementation moves forward, do not let up or give up – continue to evaluate impact, remove barriers, refine your solution to get back on track if needed, and constantly communicate to share ongoing project results and lessons learned.
6. Sustain improvements.
Sustainable improvements require well-designed solutions integrated into provider workflow, but that is just the first step. Once you demonstrate the impact, consider including the metric (e.g., telemetry or urinary catheter utilization) in your team and/or individual provider performance dashboard, regularly reviewing and discussing performance during your team meetings to maintain engagement, and if needed, making improvements to get back on track.
Successful adoption of high-value care practices requires a disciplined approach to design and implement solutions that are patient-centric, evidence-based, data-driven and integrated in provider workflow.
Dr. Farah is a hospitalist, Physician Advisor, and Lean Six Sigma Black Belt. She is a performance improvement consultant based in Corvallis, Ore., and a member of The Hospitalist’s editorial advisory board.
References
1. From the Centers for Medicare & Medicaid Services: National Health Expenditure Projections 2018-2027.
2. Peterson-Kaiser Health System Tracker: How does health spending in the U.S. compare to other countries?
3. Creating a new culture of care, in “Best care at lower cost: The path to continuously learning health care in America.” (Washington: National Academies Press, 2013, pp. 255-80).
4. Choosing Wisely: SHM – Adult Hospital Medicine; Five things physicians and patients should question.
5. Qaseem A et al. Appropriate use of screening and diagnostic tests to foster high-value, cost-conscious care. Ann Intern Med. 2012 Jan 17;156(2):147-9.
6. Cho HJ et al. Right care in hospital medicine: Co-creation of ten opportunities in overuse and underuse for improving value in hospital medicine. J Gen Intern Med. 2018 Jun;33(6):804-6.
7. Wray CM et al. Improving value by reducing unnecessary telemetry and urinary catheter utilization in hospitalized patients. Am J Med. 2017 Sep;130(9):1037-41.
U.S. spending on health care is growing rapidly and expected to reach 19.7% of gross domestic product by 2026.1 In response, the Centers for Medicare and Medicaid Services and national organizations such as the American Board of Internal Medicine (ABIM) and the American College of Physicians (ACP) have launched initiatives to ensure that the value being delivered to patients is on par with the escalating cost of care.
Over the past 10 years, I have led and advised hundreds of small- and large-scale projects that focused on improving patient care quality and cost. Below, I share what I, along with other leaders in high-value care, have observed that it takes to implement successful and lasting improvements – for the benefit of patients and hospitals.
A brief history of high-value care
When compared to other wealthy countries, the United States spends disproportionately more money on health care. In 2016, U.S. health care spending was $3.3 trillion1, or $10,348 per person.2 Hospital care alone was responsible for a third of health spending and amounted to $1.1 trillion in 20161. By 2026, national health spending is projected to reach $5.7 trillion1.
In response to escalating health care costs, CMS and other payers have shifted toward value-based reimbursements that tie payments to health care facilities and clinicians to their performance on selected quality, cost, and efficiency measures. For example, under the CMS Merit-based Incentive Payment System (MIPS), 5% of clinicians’ revenue in 2020 is tied to their 2018 performance in four categories: Quality, Cost, Improvement Activities, and Promoting Interoperability. The percentage of revenue at risk will increase to 9% in 2022, based on 2020 performance.
Rising health care costs put a burden not just on the federal and state budgets, but on individual and family budgets as well. Out-of-pocket spending grew 3.9% in 2016 to $352.5 billion1 and is expected to increase in the future. High health care costs rightfully bring into question the value individual consumers of health care services are getting in return. If value is defined as the level of benefit achieved for a given cost, what is high-value care? The 2013 Institute of Medicine report3 defined high-value care as “the best care for the patient, with the optimal result for the circumstances, delivered at the right price.” It goes beyond a set of quality and cost measures used by payers to affect provider reimbursement and is driven by day-to-day individual providers’ decisions that affect individual patients’ outcomes and their cost of care.
High-value care has been embraced by national organizations. In 2012, the ABIM Foundation launched the Choosing Wisely initiative to support and promote conversations between clinicians and patients in choosing care that is truly necessary, supported by evidence, and free from harm. The result was an evidence-based list of recommendations from 540 specialty societies, including the Society of Hospital Medicine. The SHM – Adult Hospital Medicine list4 features the following “Five things physicians and patients should question”:
- Don’t place, or leave in place, urinary catheters for incontinence or convenience or monitoring of output for non–critically ill patients.
- Don’t prescribe medications for stress ulcer prophylaxis to medical inpatients unless at high risk for GI complications.
- Avoid transfusions of red blood cells for arbitrary hemoglobin or hematocrit thresholds and in the absence of symptoms of active coronary disease, heart failure, or stroke.
- Don’t order continuous telemetry monitoring outside of the ICU without using a protocol that governs continuation.
- Don’t perform repetitive CBC and chemistry testing in the face of clinical and lab stability.
The ACP launched a high-value care initiative that offers learning resources for clinicians and medical educators, clinical guidelines, and best practice advice. In 2012, a workgroup of internists convened by ACP developed a list of 37 clinical situations in which medical tests are commonly used but do not provide high value.5 Seven of those situations are applicable to adult hospital medicine.
High-value care today: What the experts say
More than 5 years later, what progress have hospitalists made in adopting high-value care practices? To answer this and other questions, I reached out to three national experts in high-value care in hospital medicine: Amit Pahwa, MD, assistant professor of medicine and pediatrics at Johns Hopkins University, Baltimore, and a course director of “Topics in interdisciplinary medicine: High-value health care”; Christopher Petrilli, MD, clinical assistant professor in the department of medicine at New York University Langone Health and clinical lead, Manhattan campus, value-based management; and Charlie Wray, DO, MS, assistant professor of medicine at the University of California in San Francisco and a coauthor of an article on high-value care in hospital medicine published recently in the Journal of General Internal Medicine6.
The experts agree that awareness of high-value care among practicing physicians and medical trainees has increased in the last few years. Major professional publications have highlighted the topic, including The Journal of Hospital Medicine’s “Things We Do For No Reason” series, JAMA’s “Teachable Moments,” and the American Journal of Medicine’s recurring column dedicated to high-value care practice. Leading teaching institutions have built high-value care curricula as a part of their medical student and resident training. However, widespread adoption has been slow and sometimes difficult.
The barriers to adoption of high-value practices among hospitalists are numerous and deep rooted in historical practices and culture. As Dr. Petrilli said, the “culture of overordering [diagnostic tests] is hard to break.” Hospitalists may not have well-developed relationships with patients, or time to explain why some tests or treatments are unnecessary. There is a lack of cost transparency, including the cost of the tests themselves and the downstream costs of additional tests and follow-ups. The best intended interventions fail to produce durable change unless they are seamlessly integrated into a hospitalist’s daily workflow.
Six steps to implementing a successful high-value care initiative
What can hospitalists do to improve the value of care they provide to their patients and hospital partners?
1. Identify high-value care opportunities at your hospital.
Dr. Wray pointed out that “all high-value care is local.” Start by looking at the national guidelines and talking to your senior clinical leaders and colleagues. Review your hospital data to identify opportunities and understand the root causes, including variability among providers.
If you choose to analyze and present provider-specific data, first be transparent on why you are doing that. Your goal is not to tell physicians how to practice or to score them, but instead, to promote adoption of evidence-based high-value care by identifying and discussing provider practice variations, and to generate possible solutions. Second, make sure that the data you present is credible and trustworthy by clearly outlining the data source, time frame, sample size per provider, any inclusion and exclusion criteria, attribution logic, and severity adjustment methodology. Third, expect initial pushback as transparency and change can be scary. But most doctors are inherently competitive and will want to be the best at caring for their patients.
2. Assemble the team.
Identify an executive sponsor – a senior clinical executive (for example, the chief medical officer or vice president of medical affairs) whose role is to help engage stakeholders, secure resources, and remove barriers. When assembling the rest of the team, include a representative from each major stakeholder group, but keep the team small enough to be effective. For example, if your project focuses on improving telemetry utilization, seek representation from hospitalists, cardiologists, nurses, utilization managers, and possibly IT. Look for people with the relevant knowledge and experience who are respected by their peers and can influence opinion.
3. Design a sustainable solution.
To be sustainable, a solution must be evidence based, well integrated in provider workflow, and have acceptable impact on daily workload (e.g., additional time per patient). If an estimated impact is significant, you need to discuss adding resources or negotiating trade-offs.
A great example of a sustainable solution, aimed to control overutilization of telemetry and urinary catheters, is the one implemented by Dr. Wray and his team.7 They designed an EHR-based “silent” indicator that clearly signaled an active telemetry or urinary catheter order for each patient. Clicking on the indicator directed a provider to a “manage order” screen where she could cancel the order, if necessary.
4. Engage providers.
You may design the best solution, but it will not succeed unless it is embraced by others. To engage providers, you must clearly communicate why the change is urgently needed for the benefit of their patients, hospital, or community, and appeal to their minds, hearts, and competitive nature.
For example, if you are focusing on overutilization of urinary catheters, you may share your hospital’s urinary catheter device utilization ratio (# of indwelling catheter days/# patient days) against national benchmarks, or the impact on hospital catheter–associated urinary tract infections (CAUTI) rates to appeal to the physicians’ minds. Often, data alone are not enough to move people to action. You must appeal to their hearts by sharing stories of real patients whose lives were affected by preventable CAUTI. Leverage physicians’ competitive nature by using provider-specific data to compare against their peers to spark a discussion.
5. Evaluate impact.
Even before you implement a solution, select metrics to measure impact and set SMART (specific, measurable, achievable, relevant, and time-bound) goals. As your implementation moves forward, do not let up or give up – continue to evaluate impact, remove barriers, refine your solution to get back on track if needed, and constantly communicate to share ongoing project results and lessons learned.
6. Sustain improvements.
Sustainable improvements require well-designed solutions integrated into provider workflow, but that is just the first step. Once you demonstrate the impact, consider including the metric (e.g., telemetry or urinary catheter utilization) in your team and/or individual provider performance dashboard, regularly reviewing and discussing performance during your team meetings to maintain engagement, and if needed, making improvements to get back on track.
Successful adoption of high-value care practices requires a disciplined approach to design and implement solutions that are patient-centric, evidence-based, data-driven and integrated in provider workflow.
Dr. Farah is a hospitalist, Physician Advisor, and Lean Six Sigma Black Belt. She is a performance improvement consultant based in Corvallis, Ore., and a member of The Hospitalist’s editorial advisory board.
References
1. From the Centers for Medicare & Medicaid Services: National Health Expenditure Projections 2018-2027.
2. Peterson-Kaiser Health System Tracker: How does health spending in the U.S. compare to other countries?
3. Creating a new culture of care, in “Best care at lower cost: The path to continuously learning health care in America.” (Washington: National Academies Press, 2013, pp. 255-80).
4. Choosing Wisely: SHM – Adult Hospital Medicine; Five things physicians and patients should question.
5. Qaseem A et al. Appropriate use of screening and diagnostic tests to foster high-value, cost-conscious care. Ann Intern Med. 2012 Jan 17;156(2):147-9.
6. Cho HJ et al. Right care in hospital medicine: Co-creation of ten opportunities in overuse and underuse for improving value in hospital medicine. J Gen Intern Med. 2018 Jun;33(6):804-6.
7. Wray CM et al. Improving value by reducing unnecessary telemetry and urinary catheter utilization in hospitalized patients. Am J Med. 2017 Sep;130(9):1037-41.
Dapagliflozin’s cardiovascular benefits bloom in T2D with prior MI
NEW ORLEANS – Dapagliflozin markedly reduces the risks of both major adverse cardiovascular events and heart failure hospitalization in the subset of patients with type 2 diabetes (T2D) and prior MI, according to a new subanalysis of the landmark DECLARE-TIMI 58 trial.
The effectiveness of dapagliflozin (Farxiga), an oral sodium glucose transporter-2 inhibitor (SGLT-2i), was particularly striking with regard to prevention of recurrent MI, Remo H.M. Furtado, MD, reported at the annual meeting of the American College of Cardiology.
“The 22% relative risk reduction in recurrent MI with dapagliflozin is comparable to other established therapies used in secondary prevention after MI, like DAPT [dual-antiplatelet therapy] and intensive lipid lowering,” observed Dr. Furtado of Brigham and Women’s Hospital, Boston.
Not bad for a drug developed as a glucose-lowering agent.
The new DECLARE-TIMI 58 subanalysis provides information that’s relevant to ACC guidelines issued in late 2018. The guidelines, in the form of an “expert consensus decision pathway,” emphatically recommend that all patients with T2D and known atherosclerotic cardiovascular disease (ASCVD) should have metformin as their first-line glucose-lowering agent, while at the same time giving serious consideration to the addition of either an oral SGLT-2i or a subcutaneously injected glucagonlike peptide–1 receptor agonist (GLP-1RA) with demonstrated cardiovascular benefit as a second glucose-lowering agent (J Am Coll Cardiol. 2018 Dec 18;72[24]:3200-23). The DECLARE-TIMI 58 subanalysis bolsters that guidance and shows, more specifically, that the cardioprotective benefits of dapagliflozin are significantly greater in T2D with prior MI than in those with known ASCVD but no history of MI, the cardiologist explained.
The main results of DECLARE-TIMI 58 have been published. The trial included 17,160 patients with T2D, 6,974 of whom had established ASCVD, while the remainder had multiple ASCVD risk factors. Participants were randomized to oral dapagliflozin at 10 mg/day or placebo on top of background guideline-directed medical therapy and followed for a median of 4.2 years. The dapagliflozin group had a 27% reduction in heart failure hospitalizations, compared with controls, but there were no significant between-group differences in the composite MACE (major adverse cardiovascular events) endpoint of cardiovascular death, MI, or ischemic stroke (N Engl J Med. 2019 Jan 24;380[4]:347-57).
Dr. Furtado presented a prespecified subgroup analysis focused on the 3,584 study participants with prior MI. Their rate of the composite endpoint of cardiovascular death, MI, or ischemic stroke was 15.2%, compared with 17.8% in controls, for a statistically significant and clinically meaningful 16% relative risk reduction and an absolute 2.6% risk reduction. Of note, the risk of recurrent MI was reduced by 22%. In contrast, there was no difference in MACE risk between the dapagliflozin and placebo groups in patients with no prior MI, even if they had established ASCVD.
A noteworthy finding was that the benefit in MACE reduction in patients with prior MI was greater in those who were closer in time to their most recent MI at enrollment in the study. Those who started on dapagliflozin within 12 months of their last MI had a 34% relative risk reduction in MACE, compared with placebo, while those who enrolled 12-24 months after their last MI enjoyed an even more robust 58% relative risk reduction on dapagliflozin. In contrast, patients who enrolled 24-36 months post MI had only a 17% relative risk reduction, and the 2,400 patients who enrolled more than 36 months after their last MI had a subsequent MACE rate no different from controls.
Session cochair Nadia R. Sutton, MD, a cardiologist at the University of Michigan, Ann Arbor, commented that she found this time-dependent benefit fascinating.
“Do you think this has anything to do with the escalation of other therapies, such as Plavix [clopidogrel]?” she asked.
Dr. Furtado replied, “This is a finding that we should interpret with a little bit of caution.” For one thing, patients in the acute phase of an MI were excluded from participation in the trial, so nothing is known about how they would fare on dapagliflozin. For another, only 844 of the 3,584 patients with T2D and prior MI had their most recent MI within 24 months of enrollment, so even though the differences were statistically significant, the confidence intervals are fairly wide.
That being said, the finding does underscore a truism about cardiovascular prevention: The higher the risk, the greater the benefit of effective therapy – and, of course, the initial months following an MI are a particularly high-risk period.
“Also, this finding is a caution to the clinicians to avoid clinical inertia in prescribing an SGLT-2i, because maybe you can get an early benefit if you prescribe the drug closer to the acute phase and not wait until some months after the patient has tried diet and exercise and so on,” he added.
With regard to the second coprimary endpoint comprising cardiovascular death or heart failure hospitalization in T2D patients with prior MI, the rate in the dapagliflozin group was 8.6%, a 19% relative risk reduction and absolute risk reduction of 1.9%, compared with the 10.5% rate with placebo.
Dr. Furtado noted that the main results of DECLARE TIMI-58 are consistent with a recent systematic review and meta-analysis of three randomized cardiovascular outcome trials of SGLT-2is for primary and secondary prevention of cardiovascular and renal outcomes in T2D. The meta-analysis included more than 34,000 patients, roughly half drawn from DECLARE-TIMI 58. SGLT-2i therapy reduced MACE by 14% in patients with established ASCVD but not significantly in those without. And the agents reduced the risk of cardiovascular death/heart failure hospitalization by 23%, regardless of whether or not patients had known ASCVD or a history of heart failure (Lancet. 2019 Jan 5;393[10166]:31-9).
Dr. Furtado reported serving as a consultant to AstraZeneca, which funded DECLARE-TIMI 58, as well as receiving direct institutional research grants from half a dozen other pharmaceutical companies.
Simultaneous with his presentation at ACC 2019 in New Orleans, the subanalysis results were published online (Circulation. 2019 Mar 18. doi: 10.1161/CIRCULATIONAHA.119.039996. [Epub ahead of print]).
NEW ORLEANS – Dapagliflozin markedly reduces the risks of both major adverse cardiovascular events and heart failure hospitalization in the subset of patients with type 2 diabetes (T2D) and prior MI, according to a new subanalysis of the landmark DECLARE-TIMI 58 trial.
The effectiveness of dapagliflozin (Farxiga), an oral sodium glucose transporter-2 inhibitor (SGLT-2i), was particularly striking with regard to prevention of recurrent MI, Remo H.M. Furtado, MD, reported at the annual meeting of the American College of Cardiology.
“The 22% relative risk reduction in recurrent MI with dapagliflozin is comparable to other established therapies used in secondary prevention after MI, like DAPT [dual-antiplatelet therapy] and intensive lipid lowering,” observed Dr. Furtado of Brigham and Women’s Hospital, Boston.
Not bad for a drug developed as a glucose-lowering agent.
The new DECLARE-TIMI 58 subanalysis provides information that’s relevant to ACC guidelines issued in late 2018. The guidelines, in the form of an “expert consensus decision pathway,” emphatically recommend that all patients with T2D and known atherosclerotic cardiovascular disease (ASCVD) should have metformin as their first-line glucose-lowering agent, while at the same time giving serious consideration to the addition of either an oral SGLT-2i or a subcutaneously injected glucagonlike peptide–1 receptor agonist (GLP-1RA) with demonstrated cardiovascular benefit as a second glucose-lowering agent (J Am Coll Cardiol. 2018 Dec 18;72[24]:3200-23). The DECLARE-TIMI 58 subanalysis bolsters that guidance and shows, more specifically, that the cardioprotective benefits of dapagliflozin are significantly greater in T2D with prior MI than in those with known ASCVD but no history of MI, the cardiologist explained.
The main results of DECLARE-TIMI 58 have been published. The trial included 17,160 patients with T2D, 6,974 of whom had established ASCVD, while the remainder had multiple ASCVD risk factors. Participants were randomized to oral dapagliflozin at 10 mg/day or placebo on top of background guideline-directed medical therapy and followed for a median of 4.2 years. The dapagliflozin group had a 27% reduction in heart failure hospitalizations, compared with controls, but there were no significant between-group differences in the composite MACE (major adverse cardiovascular events) endpoint of cardiovascular death, MI, or ischemic stroke (N Engl J Med. 2019 Jan 24;380[4]:347-57).
Dr. Furtado presented a prespecified subgroup analysis focused on the 3,584 study participants with prior MI. Their rate of the composite endpoint of cardiovascular death, MI, or ischemic stroke was 15.2%, compared with 17.8% in controls, for a statistically significant and clinically meaningful 16% relative risk reduction and an absolute 2.6% risk reduction. Of note, the risk of recurrent MI was reduced by 22%. In contrast, there was no difference in MACE risk between the dapagliflozin and placebo groups in patients with no prior MI, even if they had established ASCVD.
A noteworthy finding was that the benefit in MACE reduction in patients with prior MI was greater in those who were closer in time to their most recent MI at enrollment in the study. Those who started on dapagliflozin within 12 months of their last MI had a 34% relative risk reduction in MACE, compared with placebo, while those who enrolled 12-24 months after their last MI enjoyed an even more robust 58% relative risk reduction on dapagliflozin. In contrast, patients who enrolled 24-36 months post MI had only a 17% relative risk reduction, and the 2,400 patients who enrolled more than 36 months after their last MI had a subsequent MACE rate no different from controls.
Session cochair Nadia R. Sutton, MD, a cardiologist at the University of Michigan, Ann Arbor, commented that she found this time-dependent benefit fascinating.
“Do you think this has anything to do with the escalation of other therapies, such as Plavix [clopidogrel]?” she asked.
Dr. Furtado replied, “This is a finding that we should interpret with a little bit of caution.” For one thing, patients in the acute phase of an MI were excluded from participation in the trial, so nothing is known about how they would fare on dapagliflozin. For another, only 844 of the 3,584 patients with T2D and prior MI had their most recent MI within 24 months of enrollment, so even though the differences were statistically significant, the confidence intervals are fairly wide.
That being said, the finding does underscore a truism about cardiovascular prevention: The higher the risk, the greater the benefit of effective therapy – and, of course, the initial months following an MI are a particularly high-risk period.
“Also, this finding is a caution to the clinicians to avoid clinical inertia in prescribing an SGLT-2i, because maybe you can get an early benefit if you prescribe the drug closer to the acute phase and not wait until some months after the patient has tried diet and exercise and so on,” he added.
With regard to the second coprimary endpoint comprising cardiovascular death or heart failure hospitalization in T2D patients with prior MI, the rate in the dapagliflozin group was 8.6%, a 19% relative risk reduction and absolute risk reduction of 1.9%, compared with the 10.5% rate with placebo.
Dr. Furtado noted that the main results of DECLARE TIMI-58 are consistent with a recent systematic review and meta-analysis of three randomized cardiovascular outcome trials of SGLT-2is for primary and secondary prevention of cardiovascular and renal outcomes in T2D. The meta-analysis included more than 34,000 patients, roughly half drawn from DECLARE-TIMI 58. SGLT-2i therapy reduced MACE by 14% in patients with established ASCVD but not significantly in those without. And the agents reduced the risk of cardiovascular death/heart failure hospitalization by 23%, regardless of whether or not patients had known ASCVD or a history of heart failure (Lancet. 2019 Jan 5;393[10166]:31-9).
Dr. Furtado reported serving as a consultant to AstraZeneca, which funded DECLARE-TIMI 58, as well as receiving direct institutional research grants from half a dozen other pharmaceutical companies.
Simultaneous with his presentation at ACC 2019 in New Orleans, the subanalysis results were published online (Circulation. 2019 Mar 18. doi: 10.1161/CIRCULATIONAHA.119.039996. [Epub ahead of print]).
NEW ORLEANS – Dapagliflozin markedly reduces the risks of both major adverse cardiovascular events and heart failure hospitalization in the subset of patients with type 2 diabetes (T2D) and prior MI, according to a new subanalysis of the landmark DECLARE-TIMI 58 trial.
The effectiveness of dapagliflozin (Farxiga), an oral sodium glucose transporter-2 inhibitor (SGLT-2i), was particularly striking with regard to prevention of recurrent MI, Remo H.M. Furtado, MD, reported at the annual meeting of the American College of Cardiology.
“The 22% relative risk reduction in recurrent MI with dapagliflozin is comparable to other established therapies used in secondary prevention after MI, like DAPT [dual-antiplatelet therapy] and intensive lipid lowering,” observed Dr. Furtado of Brigham and Women’s Hospital, Boston.
Not bad for a drug developed as a glucose-lowering agent.
The new DECLARE-TIMI 58 subanalysis provides information that’s relevant to ACC guidelines issued in late 2018. The guidelines, in the form of an “expert consensus decision pathway,” emphatically recommend that all patients with T2D and known atherosclerotic cardiovascular disease (ASCVD) should have metformin as their first-line glucose-lowering agent, while at the same time giving serious consideration to the addition of either an oral SGLT-2i or a subcutaneously injected glucagonlike peptide–1 receptor agonist (GLP-1RA) with demonstrated cardiovascular benefit as a second glucose-lowering agent (J Am Coll Cardiol. 2018 Dec 18;72[24]:3200-23). The DECLARE-TIMI 58 subanalysis bolsters that guidance and shows, more specifically, that the cardioprotective benefits of dapagliflozin are significantly greater in T2D with prior MI than in those with known ASCVD but no history of MI, the cardiologist explained.
The main results of DECLARE-TIMI 58 have been published. The trial included 17,160 patients with T2D, 6,974 of whom had established ASCVD, while the remainder had multiple ASCVD risk factors. Participants were randomized to oral dapagliflozin at 10 mg/day or placebo on top of background guideline-directed medical therapy and followed for a median of 4.2 years. The dapagliflozin group had a 27% reduction in heart failure hospitalizations, compared with controls, but there were no significant between-group differences in the composite MACE (major adverse cardiovascular events) endpoint of cardiovascular death, MI, or ischemic stroke (N Engl J Med. 2019 Jan 24;380[4]:347-57).
Dr. Furtado presented a prespecified subgroup analysis focused on the 3,584 study participants with prior MI. Their rate of the composite endpoint of cardiovascular death, MI, or ischemic stroke was 15.2%, compared with 17.8% in controls, for a statistically significant and clinically meaningful 16% relative risk reduction and an absolute 2.6% risk reduction. Of note, the risk of recurrent MI was reduced by 22%. In contrast, there was no difference in MACE risk between the dapagliflozin and placebo groups in patients with no prior MI, even if they had established ASCVD.
A noteworthy finding was that the benefit in MACE reduction in patients with prior MI was greater in those who were closer in time to their most recent MI at enrollment in the study. Those who started on dapagliflozin within 12 months of their last MI had a 34% relative risk reduction in MACE, compared with placebo, while those who enrolled 12-24 months after their last MI enjoyed an even more robust 58% relative risk reduction on dapagliflozin. In contrast, patients who enrolled 24-36 months post MI had only a 17% relative risk reduction, and the 2,400 patients who enrolled more than 36 months after their last MI had a subsequent MACE rate no different from controls.
Session cochair Nadia R. Sutton, MD, a cardiologist at the University of Michigan, Ann Arbor, commented that she found this time-dependent benefit fascinating.
“Do you think this has anything to do with the escalation of other therapies, such as Plavix [clopidogrel]?” she asked.
Dr. Furtado replied, “This is a finding that we should interpret with a little bit of caution.” For one thing, patients in the acute phase of an MI were excluded from participation in the trial, so nothing is known about how they would fare on dapagliflozin. For another, only 844 of the 3,584 patients with T2D and prior MI had their most recent MI within 24 months of enrollment, so even though the differences were statistically significant, the confidence intervals are fairly wide.
That being said, the finding does underscore a truism about cardiovascular prevention: The higher the risk, the greater the benefit of effective therapy – and, of course, the initial months following an MI are a particularly high-risk period.
“Also, this finding is a caution to the clinicians to avoid clinical inertia in prescribing an SGLT-2i, because maybe you can get an early benefit if you prescribe the drug closer to the acute phase and not wait until some months after the patient has tried diet and exercise and so on,” he added.
With regard to the second coprimary endpoint comprising cardiovascular death or heart failure hospitalization in T2D patients with prior MI, the rate in the dapagliflozin group was 8.6%, a 19% relative risk reduction and absolute risk reduction of 1.9%, compared with the 10.5% rate with placebo.
Dr. Furtado noted that the main results of DECLARE TIMI-58 are consistent with a recent systematic review and meta-analysis of three randomized cardiovascular outcome trials of SGLT-2is for primary and secondary prevention of cardiovascular and renal outcomes in T2D. The meta-analysis included more than 34,000 patients, roughly half drawn from DECLARE-TIMI 58. SGLT-2i therapy reduced MACE by 14% in patients with established ASCVD but not significantly in those without. And the agents reduced the risk of cardiovascular death/heart failure hospitalization by 23%, regardless of whether or not patients had known ASCVD or a history of heart failure (Lancet. 2019 Jan 5;393[10166]:31-9).
Dr. Furtado reported serving as a consultant to AstraZeneca, which funded DECLARE-TIMI 58, as well as receiving direct institutional research grants from half a dozen other pharmaceutical companies.
Simultaneous with his presentation at ACC 2019 in New Orleans, the subanalysis results were published online (Circulation. 2019 Mar 18. doi: 10.1161/CIRCULATIONAHA.119.039996. [Epub ahead of print]).
REPORTING FROM ACC 19
Pembrolizumab benefits some patients with advanced cervical cancer
The checkpoint inhibitor pembrolizumab conferred a durable tumor response in patients with advanced cervical cancer, who were positive for programmed death ligand 1 (PD-L1).
Among 98 patients, all of whom had progressed despite prior treatment, overall response rate was 12.2% (95% confidence interval, 6.5%-20.4%), with three complete and nine partial responses. All 12 responses were in patients with PD-L1-positive tumors, wrote Hyun Cheol Chung, MD, and his colleagues. The report is in the Journal of Clinical Oncology.
The response curve had not peaked by the end of follow-up, suggesting further benefit of the antibody, said Dr. Chung of Yonsei Cancer Center, Seoul, South Korea. On the basis of these data, first reported at the 2018 meeting of the American Society of Clinical Oncology, the Food and Drug Administration approved the antibody for advanced cervical cancer last year.
“These results show that treatment with pembrolizumab offers a clinically meaningful therapeutic option for a subset of patients with previously treated advanced cervical cancer,” the researchers said. The approval makes pembrolizumab the first immunotherapy approved for the treatment of an advanced gynecologic malignancy.
The phase 2 KEYNOTE-158 study comprised 98 women (median age 46 years) with advanced cervical cancer with progression after chemotherapy. All received pembrolizumab 200 mg every 3 weeks for 2 years or until progression, intolerable toxicity, or physician or patient decision.
Most of the patients (83.7%) were positive for PD-L1. All had received at least one or more lines of chemotherapy before entering the study. The median follow-up was 10 months and the median treatment duration 2.9 months, with a range of 1 day-22 months.
By the study’s end, most patients (85.7%) had experienced disease progression or had died. Overall survival was poor even in the PD-L1-positive patients, with 82.9% experiencing progression or death. Overall mortality was 69.4% and 64.6%, respectively. However, PD-L1 patients did live longer, a median of 11 months, compared with 9.4 months in the entire cohort.
At 6 months, overall survival estimates were 75.2% in the total cohort and 80.2% in the PD-L1 patients; 12-month overall survival estimates were 41.4% and 47.3%, respectively.
However, among the 12 responders with PD-L1-postitive tumors, just one had died by the data cutoff, suggesting that longer treatment with pembrolizumab could be beneficial in this group.
“Responses typically occurred within 2.1 months and were durable, with a median duration of response that had not been reached after a median follow-up of 10.2 months and an estimated 90.9% of responses ongoing at 6 months” Dr. Chung and his colleagues wrote.
Adverse events were common (65.3%) with 12.2% graded as serious. But none were lethal, and only four patients discontinued pembrolizumab because of a treatment-related adverse event.
“Our results from an interim analysis of data from the phase II KEYNOTE-158 clinical trial show that pembrolizumab has promising antitumor activity in patients with previously treated advanced cervical cancer,” the researchers said. “These response rates are similar or superior to those observed with other treatment options in this setting.”
Pembrolizumab is now being evaluated in combination with concurrent chemoradiotherapy and with concurrent versus sequential chemoradiotherapy.
Merck & Co. sponsored the study.
SOURCE: Chung HC et al. J Clin Oncol. 2019 April 3. doi: 10.1200/JCO.18.01265.
The checkpoint inhibitor pembrolizumab conferred a durable tumor response in patients with advanced cervical cancer, who were positive for programmed death ligand 1 (PD-L1).
Among 98 patients, all of whom had progressed despite prior treatment, overall response rate was 12.2% (95% confidence interval, 6.5%-20.4%), with three complete and nine partial responses. All 12 responses were in patients with PD-L1-positive tumors, wrote Hyun Cheol Chung, MD, and his colleagues. The report is in the Journal of Clinical Oncology.
The response curve had not peaked by the end of follow-up, suggesting further benefit of the antibody, said Dr. Chung of Yonsei Cancer Center, Seoul, South Korea. On the basis of these data, first reported at the 2018 meeting of the American Society of Clinical Oncology, the Food and Drug Administration approved the antibody for advanced cervical cancer last year.
“These results show that treatment with pembrolizumab offers a clinically meaningful therapeutic option for a subset of patients with previously treated advanced cervical cancer,” the researchers said. The approval makes pembrolizumab the first immunotherapy approved for the treatment of an advanced gynecologic malignancy.
The phase 2 KEYNOTE-158 study comprised 98 women (median age 46 years) with advanced cervical cancer with progression after chemotherapy. All received pembrolizumab 200 mg every 3 weeks for 2 years or until progression, intolerable toxicity, or physician or patient decision.
Most of the patients (83.7%) were positive for PD-L1. All had received at least one or more lines of chemotherapy before entering the study. The median follow-up was 10 months and the median treatment duration 2.9 months, with a range of 1 day-22 months.
By the study’s end, most patients (85.7%) had experienced disease progression or had died. Overall survival was poor even in the PD-L1-positive patients, with 82.9% experiencing progression or death. Overall mortality was 69.4% and 64.6%, respectively. However, PD-L1 patients did live longer, a median of 11 months, compared with 9.4 months in the entire cohort.
At 6 months, overall survival estimates were 75.2% in the total cohort and 80.2% in the PD-L1 patients; 12-month overall survival estimates were 41.4% and 47.3%, respectively.
However, among the 12 responders with PD-L1-postitive tumors, just one had died by the data cutoff, suggesting that longer treatment with pembrolizumab could be beneficial in this group.
“Responses typically occurred within 2.1 months and were durable, with a median duration of response that had not been reached after a median follow-up of 10.2 months and an estimated 90.9% of responses ongoing at 6 months” Dr. Chung and his colleagues wrote.
Adverse events were common (65.3%) with 12.2% graded as serious. But none were lethal, and only four patients discontinued pembrolizumab because of a treatment-related adverse event.
“Our results from an interim analysis of data from the phase II KEYNOTE-158 clinical trial show that pembrolizumab has promising antitumor activity in patients with previously treated advanced cervical cancer,” the researchers said. “These response rates are similar or superior to those observed with other treatment options in this setting.”
Pembrolizumab is now being evaluated in combination with concurrent chemoradiotherapy and with concurrent versus sequential chemoradiotherapy.
Merck & Co. sponsored the study.
SOURCE: Chung HC et al. J Clin Oncol. 2019 April 3. doi: 10.1200/JCO.18.01265.
The checkpoint inhibitor pembrolizumab conferred a durable tumor response in patients with advanced cervical cancer, who were positive for programmed death ligand 1 (PD-L1).
Among 98 patients, all of whom had progressed despite prior treatment, overall response rate was 12.2% (95% confidence interval, 6.5%-20.4%), with three complete and nine partial responses. All 12 responses were in patients with PD-L1-positive tumors, wrote Hyun Cheol Chung, MD, and his colleagues. The report is in the Journal of Clinical Oncology.
The response curve had not peaked by the end of follow-up, suggesting further benefit of the antibody, said Dr. Chung of Yonsei Cancer Center, Seoul, South Korea. On the basis of these data, first reported at the 2018 meeting of the American Society of Clinical Oncology, the Food and Drug Administration approved the antibody for advanced cervical cancer last year.
“These results show that treatment with pembrolizumab offers a clinically meaningful therapeutic option for a subset of patients with previously treated advanced cervical cancer,” the researchers said. The approval makes pembrolizumab the first immunotherapy approved for the treatment of an advanced gynecologic malignancy.
The phase 2 KEYNOTE-158 study comprised 98 women (median age 46 years) with advanced cervical cancer with progression after chemotherapy. All received pembrolizumab 200 mg every 3 weeks for 2 years or until progression, intolerable toxicity, or physician or patient decision.
Most of the patients (83.7%) were positive for PD-L1. All had received at least one or more lines of chemotherapy before entering the study. The median follow-up was 10 months and the median treatment duration 2.9 months, with a range of 1 day-22 months.
By the study’s end, most patients (85.7%) had experienced disease progression or had died. Overall survival was poor even in the PD-L1-positive patients, with 82.9% experiencing progression or death. Overall mortality was 69.4% and 64.6%, respectively. However, PD-L1 patients did live longer, a median of 11 months, compared with 9.4 months in the entire cohort.
At 6 months, overall survival estimates were 75.2% in the total cohort and 80.2% in the PD-L1 patients; 12-month overall survival estimates were 41.4% and 47.3%, respectively.
However, among the 12 responders with PD-L1-postitive tumors, just one had died by the data cutoff, suggesting that longer treatment with pembrolizumab could be beneficial in this group.
“Responses typically occurred within 2.1 months and were durable, with a median duration of response that had not been reached after a median follow-up of 10.2 months and an estimated 90.9% of responses ongoing at 6 months” Dr. Chung and his colleagues wrote.
Adverse events were common (65.3%) with 12.2% graded as serious. But none were lethal, and only four patients discontinued pembrolizumab because of a treatment-related adverse event.
“Our results from an interim analysis of data from the phase II KEYNOTE-158 clinical trial show that pembrolizumab has promising antitumor activity in patients with previously treated advanced cervical cancer,” the researchers said. “These response rates are similar or superior to those observed with other treatment options in this setting.”
Pembrolizumab is now being evaluated in combination with concurrent chemoradiotherapy and with concurrent versus sequential chemoradiotherapy.
Merck & Co. sponsored the study.
SOURCE: Chung HC et al. J Clin Oncol. 2019 April 3. doi: 10.1200/JCO.18.01265.
FROM JOURNAL OF CLINICAL ONCOLOGY
Look for functional esophageal disorders if GERD patients fail PPIs
In a small, first-in-kind study, functional heartburn or reflux hypersensitivity affected fully 75% of patients whose gastroesophageal reflux disease symptoms had not improved with once-daily proton pump inhibitor therapy.
At the same time, proton pump inhibitor (PPI) responders and nonresponders had similar impedance and pH parameters, reported Jason Abdallah, MD, of Case Western Reserve University, Cleveland, and his associates. The findings show “the important role of esophageal hypersensitivity in this patient population.” For these patients, the investigators suggested adding a neuromodulator and possibly psychotherapy, such as cognitive-behavioral therapy, hypnotherapy, relaxation techniques, mindfulness, and biofeedback.
Symptoms in up to 45% of patients with gastroesophageal reflux disease (GERD) persist despite once-daily PPI therapy, Dr. Abdallah and his associates wrote in Clinical Gastroenterology and Hepatology. For the study, they compared reflux characteristics and patterns between 13 patients whose GERD symptoms had fully resolved on standard once-daily PPIs and 16 patients who reported at least twice-weekly heartburn, regurgitation, or both for at least 3 months, despite treatment. Patients in both groups continued PPIs and underwent upper endoscopy and combined esophageal impedance–pH monitoring.
The average age of patients in this study was 54.5 years, with a standard deviation of 14.5 years. Demographic and clinical characteristics were similar between groups, and patients in both groups were receiving omeprazole, esomeprazole, or pantoprazole. Four (31%) PPI responders had abnormal pH test results, as did four (25%) nonresponders. Responders and nonresponders had similar numbers of reflux events; proportions of events that were acidic, weakly acidic, or alkaline; and mean total time with pH less than 4.0.
Additionally, most patients in both groups had normal endoscopic findings. One PPI responder had Los Angeles grade A erosive esophagitis, and two PPI responders had short-segment Barrett’s esophagus. Three PPI responders and two PPI nonresponders had nonobstructive Schatzki rings, and one PPI nonresponder had an esophageal stricture. Finally, five PPI responders (38%) and three nonresponders (19%) had hiatal hernias (P = .41).
In patients with GERD, “PPI failure” does not reflect a unique pattern of reflux events, acid exposure, or nonacidic reflux parameters, Dr. Abdallah and his associates concluded. The fact that most PPI nonresponders had a concurrent functional esophageal disorder – either reflux hypersensitivity or functional heartburn – “support[s] the hypothesis that PPI failure is primarily driven by esophageal hypersensitivity.”
This was a small study – recruitment “was hampered by the invasive nature of some of the procedures,” they wrote. “In addition, it is our experience that many patients who have responded to PPI [therapy] are reluctant to undergo invasive testing as part of a study protocol. Therefore, we believe that these types of prospective, invasive studies are rather difficult to perform but, at the same time, provide essential insight into the understanding of refractory GERD.”
No external funding sources were acknowledged. The senior author reported ties to Ironwood Pharmaceuticals, Mederi Therapeutics, Ethicon, AstraZeneca, and Takeda. The other researchers reported no conflicts of interest.
SOURCE: Abdallah J et al. Clin Gastroenterol Hepatol. 2018 Jun 15. doi: 10.1016/j.cgh.2018.09.006.
In a small, first-in-kind study, functional heartburn or reflux hypersensitivity affected fully 75% of patients whose gastroesophageal reflux disease symptoms had not improved with once-daily proton pump inhibitor therapy.
At the same time, proton pump inhibitor (PPI) responders and nonresponders had similar impedance and pH parameters, reported Jason Abdallah, MD, of Case Western Reserve University, Cleveland, and his associates. The findings show “the important role of esophageal hypersensitivity in this patient population.” For these patients, the investigators suggested adding a neuromodulator and possibly psychotherapy, such as cognitive-behavioral therapy, hypnotherapy, relaxation techniques, mindfulness, and biofeedback.
Symptoms in up to 45% of patients with gastroesophageal reflux disease (GERD) persist despite once-daily PPI therapy, Dr. Abdallah and his associates wrote in Clinical Gastroenterology and Hepatology. For the study, they compared reflux characteristics and patterns between 13 patients whose GERD symptoms had fully resolved on standard once-daily PPIs and 16 patients who reported at least twice-weekly heartburn, regurgitation, or both for at least 3 months, despite treatment. Patients in both groups continued PPIs and underwent upper endoscopy and combined esophageal impedance–pH monitoring.
The average age of patients in this study was 54.5 years, with a standard deviation of 14.5 years. Demographic and clinical characteristics were similar between groups, and patients in both groups were receiving omeprazole, esomeprazole, or pantoprazole. Four (31%) PPI responders had abnormal pH test results, as did four (25%) nonresponders. Responders and nonresponders had similar numbers of reflux events; proportions of events that were acidic, weakly acidic, or alkaline; and mean total time with pH less than 4.0.
Additionally, most patients in both groups had normal endoscopic findings. One PPI responder had Los Angeles grade A erosive esophagitis, and two PPI responders had short-segment Barrett’s esophagus. Three PPI responders and two PPI nonresponders had nonobstructive Schatzki rings, and one PPI nonresponder had an esophageal stricture. Finally, five PPI responders (38%) and three nonresponders (19%) had hiatal hernias (P = .41).
In patients with GERD, “PPI failure” does not reflect a unique pattern of reflux events, acid exposure, or nonacidic reflux parameters, Dr. Abdallah and his associates concluded. The fact that most PPI nonresponders had a concurrent functional esophageal disorder – either reflux hypersensitivity or functional heartburn – “support[s] the hypothesis that PPI failure is primarily driven by esophageal hypersensitivity.”
This was a small study – recruitment “was hampered by the invasive nature of some of the procedures,” they wrote. “In addition, it is our experience that many patients who have responded to PPI [therapy] are reluctant to undergo invasive testing as part of a study protocol. Therefore, we believe that these types of prospective, invasive studies are rather difficult to perform but, at the same time, provide essential insight into the understanding of refractory GERD.”
No external funding sources were acknowledged. The senior author reported ties to Ironwood Pharmaceuticals, Mederi Therapeutics, Ethicon, AstraZeneca, and Takeda. The other researchers reported no conflicts of interest.
SOURCE: Abdallah J et al. Clin Gastroenterol Hepatol. 2018 Jun 15. doi: 10.1016/j.cgh.2018.09.006.
In a small, first-in-kind study, functional heartburn or reflux hypersensitivity affected fully 75% of patients whose gastroesophageal reflux disease symptoms had not improved with once-daily proton pump inhibitor therapy.
At the same time, proton pump inhibitor (PPI) responders and nonresponders had similar impedance and pH parameters, reported Jason Abdallah, MD, of Case Western Reserve University, Cleveland, and his associates. The findings show “the important role of esophageal hypersensitivity in this patient population.” For these patients, the investigators suggested adding a neuromodulator and possibly psychotherapy, such as cognitive-behavioral therapy, hypnotherapy, relaxation techniques, mindfulness, and biofeedback.
Symptoms in up to 45% of patients with gastroesophageal reflux disease (GERD) persist despite once-daily PPI therapy, Dr. Abdallah and his associates wrote in Clinical Gastroenterology and Hepatology. For the study, they compared reflux characteristics and patterns between 13 patients whose GERD symptoms had fully resolved on standard once-daily PPIs and 16 patients who reported at least twice-weekly heartburn, regurgitation, or both for at least 3 months, despite treatment. Patients in both groups continued PPIs and underwent upper endoscopy and combined esophageal impedance–pH monitoring.
The average age of patients in this study was 54.5 years, with a standard deviation of 14.5 years. Demographic and clinical characteristics were similar between groups, and patients in both groups were receiving omeprazole, esomeprazole, or pantoprazole. Four (31%) PPI responders had abnormal pH test results, as did four (25%) nonresponders. Responders and nonresponders had similar numbers of reflux events; proportions of events that were acidic, weakly acidic, or alkaline; and mean total time with pH less than 4.0.
Additionally, most patients in both groups had normal endoscopic findings. One PPI responder had Los Angeles grade A erosive esophagitis, and two PPI responders had short-segment Barrett’s esophagus. Three PPI responders and two PPI nonresponders had nonobstructive Schatzki rings, and one PPI nonresponder had an esophageal stricture. Finally, five PPI responders (38%) and three nonresponders (19%) had hiatal hernias (P = .41).
In patients with GERD, “PPI failure” does not reflect a unique pattern of reflux events, acid exposure, or nonacidic reflux parameters, Dr. Abdallah and his associates concluded. The fact that most PPI nonresponders had a concurrent functional esophageal disorder – either reflux hypersensitivity or functional heartburn – “support[s] the hypothesis that PPI failure is primarily driven by esophageal hypersensitivity.”
This was a small study – recruitment “was hampered by the invasive nature of some of the procedures,” they wrote. “In addition, it is our experience that many patients who have responded to PPI [therapy] are reluctant to undergo invasive testing as part of a study protocol. Therefore, we believe that these types of prospective, invasive studies are rather difficult to perform but, at the same time, provide essential insight into the understanding of refractory GERD.”
No external funding sources were acknowledged. The senior author reported ties to Ironwood Pharmaceuticals, Mederi Therapeutics, Ethicon, AstraZeneca, and Takeda. The other researchers reported no conflicts of interest.
SOURCE: Abdallah J et al. Clin Gastroenterol Hepatol. 2018 Jun 15. doi: 10.1016/j.cgh.2018.09.006.
FROM CLINICAL GASTROENTEROLOGY AND HEPATOLOGY
Alirocumab reduces both type 1 and 2 MIs
NEW ORLEANS – Lowering LDL cholesterol with alirocumab to levels below what’s achievable with intensive statin therapy appears to be an important strategy for prevention of type 1 MI – and perhaps even more impressively, type 2 MI – following acute coronary syndrome, Harvey D. White, MD, reported at the annual meeting of the American College of Cardiology.
What’s so important about the 23% reduction in risk of type 2 MI achieved with alirocumab (Praluent) relative to placebo documented in a prespecified secondary analysis from the ODYSSEY Outcomes trial?
“For type 2 MI, this is the first data indicating that a lipid-lowering therapy can attenuate risk,” according to Dr. White, a cardiologist at Auckland (N.Z.) City Hospital.
The ODYSSEY Outcomes trial compared the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor alirocumab to placebo in 18,924 patients with a recent acute coronary syndrome and an LDL cholesterol level of at least 70 mg/dL despite intensive statin therapy. At 4 months, the PCSK9 inhibitor plus statin therapy reduced participants’ mean LDL by 54%, from 93 to 48 mg/dL, while the LDL level actually drifted upward in the control group on placebo plus statin therapy. In the previously reported primary results of this landmark randomized clinical trial, alirocumab on top of background intensive statin therapy reduced the primary composite endpoint of death attributable to coronary heart disease, ischemic stroke, MI, or unstable angina requiring hospitalization by 15%, compared with controls (N Engl J Med. 2018 Nov 29;379[22]:2097-107).
During a median 2.8 years of prospective follow-up, there were 1,860 new MIs in study participants. A blinded clinical events committee evaluated the myocardial infarctions according to the Third Universal Definition and determined 66% were type 1 MIs, 21% were type 2, and 13% were type 4, with lesser numbers of types 3 and 5 MI.
Alirocumab reduced the risk of any MI by 15%, with a 6.8% incidence during follow-up, compared with 7.9% on placebo. The risk of type 1 MI, typically attributable to plaque rupture, was reduced by 13%, with an incidence of 4.9% with alirocumab and 5.6% with placebo. The risk reduction conferred by the PCSK9 inhibitor was even more robust for type 2 MI, the type caused by an oxygen supply/demand imbalance most commonly attributable to coronary artery spasm, coronary embolism, arrhythmias, anemia, hypertension, or hypotension: a 23% relative risk reduction as reflected in a 1.3% incidence in the alirocumab group, compared with a 1.7% rate in controls.
In contrast, alirocumab had no impact on the incidence of type 4 MI, a category that includes peri–percutaneous coronary intervention MIs as well as those attributable to stent thrombosis or restenosis.
The beneficial effect of alirocumab on MI risk mostly involved a reduction in larger MIs – those with a biomarker peak greater than three times the upper limit of normal.
An emphatic difference was found in the risk of death following type 1 as opposed to type 2 MI. Patients who experienced a type 1 MI during the study had an 11.9% mortality rate during an average of 1.6 years of post-MI follow-up, as compared with a 25.4% rate during 1.3 years of follow-up after a type 2 MI.
Alirocumab significantly reduced the risk of mortality following a type 1 MI, with a 10.2% rate as compared to 13.4% with placebo; that’s a 31% relative risk reduction. Yet the PCSK9 inhibitor had no impact on the risk of death after a type 2 MI: 24.8% in the alirocumab group and 25.9% in controls.
Asked for his thoughts as to possible explanatory mechanistic pathways for the benefit of alirocumab in preventing type 2 MI, Dr. White noted that in a Scottish study of the PCSK9 inhibitor evolocumab (Repatha), over the course of 72 months the drug appeared to reduce atherosclerotic progression and induce plaque stabilization and perhaps even regression.
“I think that’s the probable mechanism. And we also know that statins improve endothelial function,” he said.
He reported receiving research grant support and consultant fees from Sanofi and Regeneron, funders of the ODYSSEY Outcomes trial.
NEW ORLEANS – Lowering LDL cholesterol with alirocumab to levels below what’s achievable with intensive statin therapy appears to be an important strategy for prevention of type 1 MI – and perhaps even more impressively, type 2 MI – following acute coronary syndrome, Harvey D. White, MD, reported at the annual meeting of the American College of Cardiology.
What’s so important about the 23% reduction in risk of type 2 MI achieved with alirocumab (Praluent) relative to placebo documented in a prespecified secondary analysis from the ODYSSEY Outcomes trial?
“For type 2 MI, this is the first data indicating that a lipid-lowering therapy can attenuate risk,” according to Dr. White, a cardiologist at Auckland (N.Z.) City Hospital.
The ODYSSEY Outcomes trial compared the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor alirocumab to placebo in 18,924 patients with a recent acute coronary syndrome and an LDL cholesterol level of at least 70 mg/dL despite intensive statin therapy. At 4 months, the PCSK9 inhibitor plus statin therapy reduced participants’ mean LDL by 54%, from 93 to 48 mg/dL, while the LDL level actually drifted upward in the control group on placebo plus statin therapy. In the previously reported primary results of this landmark randomized clinical trial, alirocumab on top of background intensive statin therapy reduced the primary composite endpoint of death attributable to coronary heart disease, ischemic stroke, MI, or unstable angina requiring hospitalization by 15%, compared with controls (N Engl J Med. 2018 Nov 29;379[22]:2097-107).
During a median 2.8 years of prospective follow-up, there were 1,860 new MIs in study participants. A blinded clinical events committee evaluated the myocardial infarctions according to the Third Universal Definition and determined 66% were type 1 MIs, 21% were type 2, and 13% were type 4, with lesser numbers of types 3 and 5 MI.
Alirocumab reduced the risk of any MI by 15%, with a 6.8% incidence during follow-up, compared with 7.9% on placebo. The risk of type 1 MI, typically attributable to plaque rupture, was reduced by 13%, with an incidence of 4.9% with alirocumab and 5.6% with placebo. The risk reduction conferred by the PCSK9 inhibitor was even more robust for type 2 MI, the type caused by an oxygen supply/demand imbalance most commonly attributable to coronary artery spasm, coronary embolism, arrhythmias, anemia, hypertension, or hypotension: a 23% relative risk reduction as reflected in a 1.3% incidence in the alirocumab group, compared with a 1.7% rate in controls.
In contrast, alirocumab had no impact on the incidence of type 4 MI, a category that includes peri–percutaneous coronary intervention MIs as well as those attributable to stent thrombosis or restenosis.
The beneficial effect of alirocumab on MI risk mostly involved a reduction in larger MIs – those with a biomarker peak greater than three times the upper limit of normal.
An emphatic difference was found in the risk of death following type 1 as opposed to type 2 MI. Patients who experienced a type 1 MI during the study had an 11.9% mortality rate during an average of 1.6 years of post-MI follow-up, as compared with a 25.4% rate during 1.3 years of follow-up after a type 2 MI.
Alirocumab significantly reduced the risk of mortality following a type 1 MI, with a 10.2% rate as compared to 13.4% with placebo; that’s a 31% relative risk reduction. Yet the PCSK9 inhibitor had no impact on the risk of death after a type 2 MI: 24.8% in the alirocumab group and 25.9% in controls.
Asked for his thoughts as to possible explanatory mechanistic pathways for the benefit of alirocumab in preventing type 2 MI, Dr. White noted that in a Scottish study of the PCSK9 inhibitor evolocumab (Repatha), over the course of 72 months the drug appeared to reduce atherosclerotic progression and induce plaque stabilization and perhaps even regression.
“I think that’s the probable mechanism. And we also know that statins improve endothelial function,” he said.
He reported receiving research grant support and consultant fees from Sanofi and Regeneron, funders of the ODYSSEY Outcomes trial.
NEW ORLEANS – Lowering LDL cholesterol with alirocumab to levels below what’s achievable with intensive statin therapy appears to be an important strategy for prevention of type 1 MI – and perhaps even more impressively, type 2 MI – following acute coronary syndrome, Harvey D. White, MD, reported at the annual meeting of the American College of Cardiology.
What’s so important about the 23% reduction in risk of type 2 MI achieved with alirocumab (Praluent) relative to placebo documented in a prespecified secondary analysis from the ODYSSEY Outcomes trial?
“For type 2 MI, this is the first data indicating that a lipid-lowering therapy can attenuate risk,” according to Dr. White, a cardiologist at Auckland (N.Z.) City Hospital.
The ODYSSEY Outcomes trial compared the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor alirocumab to placebo in 18,924 patients with a recent acute coronary syndrome and an LDL cholesterol level of at least 70 mg/dL despite intensive statin therapy. At 4 months, the PCSK9 inhibitor plus statin therapy reduced participants’ mean LDL by 54%, from 93 to 48 mg/dL, while the LDL level actually drifted upward in the control group on placebo plus statin therapy. In the previously reported primary results of this landmark randomized clinical trial, alirocumab on top of background intensive statin therapy reduced the primary composite endpoint of death attributable to coronary heart disease, ischemic stroke, MI, or unstable angina requiring hospitalization by 15%, compared with controls (N Engl J Med. 2018 Nov 29;379[22]:2097-107).
During a median 2.8 years of prospective follow-up, there were 1,860 new MIs in study participants. A blinded clinical events committee evaluated the myocardial infarctions according to the Third Universal Definition and determined 66% were type 1 MIs, 21% were type 2, and 13% were type 4, with lesser numbers of types 3 and 5 MI.
Alirocumab reduced the risk of any MI by 15%, with a 6.8% incidence during follow-up, compared with 7.9% on placebo. The risk of type 1 MI, typically attributable to plaque rupture, was reduced by 13%, with an incidence of 4.9% with alirocumab and 5.6% with placebo. The risk reduction conferred by the PCSK9 inhibitor was even more robust for type 2 MI, the type caused by an oxygen supply/demand imbalance most commonly attributable to coronary artery spasm, coronary embolism, arrhythmias, anemia, hypertension, or hypotension: a 23% relative risk reduction as reflected in a 1.3% incidence in the alirocumab group, compared with a 1.7% rate in controls.
In contrast, alirocumab had no impact on the incidence of type 4 MI, a category that includes peri–percutaneous coronary intervention MIs as well as those attributable to stent thrombosis or restenosis.
The beneficial effect of alirocumab on MI risk mostly involved a reduction in larger MIs – those with a biomarker peak greater than three times the upper limit of normal.
An emphatic difference was found in the risk of death following type 1 as opposed to type 2 MI. Patients who experienced a type 1 MI during the study had an 11.9% mortality rate during an average of 1.6 years of post-MI follow-up, as compared with a 25.4% rate during 1.3 years of follow-up after a type 2 MI.
Alirocumab significantly reduced the risk of mortality following a type 1 MI, with a 10.2% rate as compared to 13.4% with placebo; that’s a 31% relative risk reduction. Yet the PCSK9 inhibitor had no impact on the risk of death after a type 2 MI: 24.8% in the alirocumab group and 25.9% in controls.
Asked for his thoughts as to possible explanatory mechanistic pathways for the benefit of alirocumab in preventing type 2 MI, Dr. White noted that in a Scottish study of the PCSK9 inhibitor evolocumab (Repatha), over the course of 72 months the drug appeared to reduce atherosclerotic progression and induce plaque stabilization and perhaps even regression.
“I think that’s the probable mechanism. And we also know that statins improve endothelial function,” he said.
He reported receiving research grant support and consultant fees from Sanofi and Regeneron, funders of the ODYSSEY Outcomes trial.
REPORTING FROM ACC 19
Social media for physicians: Strong medicine or snake oil?
For most of us, social media is a daunting new reality that we are pressured to be part of but that we struggle to fit into our increasingly demanding schedules. My first social media foray as a physician was a Facebook fan page as a hobby rather than a professional presence. Years later, I have learned the incredible benefit that being on social media in other platforms brought to my profession.
What’s social media going to bring to my medical practice?
The days where physicians retreat to the safety of our offices to deliver our care, or to issue carefully structured opinions, or interactions with patients have made way for more direct interaction. Social media has, indeed, allowed us to share more personal glimpses of our daily struggle to save lives, behind-the-scenes snapshot of ethical struggles in decision making, our difficulties qualifying patients for therapies due to insurance complications, or real-time addressing medical news and combating misinformation. Moreover, when patients self-refer, or are referred to my practice, they look me up online before coming to my office. Online profiles are the new “first impression” of the bedside manner of a physician.
Other personal examples of social media benefits include being informed of new publications, since many journals now have an online presence; being able to interact in real-time with authors; learning from physicians in other countries how they handled issues, such as shortage of critical medications; or earning CME, such as the Twitter chats hosted by CHEST (eg, new biologic agents in difficult to treat asthma, or patient selection in triple therapy for COPD).
Why should I pay attention to social media presence?
The pace by which social media changed the landscape took the medical community by surprise. Patients, third-party websites, and online review agencies (official or not) adopted it well before physicians became comfortable with it. As such, when I decided to google myself online, I was shocked at the level of misinformation about me (as a pulmonologist, I didn’t know I had performed sigmoidoscopies, yet that’s what my patients learned before they met me). That was an important lesson: If I don’t control the narrative, someone else will. Consequently, I dedicated a few hours to establish an online presence in order to introduce myself accurately and to be accessible to my patients and colleagues online.
Who decides what’s ethical and what’s not?
As the lines blurred, our community struggled to define what was appropriate and what was not. Finally, we welcomed with relief the issuance of a Code of Ethics, regarding social media use by physicians, from several societies, including the American Medical Association (https://www.ama-assn.org/delivering-care/ethics/professionalism-use-social-media). The principles guiding physicians use of social media include respect for human dignity and rights, honesty and upholding the standards of professionalism, and the duty to safeguard patient confidences and privacy.
Which platform should I use? There are so many.
While any content can be shared on any platform, social media sites have organically differentiated into being more amenable to one content vs the other. Some accounts tend to be more for professional use (ie, Twitter and LinkedIn), and other accounts for personal use (ie, Facebook, Instagram, Snapchat, and Pinterest). CHEST has selected Twitter to host its CME chats regarding preselected topics, post information about an upcoming lecture during the CHEST meeting, etc. New social media sites are now “physician only,” such as Sermo, Doximity, QuantiMD, and Doc2Doc. Many of these sites require doctors to submit their credentials to a site gatekeeper, recreating the intimacy of a “physicians’ lounge” in an online environment (J Med Internet Res. 2014:Feb 11;16[2]:e13). Lastly, Figure1 is a media sharing app between physicians allowing discussions of de-identified images or cases, recreating the “curbside” consult concept online.
I heard about hashtags. What are they?
Hashtags are simply clickable topic titles (#COPD #Sepsis # Education, etc.) that can be added to a post, in order to widen its reach. For instance, if I am interested in sepsis, I can click on the hashtag #Sepsis, and it would bring up all the posts on any Twitter account that added that hashtag. It’s a filter that takes me to that topic of interest. I can then click on the button “Like” on the message or the account itself where the post was found. The “Like” is similar to a bookmark for that account on my own Twitter. In the future, all the posts from that account would be available to me.
What are influencers or thought leaders?
Anyone who “liked” my account is now “following” me. The number of followers has become a measure of the popularity of anyone on social media. If it reaches a high level, then the person with the account is dubbed an “influencer.” Social media “influencers” are individuals whose opinion is followed by hundreds of thousands. Influencers may even be rewarded for harnessing their reach to make money off advertising. One can easily see how it is powerful for a physician to become an influencer or a “thought leader,” not to make money but to expand their reach on social media to spread the correct information about diets, drugs, e-cigarettes, and vaccinations, to name a few.
Can social media get me in trouble?
In 2012, a survey of the state medical boards published by JAMA (2012;307[11]:1141) revealed that approximately 30% of state medical boards reported complaints of “online violations of patient confidentiality.” More than 10% stated they had encountered a case of an “online depiction of intoxication.”
Another study a year earlier revealed that 13% of physicians reported they have discussed individual, though anonymized, cases with other physicians in public online forums (http://www.quantiamd.com/qqcp/DoctorsPatientSocialMedia.pdf).
Even if posted anonymously, or on a “personal” rather than professional social media site, various investigative methods may potentially be used to directly link information to a specific person or incident. The most current case law dictates that such information is “discoverable.” In fact, Facebook’s policy for the use of data informs users that, “we may access, preserve, and share your information in response to a legal request” both within and outside of U.S. jurisdiction”.
What kind of trouble could I be exposed to?
Poor quality of information, damage to our professional image, breaches of patient’s privacy, violation of patient-physician boundary, license revoking by state boards, and erroneous medical advice given in the absence of examining a patient, are all potential pitfalls for physicians in the careless use of social media.
How can I minimize my legal risk when interacting online?
It has been suggested that a legally sound approach in response to requests for online medical advice would be to send a standard response form that:
• informs the inquirer that the health-care provider does not answer online questions;
• supplies offline contact information so that an appointment can be made, if desired; and
• identifies a source for emergency services if the inquirer cannot wait for an appointment.
In circumstances where a patient–physician relationship already exists, informed consent should be obtained, which should include a careful explanation regarding the risks of online communication, expected response times, and the handling of emergencies, then documented in the patient’s chart (PT. 2014 Jul;39[7]:491,520).
In Summary
Social media, much like any area of medicine one is interested in, can be daunting and exciting but fraught with potential difficulties. I liken its adoption in our daily practice to any other decision or interest, including being in a private or academic setting, adopting procedural medicine or sticking to diagnostic consultations, or participating in research. In the end, it’s an individual expression of our desire to practice medicine. However, verifying information already existing online about us is of paramount importance. If I don’t tell my story, someone else will, and they may not be as truthful.
Dr. Bencheqroun is Assistant Professor, University of California Riverside School of Medicine, Pulmonary/Critical Care Faculty Program Coordinator & Research Mentor - Internal Medicine Residency Program Desert Regional Medical Center, Palm Springs CA; and Immediate Past Chair of the CHEST Council of Networks.
For most of us, social media is a daunting new reality that we are pressured to be part of but that we struggle to fit into our increasingly demanding schedules. My first social media foray as a physician was a Facebook fan page as a hobby rather than a professional presence. Years later, I have learned the incredible benefit that being on social media in other platforms brought to my profession.
What’s social media going to bring to my medical practice?
The days where physicians retreat to the safety of our offices to deliver our care, or to issue carefully structured opinions, or interactions with patients have made way for more direct interaction. Social media has, indeed, allowed us to share more personal glimpses of our daily struggle to save lives, behind-the-scenes snapshot of ethical struggles in decision making, our difficulties qualifying patients for therapies due to insurance complications, or real-time addressing medical news and combating misinformation. Moreover, when patients self-refer, or are referred to my practice, they look me up online before coming to my office. Online profiles are the new “first impression” of the bedside manner of a physician.
Other personal examples of social media benefits include being informed of new publications, since many journals now have an online presence; being able to interact in real-time with authors; learning from physicians in other countries how they handled issues, such as shortage of critical medications; or earning CME, such as the Twitter chats hosted by CHEST (eg, new biologic agents in difficult to treat asthma, or patient selection in triple therapy for COPD).
Why should I pay attention to social media presence?
The pace by which social media changed the landscape took the medical community by surprise. Patients, third-party websites, and online review agencies (official or not) adopted it well before physicians became comfortable with it. As such, when I decided to google myself online, I was shocked at the level of misinformation about me (as a pulmonologist, I didn’t know I had performed sigmoidoscopies, yet that’s what my patients learned before they met me). That was an important lesson: If I don’t control the narrative, someone else will. Consequently, I dedicated a few hours to establish an online presence in order to introduce myself accurately and to be accessible to my patients and colleagues online.
Who decides what’s ethical and what’s not?
As the lines blurred, our community struggled to define what was appropriate and what was not. Finally, we welcomed with relief the issuance of a Code of Ethics, regarding social media use by physicians, from several societies, including the American Medical Association (https://www.ama-assn.org/delivering-care/ethics/professionalism-use-social-media). The principles guiding physicians use of social media include respect for human dignity and rights, honesty and upholding the standards of professionalism, and the duty to safeguard patient confidences and privacy.
Which platform should I use? There are so many.
While any content can be shared on any platform, social media sites have organically differentiated into being more amenable to one content vs the other. Some accounts tend to be more for professional use (ie, Twitter and LinkedIn), and other accounts for personal use (ie, Facebook, Instagram, Snapchat, and Pinterest). CHEST has selected Twitter to host its CME chats regarding preselected topics, post information about an upcoming lecture during the CHEST meeting, etc. New social media sites are now “physician only,” such as Sermo, Doximity, QuantiMD, and Doc2Doc. Many of these sites require doctors to submit their credentials to a site gatekeeper, recreating the intimacy of a “physicians’ lounge” in an online environment (J Med Internet Res. 2014:Feb 11;16[2]:e13). Lastly, Figure1 is a media sharing app between physicians allowing discussions of de-identified images or cases, recreating the “curbside” consult concept online.
I heard about hashtags. What are they?
Hashtags are simply clickable topic titles (#COPD #Sepsis # Education, etc.) that can be added to a post, in order to widen its reach. For instance, if I am interested in sepsis, I can click on the hashtag #Sepsis, and it would bring up all the posts on any Twitter account that added that hashtag. It’s a filter that takes me to that topic of interest. I can then click on the button “Like” on the message or the account itself where the post was found. The “Like” is similar to a bookmark for that account on my own Twitter. In the future, all the posts from that account would be available to me.
What are influencers or thought leaders?
Anyone who “liked” my account is now “following” me. The number of followers has become a measure of the popularity of anyone on social media. If it reaches a high level, then the person with the account is dubbed an “influencer.” Social media “influencers” are individuals whose opinion is followed by hundreds of thousands. Influencers may even be rewarded for harnessing their reach to make money off advertising. One can easily see how it is powerful for a physician to become an influencer or a “thought leader,” not to make money but to expand their reach on social media to spread the correct information about diets, drugs, e-cigarettes, and vaccinations, to name a few.
Can social media get me in trouble?
In 2012, a survey of the state medical boards published by JAMA (2012;307[11]:1141) revealed that approximately 30% of state medical boards reported complaints of “online violations of patient confidentiality.” More than 10% stated they had encountered a case of an “online depiction of intoxication.”
Another study a year earlier revealed that 13% of physicians reported they have discussed individual, though anonymized, cases with other physicians in public online forums (http://www.quantiamd.com/qqcp/DoctorsPatientSocialMedia.pdf).
Even if posted anonymously, or on a “personal” rather than professional social media site, various investigative methods may potentially be used to directly link information to a specific person or incident. The most current case law dictates that such information is “discoverable.” In fact, Facebook’s policy for the use of data informs users that, “we may access, preserve, and share your information in response to a legal request” both within and outside of U.S. jurisdiction”.
What kind of trouble could I be exposed to?
Poor quality of information, damage to our professional image, breaches of patient’s privacy, violation of patient-physician boundary, license revoking by state boards, and erroneous medical advice given in the absence of examining a patient, are all potential pitfalls for physicians in the careless use of social media.
How can I minimize my legal risk when interacting online?
It has been suggested that a legally sound approach in response to requests for online medical advice would be to send a standard response form that:
• informs the inquirer that the health-care provider does not answer online questions;
• supplies offline contact information so that an appointment can be made, if desired; and
• identifies a source for emergency services if the inquirer cannot wait for an appointment.
In circumstances where a patient–physician relationship already exists, informed consent should be obtained, which should include a careful explanation regarding the risks of online communication, expected response times, and the handling of emergencies, then documented in the patient’s chart (PT. 2014 Jul;39[7]:491,520).
In Summary
Social media, much like any area of medicine one is interested in, can be daunting and exciting but fraught with potential difficulties. I liken its adoption in our daily practice to any other decision or interest, including being in a private or academic setting, adopting procedural medicine or sticking to diagnostic consultations, or participating in research. In the end, it’s an individual expression of our desire to practice medicine. However, verifying information already existing online about us is of paramount importance. If I don’t tell my story, someone else will, and they may not be as truthful.
Dr. Bencheqroun is Assistant Professor, University of California Riverside School of Medicine, Pulmonary/Critical Care Faculty Program Coordinator & Research Mentor - Internal Medicine Residency Program Desert Regional Medical Center, Palm Springs CA; and Immediate Past Chair of the CHEST Council of Networks.
For most of us, social media is a daunting new reality that we are pressured to be part of but that we struggle to fit into our increasingly demanding schedules. My first social media foray as a physician was a Facebook fan page as a hobby rather than a professional presence. Years later, I have learned the incredible benefit that being on social media in other platforms brought to my profession.
What’s social media going to bring to my medical practice?
The days where physicians retreat to the safety of our offices to deliver our care, or to issue carefully structured opinions, or interactions with patients have made way for more direct interaction. Social media has, indeed, allowed us to share more personal glimpses of our daily struggle to save lives, behind-the-scenes snapshot of ethical struggles in decision making, our difficulties qualifying patients for therapies due to insurance complications, or real-time addressing medical news and combating misinformation. Moreover, when patients self-refer, or are referred to my practice, they look me up online before coming to my office. Online profiles are the new “first impression” of the bedside manner of a physician.
Other personal examples of social media benefits include being informed of new publications, since many journals now have an online presence; being able to interact in real-time with authors; learning from physicians in other countries how they handled issues, such as shortage of critical medications; or earning CME, such as the Twitter chats hosted by CHEST (eg, new biologic agents in difficult to treat asthma, or patient selection in triple therapy for COPD).
Why should I pay attention to social media presence?
The pace by which social media changed the landscape took the medical community by surprise. Patients, third-party websites, and online review agencies (official or not) adopted it well before physicians became comfortable with it. As such, when I decided to google myself online, I was shocked at the level of misinformation about me (as a pulmonologist, I didn’t know I had performed sigmoidoscopies, yet that’s what my patients learned before they met me). That was an important lesson: If I don’t control the narrative, someone else will. Consequently, I dedicated a few hours to establish an online presence in order to introduce myself accurately and to be accessible to my patients and colleagues online.
Who decides what’s ethical and what’s not?
As the lines blurred, our community struggled to define what was appropriate and what was not. Finally, we welcomed with relief the issuance of a Code of Ethics, regarding social media use by physicians, from several societies, including the American Medical Association (https://www.ama-assn.org/delivering-care/ethics/professionalism-use-social-media). The principles guiding physicians use of social media include respect for human dignity and rights, honesty and upholding the standards of professionalism, and the duty to safeguard patient confidences and privacy.
Which platform should I use? There are so many.
While any content can be shared on any platform, social media sites have organically differentiated into being more amenable to one content vs the other. Some accounts tend to be more for professional use (ie, Twitter and LinkedIn), and other accounts for personal use (ie, Facebook, Instagram, Snapchat, and Pinterest). CHEST has selected Twitter to host its CME chats regarding preselected topics, post information about an upcoming lecture during the CHEST meeting, etc. New social media sites are now “physician only,” such as Sermo, Doximity, QuantiMD, and Doc2Doc. Many of these sites require doctors to submit their credentials to a site gatekeeper, recreating the intimacy of a “physicians’ lounge” in an online environment (J Med Internet Res. 2014:Feb 11;16[2]:e13). Lastly, Figure1 is a media sharing app between physicians allowing discussions of de-identified images or cases, recreating the “curbside” consult concept online.
I heard about hashtags. What are they?
Hashtags are simply clickable topic titles (#COPD #Sepsis # Education, etc.) that can be added to a post, in order to widen its reach. For instance, if I am interested in sepsis, I can click on the hashtag #Sepsis, and it would bring up all the posts on any Twitter account that added that hashtag. It’s a filter that takes me to that topic of interest. I can then click on the button “Like” on the message or the account itself where the post was found. The “Like” is similar to a bookmark for that account on my own Twitter. In the future, all the posts from that account would be available to me.
What are influencers or thought leaders?
Anyone who “liked” my account is now “following” me. The number of followers has become a measure of the popularity of anyone on social media. If it reaches a high level, then the person with the account is dubbed an “influencer.” Social media “influencers” are individuals whose opinion is followed by hundreds of thousands. Influencers may even be rewarded for harnessing their reach to make money off advertising. One can easily see how it is powerful for a physician to become an influencer or a “thought leader,” not to make money but to expand their reach on social media to spread the correct information about diets, drugs, e-cigarettes, and vaccinations, to name a few.
Can social media get me in trouble?
In 2012, a survey of the state medical boards published by JAMA (2012;307[11]:1141) revealed that approximately 30% of state medical boards reported complaints of “online violations of patient confidentiality.” More than 10% stated they had encountered a case of an “online depiction of intoxication.”
Another study a year earlier revealed that 13% of physicians reported they have discussed individual, though anonymized, cases with other physicians in public online forums (http://www.quantiamd.com/qqcp/DoctorsPatientSocialMedia.pdf).
Even if posted anonymously, or on a “personal” rather than professional social media site, various investigative methods may potentially be used to directly link information to a specific person or incident. The most current case law dictates that such information is “discoverable.” In fact, Facebook’s policy for the use of data informs users that, “we may access, preserve, and share your information in response to a legal request” both within and outside of U.S. jurisdiction”.
What kind of trouble could I be exposed to?
Poor quality of information, damage to our professional image, breaches of patient’s privacy, violation of patient-physician boundary, license revoking by state boards, and erroneous medical advice given in the absence of examining a patient, are all potential pitfalls for physicians in the careless use of social media.
How can I minimize my legal risk when interacting online?
It has been suggested that a legally sound approach in response to requests for online medical advice would be to send a standard response form that:
• informs the inquirer that the health-care provider does not answer online questions;
• supplies offline contact information so that an appointment can be made, if desired; and
• identifies a source for emergency services if the inquirer cannot wait for an appointment.
In circumstances where a patient–physician relationship already exists, informed consent should be obtained, which should include a careful explanation regarding the risks of online communication, expected response times, and the handling of emergencies, then documented in the patient’s chart (PT. 2014 Jul;39[7]:491,520).
In Summary
Social media, much like any area of medicine one is interested in, can be daunting and exciting but fraught with potential difficulties. I liken its adoption in our daily practice to any other decision or interest, including being in a private or academic setting, adopting procedural medicine or sticking to diagnostic consultations, or participating in research. In the end, it’s an individual expression of our desire to practice medicine. However, verifying information already existing online about us is of paramount importance. If I don’t tell my story, someone else will, and they may not be as truthful.
Dr. Bencheqroun is Assistant Professor, University of California Riverside School of Medicine, Pulmonary/Critical Care Faculty Program Coordinator & Research Mentor - Internal Medicine Residency Program Desert Regional Medical Center, Palm Springs CA; and Immediate Past Chair of the CHEST Council of Networks.
This month in the journal CHEST®
Editor’s Picks
Richard S. Irwin, MD, Master FCCP
Giants in Chest Medicine
David C. Zavala, MD, FCCP
Original Research
Accuracy of Algorithms to Identify Pulmonary Arterial Hypertension in Administrative Data:
A Systematic Review. By K. R. Gillmeyer, et al.
Hypersensitivity Pneumonitis: Radiologic Phenotypes Are Associated With Distinct Survival
Time and Pulmonary Function Trajectory. By M. L. Salisbury, et al.
The Effects of Long-term CPAP on Weight Change in Patients With Comorbid OSA and
Cardiovascular Disease: Data From the SAVE Trial. By Q. Ou, et al, on behalf of the SAVE investigators.
Editor’s Picks
Richard S. Irwin, MD, Master FCCP
Giants in Chest Medicine
David C. Zavala, MD, FCCP
Original Research
Accuracy of Algorithms to Identify Pulmonary Arterial Hypertension in Administrative Data:
A Systematic Review. By K. R. Gillmeyer, et al.
Hypersensitivity Pneumonitis: Radiologic Phenotypes Are Associated With Distinct Survival
Time and Pulmonary Function Trajectory. By M. L. Salisbury, et al.
The Effects of Long-term CPAP on Weight Change in Patients With Comorbid OSA and
Cardiovascular Disease: Data From the SAVE Trial. By Q. Ou, et al, on behalf of the SAVE investigators.
Editor’s Picks
Richard S. Irwin, MD, Master FCCP
Giants in Chest Medicine
David C. Zavala, MD, FCCP
Original Research
Accuracy of Algorithms to Identify Pulmonary Arterial Hypertension in Administrative Data:
A Systematic Review. By K. R. Gillmeyer, et al.
Hypersensitivity Pneumonitis: Radiologic Phenotypes Are Associated With Distinct Survival
Time and Pulmonary Function Trajectory. By M. L. Salisbury, et al.
The Effects of Long-term CPAP on Weight Change in Patients With Comorbid OSA and
Cardiovascular Disease: Data From the SAVE Trial. By Q. Ou, et al, on behalf of the SAVE investigators.
Updates from your CHEST Board of Regents
In late January, your Board of Regents met for its first face-to-face quarterly meeting under the leadership of new President Clayton Cowl, MD, MS, FCCP. One of the most valuable aspects of serving on the Board is an opportunity to take an overall look at the direction of the organization. The Board makes a concerted effort not to get too deep into the weeds planning out specific tactics for achieving goals; we have a great many outstanding volunteers serving on dozens of our committees who do an incredible job of making things happen. The Board tries to focus on overall organizational strategy. Are we going in the right direction? Are there opportunities of which we should be taking better advantage? Are there efforts in which we are currently engaged that may not be yielding outcomes as we expected? To better answer these questions, Dr. Cowl and his team asked all members of the Board of Regents and the Strategic Planning Subcommittee members of the Foundation Board of Trustees, as well as senior CHEST staff, to engage in an environmental scan to take an aggressive look at where we are and where we are headed. The output from our first environmental scan is currently being curated into a list of highest priority items that will be shared with the general membership in the coming months.
A review of our accomplishments over the last 6 months came next. Our new Executive Vice President and Chief Operating Officer, Dr. Robert Musacchio, has superseded all expectations in his first few months in the role. In addition to continuing to push the organization toward the “One CHEST” model by better integrating the Foundation with the College, as well as refining our operating principles in working with industry, Bob is further developing our international reach—exploring collaborations with a number of large international societies and planning meetings abroad later this year (CHEST Congress Thailand and CHEST Regional Congress Athens) and into the next (in Italy, with the regional meeting location to be determined). We are also in the process of recruiting for a new position, Chief Learning Officer, a role that will serve not only to better organize the educational activities of CHEST, but to also serve as a visionary to better imagine what future projects we should be pursuing to be of better service and value to our members.
We took a few moments to recognize the new, incoming Editor in Chief of the journal CHEST®; Peter Mazzone, MD, FCCP, will have some huge shoes to fill in taking the editor’s chair from Richard Irwin, MD, Master FCCP, who has served the journal in this role for more than a decade. Under Dr. Irwin’s leadership, CHEST has been the most-read publication amongst practicing pulmonary specialists; he is also responsible for having launched CHEST’s social media presence, including both video series that integrated directly with the journal (such as Ultrasound Corner) and podcasts. Richard also spoke beautifully about his passion for patient-centered care as a keynote speaker at CHEST 2018. Peter has outlined a number of different areas of focus for the journal in the next year, including putting a high priority on improving the reader experience and crafting an even better web and multimedia presence. We look forward to great things from the journal!
Chris Carroll, MD, FCCP, who chairs CHEST’s Digital Strategy Task Force, presented to the Board on their progress to date. The goal of this group is to evaluate the user experience for CHEST’s content delivery platforms, including the website, apps, and our social media platforms to identify opportunities for improvements that will enable us to better provide our members with on demand, high quality information to improve patient care through a personalized, seamless digital user experience. The team is being co-led by Nicki Augustyn, Senior Vice President for Marketing, Communications, and Publishing, and Ron Moen, Chief Information Officer. We look forward to further updates on this important project.
As I stated in my opening, many of the good things that CHEST does can only happen with the participation of our great members, and so I want to take the time to recognize the NetWorks and everything that they do for the College. In the past year, under the leadership of Council of NetWorks Chairs Hassan Bencheqroun, MD, FCCP, and David Zielinski, MD, FCCP, the NetWorks produced more than 60% of the content at the 2018 CHEST meeting and are actively working on projects ranging from creating educational videos for public consumption to CHEST guidelines proposals and crafting a donor registry for lung transplantation. Our volunteer leaders are our most valuable resource; if you are not currently engaged in the NetWorks, please consider getting involved this spring during the nomination process!
It remains a privilege for the Board to serve this great organization. If you are interested in hearing more, or getting more engaged, please send me an email at chestphysiciannews@chestnet.org.
David A. Schulman, MD, FCCP
In late January, your Board of Regents met for its first face-to-face quarterly meeting under the leadership of new President Clayton Cowl, MD, MS, FCCP. One of the most valuable aspects of serving on the Board is an opportunity to take an overall look at the direction of the organization. The Board makes a concerted effort not to get too deep into the weeds planning out specific tactics for achieving goals; we have a great many outstanding volunteers serving on dozens of our committees who do an incredible job of making things happen. The Board tries to focus on overall organizational strategy. Are we going in the right direction? Are there opportunities of which we should be taking better advantage? Are there efforts in which we are currently engaged that may not be yielding outcomes as we expected? To better answer these questions, Dr. Cowl and his team asked all members of the Board of Regents and the Strategic Planning Subcommittee members of the Foundation Board of Trustees, as well as senior CHEST staff, to engage in an environmental scan to take an aggressive look at where we are and where we are headed. The output from our first environmental scan is currently being curated into a list of highest priority items that will be shared with the general membership in the coming months.
A review of our accomplishments over the last 6 months came next. Our new Executive Vice President and Chief Operating Officer, Dr. Robert Musacchio, has superseded all expectations in his first few months in the role. In addition to continuing to push the organization toward the “One CHEST” model by better integrating the Foundation with the College, as well as refining our operating principles in working with industry, Bob is further developing our international reach—exploring collaborations with a number of large international societies and planning meetings abroad later this year (CHEST Congress Thailand and CHEST Regional Congress Athens) and into the next (in Italy, with the regional meeting location to be determined). We are also in the process of recruiting for a new position, Chief Learning Officer, a role that will serve not only to better organize the educational activities of CHEST, but to also serve as a visionary to better imagine what future projects we should be pursuing to be of better service and value to our members.
We took a few moments to recognize the new, incoming Editor in Chief of the journal CHEST®; Peter Mazzone, MD, FCCP, will have some huge shoes to fill in taking the editor’s chair from Richard Irwin, MD, Master FCCP, who has served the journal in this role for more than a decade. Under Dr. Irwin’s leadership, CHEST has been the most-read publication amongst practicing pulmonary specialists; he is also responsible for having launched CHEST’s social media presence, including both video series that integrated directly with the journal (such as Ultrasound Corner) and podcasts. Richard also spoke beautifully about his passion for patient-centered care as a keynote speaker at CHEST 2018. Peter has outlined a number of different areas of focus for the journal in the next year, including putting a high priority on improving the reader experience and crafting an even better web and multimedia presence. We look forward to great things from the journal!
Chris Carroll, MD, FCCP, who chairs CHEST’s Digital Strategy Task Force, presented to the Board on their progress to date. The goal of this group is to evaluate the user experience for CHEST’s content delivery platforms, including the website, apps, and our social media platforms to identify opportunities for improvements that will enable us to better provide our members with on demand, high quality information to improve patient care through a personalized, seamless digital user experience. The team is being co-led by Nicki Augustyn, Senior Vice President for Marketing, Communications, and Publishing, and Ron Moen, Chief Information Officer. We look forward to further updates on this important project.
As I stated in my opening, many of the good things that CHEST does can only happen with the participation of our great members, and so I want to take the time to recognize the NetWorks and everything that they do for the College. In the past year, under the leadership of Council of NetWorks Chairs Hassan Bencheqroun, MD, FCCP, and David Zielinski, MD, FCCP, the NetWorks produced more than 60% of the content at the 2018 CHEST meeting and are actively working on projects ranging from creating educational videos for public consumption to CHEST guidelines proposals and crafting a donor registry for lung transplantation. Our volunteer leaders are our most valuable resource; if you are not currently engaged in the NetWorks, please consider getting involved this spring during the nomination process!
It remains a privilege for the Board to serve this great organization. If you are interested in hearing more, or getting more engaged, please send me an email at chestphysiciannews@chestnet.org.
David A. Schulman, MD, FCCP
In late January, your Board of Regents met for its first face-to-face quarterly meeting under the leadership of new President Clayton Cowl, MD, MS, FCCP. One of the most valuable aspects of serving on the Board is an opportunity to take an overall look at the direction of the organization. The Board makes a concerted effort not to get too deep into the weeds planning out specific tactics for achieving goals; we have a great many outstanding volunteers serving on dozens of our committees who do an incredible job of making things happen. The Board tries to focus on overall organizational strategy. Are we going in the right direction? Are there opportunities of which we should be taking better advantage? Are there efforts in which we are currently engaged that may not be yielding outcomes as we expected? To better answer these questions, Dr. Cowl and his team asked all members of the Board of Regents and the Strategic Planning Subcommittee members of the Foundation Board of Trustees, as well as senior CHEST staff, to engage in an environmental scan to take an aggressive look at where we are and where we are headed. The output from our first environmental scan is currently being curated into a list of highest priority items that will be shared with the general membership in the coming months.
A review of our accomplishments over the last 6 months came next. Our new Executive Vice President and Chief Operating Officer, Dr. Robert Musacchio, has superseded all expectations in his first few months in the role. In addition to continuing to push the organization toward the “One CHEST” model by better integrating the Foundation with the College, as well as refining our operating principles in working with industry, Bob is further developing our international reach—exploring collaborations with a number of large international societies and planning meetings abroad later this year (CHEST Congress Thailand and CHEST Regional Congress Athens) and into the next (in Italy, with the regional meeting location to be determined). We are also in the process of recruiting for a new position, Chief Learning Officer, a role that will serve not only to better organize the educational activities of CHEST, but to also serve as a visionary to better imagine what future projects we should be pursuing to be of better service and value to our members.
We took a few moments to recognize the new, incoming Editor in Chief of the journal CHEST®; Peter Mazzone, MD, FCCP, will have some huge shoes to fill in taking the editor’s chair from Richard Irwin, MD, Master FCCP, who has served the journal in this role for more than a decade. Under Dr. Irwin’s leadership, CHEST has been the most-read publication amongst practicing pulmonary specialists; he is also responsible for having launched CHEST’s social media presence, including both video series that integrated directly with the journal (such as Ultrasound Corner) and podcasts. Richard also spoke beautifully about his passion for patient-centered care as a keynote speaker at CHEST 2018. Peter has outlined a number of different areas of focus for the journal in the next year, including putting a high priority on improving the reader experience and crafting an even better web and multimedia presence. We look forward to great things from the journal!
Chris Carroll, MD, FCCP, who chairs CHEST’s Digital Strategy Task Force, presented to the Board on their progress to date. The goal of this group is to evaluate the user experience for CHEST’s content delivery platforms, including the website, apps, and our social media platforms to identify opportunities for improvements that will enable us to better provide our members with on demand, high quality information to improve patient care through a personalized, seamless digital user experience. The team is being co-led by Nicki Augustyn, Senior Vice President for Marketing, Communications, and Publishing, and Ron Moen, Chief Information Officer. We look forward to further updates on this important project.
As I stated in my opening, many of the good things that CHEST does can only happen with the participation of our great members, and so I want to take the time to recognize the NetWorks and everything that they do for the College. In the past year, under the leadership of Council of NetWorks Chairs Hassan Bencheqroun, MD, FCCP, and David Zielinski, MD, FCCP, the NetWorks produced more than 60% of the content at the 2018 CHEST meeting and are actively working on projects ranging from creating educational videos for public consumption to CHEST guidelines proposals and crafting a donor registry for lung transplantation. Our volunteer leaders are our most valuable resource; if you are not currently engaged in the NetWorks, please consider getting involved this spring during the nomination process!
It remains a privilege for the Board to serve this great organization. If you are interested in hearing more, or getting more engaged, please send me an email at chestphysiciannews@chestnet.org.
David A. Schulman, MD, FCCP