HT Staff

Image by Marquet Minor

Gene editing has revealed how dyskeratosis congenita (DC) impairs the formation of blood cells, according to research published in Stem Cell Reports.

The discovery has opened up new lines of investigation into how to treat DC, which is characterized by shortened telomeres.

“Lengthening telomeres seems like a logical way to help these patients, but it could possibly come with its own set of problems,” said study author Luis F.Z. Batista, PhD, of the Washington University School of Medicine in St. Louis, Missouri.

“We would worry about encouraging cancer formation, for example, as high levels of the protein that lengthens telomeres—telomerase—are commonly found with cancer. But if we could find a way to block the signaling pathways that short telomeres activate—that specifically lead to the problems in blood cell formation—it could allow these patients to continue making blood cells.”

With this in mind, Dr Batista and his colleagues used CRISPR to edit into human embryonic stem cells a pair of mutations associated with DC— DKC1_A353V and TERT_P704S. These cells reproduced the telomere-shortening defect seen in patients with DC.

With this model, the researchers showed how the telomere defect leads to the gradual loss of blood cell formation and how blocking the downstream effects of the defect can reverse this loss, leading to normal production of blood cells.

Blocking this signaling pathway did not lengthen telomeres or stop their shortening, but it allowed the manufacturing of different types of blood cells to continue.

The researchers also made a discovery that provides a distinction regarding the detrimental effect of short telomeres during early development. The team found the defect did not hinder primitive hematopoiesis, but it did impair definitive hematopoiesis.

“This was tremendously interesting from a developmental biology perspective as well as from a disease modeling perspective,” said study author Christopher M. Sturgeon, PhD, of the Washington University School of Medicine. “We now have a platform to really dig deeper into understanding the mechanisms behind some forms of bone marrow failure.”

The researchers implicated high levels of the protein p53 as one of the signals that leads to the drop in definitive hematopoiesis.

“P53 is thought of as a guardian of the genome,” Dr Batista noted. “Mutations that disable p53 are associated with different types of cancer. Because of this, we would not consider directly trying to block p53 in these patients.”

“But what this study provides is proof-of-concept that this pathway is involved in this response. So we now are looking for ways to block the pathway further downstream without necessarily blocking p53 directly.”

Drs Batista and Sturgeon recently received a grant from the Department of Defense to investigate the pathway. The pair believes the strategy used in this study could be relevant for other bone marrow failure syndromes as well, such as Fanconi anemia and aplastic anemia.

Image by Marquet Minor

Gene editing has revealed how dyskeratosis congenita (DC) impairs the formation of blood cells, according to research published in Stem Cell Reports.

The discovery has opened up new lines of investigation into how to treat DC, which is characterized by shortened telomeres.

“Lengthening telomeres seems like a logical way to help these patients, but it could possibly come with its own set of problems,” said study author Luis F.Z. Batista, PhD, of the Washington University School of Medicine in St. Louis, Missouri.

“We would worry about encouraging cancer formation, for example, as high levels of the protein that lengthens telomeres—telomerase—are commonly found with cancer. But if we could find a way to block the signaling pathways that short telomeres activate—that specifically lead to the problems in blood cell formation—it could allow these patients to continue making blood cells.”

With this in mind, Dr Batista and his colleagues used CRISPR to edit into human embryonic stem cells a pair of mutations associated with DC— DKC1_A353V and TERT_P704S. These cells reproduced the telomere-shortening defect seen in patients with DC.

With this model, the researchers showed how the telomere defect leads to the gradual loss of blood cell formation and how blocking the downstream effects of the defect can reverse this loss, leading to normal production of blood cells.

Blocking this signaling pathway did not lengthen telomeres or stop their shortening, but it allowed the manufacturing of different types of blood cells to continue.

The researchers also made a discovery that provides a distinction regarding the detrimental effect of short telomeres during early development. The team found the defect did not hinder primitive hematopoiesis, but it did impair definitive hematopoiesis.

“This was tremendously interesting from a developmental biology perspective as well as from a disease modeling perspective,” said study author Christopher M. Sturgeon, PhD, of the Washington University School of Medicine. “We now have a platform to really dig deeper into understanding the mechanisms behind some forms of bone marrow failure.”

The researchers implicated high levels of the protein p53 as one of the signals that leads to the drop in definitive hematopoiesis.

“P53 is thought of as a guardian of the genome,” Dr Batista noted. “Mutations that disable p53 are associated with different types of cancer. Because of this, we would not consider directly trying to block p53 in these patients.”

“But what this study provides is proof-of-concept that this pathway is involved in this response. So we now are looking for ways to block the pathway further downstream without necessarily blocking p53 directly.”

Drs Batista and Sturgeon recently received a grant from the Department of Defense to investigate the pathway. The pair believes the strategy used in this study could be relevant for other bone marrow failure syndromes as well, such as Fanconi anemia and aplastic anemia.

Image by Marquet Minor

Gene editing has revealed how dyskeratosis congenita (DC) impairs the formation of blood cells, according to research published in Stem Cell Reports.

The discovery has opened up new lines of investigation into how to treat DC, which is characterized by shortened telomeres.

“Lengthening telomeres seems like a logical way to help these patients, but it could possibly come with its own set of problems,” said study author Luis F.Z. Batista, PhD, of the Washington University School of Medicine in St. Louis, Missouri.

“We would worry about encouraging cancer formation, for example, as high levels of the protein that lengthens telomeres—telomerase—are commonly found with cancer. But if we could find a way to block the signaling pathways that short telomeres activate—that specifically lead to the problems in blood cell formation—it could allow these patients to continue making blood cells.”

With this in mind, Dr Batista and his colleagues used CRISPR to edit into human embryonic stem cells a pair of mutations associated with DC— DKC1_A353V and TERT_P704S. These cells reproduced the telomere-shortening defect seen in patients with DC.

With this model, the researchers showed how the telomere defect leads to the gradual loss of blood cell formation and how blocking the downstream effects of the defect can reverse this loss, leading to normal production of blood cells.

Blocking this signaling pathway did not lengthen telomeres or stop their shortening, but it allowed the manufacturing of different types of blood cells to continue.

The researchers also made a discovery that provides a distinction regarding the detrimental effect of short telomeres during early development. The team found the defect did not hinder primitive hematopoiesis, but it did impair definitive hematopoiesis.

“This was tremendously interesting from a developmental biology perspective as well as from a disease modeling perspective,” said study author Christopher M. Sturgeon, PhD, of the Washington University School of Medicine. “We now have a platform to really dig deeper into understanding the mechanisms behind some forms of bone marrow failure.”

The researchers implicated high levels of the protein p53 as one of the signals that leads to the drop in definitive hematopoiesis.

“P53 is thought of as a guardian of the genome,” Dr Batista noted. “Mutations that disable p53 are associated with different types of cancer. Because of this, we would not consider directly trying to block p53 in these patients.”

“But what this study provides is proof-of-concept that this pathway is involved in this response. So we now are looking for ways to block the pathway further downstream without necessarily blocking p53 directly.”

Drs Batista and Sturgeon recently received a grant from the Department of Defense to investigate the pathway. The pair believes the strategy used in this study could be relevant for other bone marrow failure syndromes as well, such as Fanconi anemia and aplastic anemia.

Photo by Aaron Logan

Researchers say they have developed a new approach to model human immune variation that overcomes the limitations of traditional mouse models.

With this approach, the team identified genetic markers that directly correlate with the outcome of inflammatory and malignant diseases in humans, including chronic lymphocytic leukemia and Burkitt lymphoma.

The findings suggest that accounting for immune diversity is critical to the success of predicting disease outcomes based on immune cell measurements.

The team reported these findings in Nature Communications.

Traditionally, researchers have relied on inbred mouse strains to gain insight into human diseases while reducing experimental noise.

“If you take a black, a brown, or a white mouse, each one will give you a different answer in the same assay,” said Klaus Ley, MD, of La Jolla Institute for Allergy and Immunology in California.

“For example, if you vaccinate them, their responses will be different, which creates a lot of experimental noise. However, when you think about patients, or even healthy people, we are all different.”

To mine those differences for information, the researchers embraced the experimental noise. Instead of analyzing a single inbred mouse strain, they turned to the hybrid mouse diversity panel (HDMP).

The HDMP is a panel of about 100 different inbred mouse strains that mirror the breadth of genetic and immunological diversity found in the human population.

The researchers studied the natural variation in the activation pattern of abdominal macrophages. Macrophages isolated from 83 different mouse strains from the HDMP were exposed to lipopolysaccharide (LPS), a major component of the outer wall of gram-negative bacteria.

“Fundamentally, when the immune system is confronted with gram-negative bacteria, it can deal with the situation in 2 ways—either it gets very angry and tries to kill the bacteria or it can wall them off in an attempt to live with it,” explained Dr Ley. “Both strategies carry a certain risk, but a long evolutionary history has ensured that mice and people can survive with either strategy.”

The LPS-induced reactions of the macrophages covered the whole spectrum—from very aggressive (LPS+) to very tolerant (LPS-), depending on the mouse strain.

Next, the researchers asked which genes were active during each type of response to identify gene signatures that correlated with LPS responsiveness.

The team then ran these gene signatures across various human gene expression data sets and discovered they strongly correlated with human disease outcomes.

For example, macrophages isolated from healthy joints were enriched in LPS-tolerant genes, whereas macrophages from rheumatoid arthritis patients were strongly skewed toward LPS-aggressive.

Since it was known that mice and people with the aggressive phenotype are better at fighting cancer, the researchers specifically asked whether the level of LPS responsiveness could predict tumor survival.

After analyzing data from 18,000 biopsies across 39 different tumor types, the team found the LPS+ gene signature strongly correlated with survival, while the LPS- signature correlated with cancer death.

The pattern was significant across many types of cancer, including chronic lymphocytic leukemia, Burkitt lymphoma, osteosarcoma, melanoma, and large-cell lung carcinoma.

Photo by Aaron Logan

Researchers say they have developed a new approach to model human immune variation that overcomes the limitations of traditional mouse models.

With this approach, the team identified genetic markers that directly correlate with the outcome of inflammatory and malignant diseases in humans, including chronic lymphocytic leukemia and Burkitt lymphoma.

The findings suggest that accounting for immune diversity is critical to the success of predicting disease outcomes based on immune cell measurements.

The team reported these findings in Nature Communications.

Traditionally, researchers have relied on inbred mouse strains to gain insight into human diseases while reducing experimental noise.

“If you take a black, a brown, or a white mouse, each one will give you a different answer in the same assay,” said Klaus Ley, MD, of La Jolla Institute for Allergy and Immunology in California.

“For example, if you vaccinate them, their responses will be different, which creates a lot of experimental noise. However, when you think about patients, or even healthy people, we are all different.”

To mine those differences for information, the researchers embraced the experimental noise. Instead of analyzing a single inbred mouse strain, they turned to the hybrid mouse diversity panel (HDMP).

The HDMP is a panel of about 100 different inbred mouse strains that mirror the breadth of genetic and immunological diversity found in the human population.

The researchers studied the natural variation in the activation pattern of abdominal macrophages. Macrophages isolated from 83 different mouse strains from the HDMP were exposed to lipopolysaccharide (LPS), a major component of the outer wall of gram-negative bacteria.

“Fundamentally, when the immune system is confronted with gram-negative bacteria, it can deal with the situation in 2 ways—either it gets very angry and tries to kill the bacteria or it can wall them off in an attempt to live with it,” explained Dr Ley. “Both strategies carry a certain risk, but a long evolutionary history has ensured that mice and people can survive with either strategy.”

The LPS-induced reactions of the macrophages covered the whole spectrum—from very aggressive (LPS+) to very tolerant (LPS-), depending on the mouse strain.

Next, the researchers asked which genes were active during each type of response to identify gene signatures that correlated with LPS responsiveness.

The team then ran these gene signatures across various human gene expression data sets and discovered they strongly correlated with human disease outcomes.

For example, macrophages isolated from healthy joints were enriched in LPS-tolerant genes, whereas macrophages from rheumatoid arthritis patients were strongly skewed toward LPS-aggressive.

Since it was known that mice and people with the aggressive phenotype are better at fighting cancer, the researchers specifically asked whether the level of LPS responsiveness could predict tumor survival.

After analyzing data from 18,000 biopsies across 39 different tumor types, the team found the LPS+ gene signature strongly correlated with survival, while the LPS- signature correlated with cancer death.

The pattern was significant across many types of cancer, including chronic lymphocytic leukemia, Burkitt lymphoma, osteosarcoma, melanoma, and large-cell lung carcinoma.

Photo by Aaron Logan

Researchers say they have developed a new approach to model human immune variation that overcomes the limitations of traditional mouse models.

With this approach, the team identified genetic markers that directly correlate with the outcome of inflammatory and malignant diseases in humans, including chronic lymphocytic leukemia and Burkitt lymphoma.

The findings suggest that accounting for immune diversity is critical to the success of predicting disease outcomes based on immune cell measurements.

The team reported these findings in Nature Communications.

Traditionally, researchers have relied on inbred mouse strains to gain insight into human diseases while reducing experimental noise.

“If you take a black, a brown, or a white mouse, each one will give you a different answer in the same assay,” said Klaus Ley, MD, of La Jolla Institute for Allergy and Immunology in California.

“For example, if you vaccinate them, their responses will be different, which creates a lot of experimental noise. However, when you think about patients, or even healthy people, we are all different.”

To mine those differences for information, the researchers embraced the experimental noise. Instead of analyzing a single inbred mouse strain, they turned to the hybrid mouse diversity panel (HDMP).

The HDMP is a panel of about 100 different inbred mouse strains that mirror the breadth of genetic and immunological diversity found in the human population.

The researchers studied the natural variation in the activation pattern of abdominal macrophages. Macrophages isolated from 83 different mouse strains from the HDMP were exposed to lipopolysaccharide (LPS), a major component of the outer wall of gram-negative bacteria.

“Fundamentally, when the immune system is confronted with gram-negative bacteria, it can deal with the situation in 2 ways—either it gets very angry and tries to kill the bacteria or it can wall them off in an attempt to live with it,” explained Dr Ley. “Both strategies carry a certain risk, but a long evolutionary history has ensured that mice and people can survive with either strategy.”

The LPS-induced reactions of the macrophages covered the whole spectrum—from very aggressive (LPS+) to very tolerant (LPS-), depending on the mouse strain.

Next, the researchers asked which genes were active during each type of response to identify gene signatures that correlated with LPS responsiveness.

The team then ran these gene signatures across various human gene expression data sets and discovered they strongly correlated with human disease outcomes.

For example, macrophages isolated from healthy joints were enriched in LPS-tolerant genes, whereas macrophages from rheumatoid arthritis patients were strongly skewed toward LPS-aggressive.

Since it was known that mice and people with the aggressive phenotype are better at fighting cancer, the researchers specifically asked whether the level of LPS responsiveness could predict tumor survival.

After analyzing data from 18,000 biopsies across 39 different tumor types, the team found the LPS+ gene signature strongly correlated with survival, while the LPS- signature correlated with cancer death.

The pattern was significant across many types of cancer, including chronic lymphocytic leukemia, Burkitt lymphoma, osteosarcoma, melanoma, and large-cell lung carcinoma.

Image by Joshua Stokes

A newly identified pathway plays a “fundamental” role in acute myeloid leukemia (AML), according to researchers.

The team discovered that AML cells have a secretory pathway that leads to the production and release of the immune receptor Tim-3 and its ligand galectin-9, both of which prevent natural killer (NK) and other cytotoxic cells from killing the AML cells.

Vadim Sumbayev, PhD, of the University of Kent in the UK, and his colleagues recounted these findings in EBioMedicine.

The researchers found that AML cells—but not healthy blood cells—express a receptor called latrophilin 1 (LPHN1). LPHN1 induces activation of PKCα, which triggers the translation and secretion of Tim-3 and galectin-9.

Soluble Tim-3 prevents the secretion of interleukin 2, which is required for the activation of NK cells and cytotoxic T cells. Galectin-9 impairs the AML-cell-killing ability of NK cells and other cytotoxic lymphocytes.

The researchers said their work revealed both biomarkers for AML diagnostics and potential targets for AML treatment.

“Targeting this pathway will crucially enhance patients’ own immune defenses, helping them to eliminate leukemia cells,” Dr Sumbayev said.

He added that his group’s discovery might be applied to the treatment of other cancers as well.

Image by Joshua Stokes

A newly identified pathway plays a “fundamental” role in acute myeloid leukemia (AML), according to researchers.

The team discovered that AML cells have a secretory pathway that leads to the production and release of the immune receptor Tim-3 and its ligand galectin-9, both of which prevent natural killer (NK) and other cytotoxic cells from killing the AML cells.

Vadim Sumbayev, PhD, of the University of Kent in the UK, and his colleagues recounted these findings in EBioMedicine.

The researchers found that AML cells—but not healthy blood cells—express a receptor called latrophilin 1 (LPHN1). LPHN1 induces activation of PKCα, which triggers the translation and secretion of Tim-3 and galectin-9.

Soluble Tim-3 prevents the secretion of interleukin 2, which is required for the activation of NK cells and cytotoxic T cells. Galectin-9 impairs the AML-cell-killing ability of NK cells and other cytotoxic lymphocytes.

The researchers said their work revealed both biomarkers for AML diagnostics and potential targets for AML treatment.

“Targeting this pathway will crucially enhance patients’ own immune defenses, helping them to eliminate leukemia cells,” Dr Sumbayev said.

He added that his group’s discovery might be applied to the treatment of other cancers as well.

Image by Joshua Stokes

A newly identified pathway plays a “fundamental” role in acute myeloid leukemia (AML), according to researchers.

The team discovered that AML cells have a secretory pathway that leads to the production and release of the immune receptor Tim-3 and its ligand galectin-9, both of which prevent natural killer (NK) and other cytotoxic cells from killing the AML cells.

Vadim Sumbayev, PhD, of the University of Kent in the UK, and his colleagues recounted these findings in EBioMedicine.

The researchers found that AML cells—but not healthy blood cells—express a receptor called latrophilin 1 (LPHN1). LPHN1 induces activation of PKCα, which triggers the translation and secretion of Tim-3 and galectin-9.

Soluble Tim-3 prevents the secretion of interleukin 2, which is required for the activation of NK cells and cytotoxic T cells. Galectin-9 impairs the AML-cell-killing ability of NK cells and other cytotoxic lymphocytes.

The researchers said their work revealed both biomarkers for AML diagnostics and potential targets for AML treatment.

“Targeting this pathway will crucially enhance patients’ own immune defenses, helping them to eliminate leukemia cells,” Dr Sumbayev said.

He added that his group’s discovery might be applied to the treatment of other cancers as well.

Photo by Elise Amendola

Researchers have reported that interventions designed to decrease the need for red blood cell (RBC) transfusions proved effective when implemented at an academic medical center, and they resulted in an annual cost savings of more than $1 million.

The interventions included educational tools, a “best practice advisory,” and changes to the computerized provider order entry system.

They enabled the medical center to reduce multi-unit transfusions by 67.1% and transfusions for patients with hemoglobin (Hb) values of at least 7 g/dL by 47.4%.

Ian Jenkins, MD, of University of California San Diego (UCSD) Health, and his colleagues reported these results in The Joint Commission Journal on Quality and Patient Safety.

This work began with a multidisciplinary team at UCSD Health reviewing the transfusion literature on clinical trials, meta-analyses, guidelines, and improvement efforts.

The team used the information gleaned from this review and implemented the following interventions in an attempt to reduce unnecessary RBC transfusions:

• Educational tools (handouts, a video, PowerPoint presentations, etc)
• Enhancements to the health system’s computerized provider order entry system (eg, changing default RBC dose from 2 units to 1 unit)
• A “best practice advisory” intended to reduce unnecessary blood product use and costs by using real-time clinical decision support, a process for providing information at the point of care to help inform decisions about a patient’s care.

To assess the impact of their interventions, the researchers evaluated data on most non-infant, inpatient RBC transfusions given at UCSD Health between January 1, 2014, and September 30, 2016.

They excluded units given to patients with gastrointestinal bleeding and units given within 12 hours of a surgical procedure. The team said they excluded these transfusions because of the higher probability of unstable blood volume or special circumstances that might justify off-protocol transfusion.

The researchers then calculated the rate of inpatient RBC units transfused per 1000 patient-days (without exclusions), the percentage of inpatient RBC units transfused for patients with Hb ≥ 7 g/dL, and the percentage of multi-unit RBC transfusions, divided into 3 periods:

• Pre-intervention or baseline (January 1, 2014–September 30, 2014)
• During the intervention (October 1, 2014–April 30, 2015)
• Post-intervention (May 1, 2015–September 30, 2016).

There were 36,386 RBC units administered during the study period, which was 464,424 patient days.

Multi-unit transfusions decreased from 59.9% at baseline to 41.7% during the intervention period and 19.7% during the post-intervention period (P<0.0001).

Transfusions in patients with Hb values ≥ 7 g/dL decreased from 72.3% at baseline to 57.8% during the intervention period and 38.0% during the post-intervention period (P<0.0001).

The total RBC transfusion rate (units per 1000 patient-days) decreased from 89.8 at baseline to 78.1 during the intervention period and 72.7 during the post-intervention period (P<0.0001).

The estimated savings, based on a cost of $250 per RBC unit, was about $1,050,750 per year.

Photo by Elise Amendola

Researchers have reported that interventions designed to decrease the need for red blood cell (RBC) transfusions proved effective when implemented at an academic medical center, and they resulted in an annual cost savings of more than $1 million.

The interventions included educational tools, a “best practice advisory,” and changes to the computerized provider order entry system.

They enabled the medical center to reduce multi-unit transfusions by 67.1% and transfusions for patients with hemoglobin (Hb) values of at least 7 g/dL by 47.4%.

Ian Jenkins, MD, of University of California San Diego (UCSD) Health, and his colleagues reported these results in The Joint Commission Journal on Quality and Patient Safety.

This work began with a multidisciplinary team at UCSD Health reviewing the transfusion literature on clinical trials, meta-analyses, guidelines, and improvement efforts.

The team used the information gleaned from this review and implemented the following interventions in an attempt to reduce unnecessary RBC transfusions:

• Educational tools (handouts, a video, PowerPoint presentations, etc)
• Enhancements to the health system’s computerized provider order entry system (eg, changing default RBC dose from 2 units to 1 unit)
• A “best practice advisory” intended to reduce unnecessary blood product use and costs by using real-time clinical decision support, a process for providing information at the point of care to help inform decisions about a patient’s care.

To assess the impact of their interventions, the researchers evaluated data on most non-infant, inpatient RBC transfusions given at UCSD Health between January 1, 2014, and September 30, 2016.

They excluded units given to patients with gastrointestinal bleeding and units given within 12 hours of a surgical procedure. The team said they excluded these transfusions because of the higher probability of unstable blood volume or special circumstances that might justify off-protocol transfusion.

The researchers then calculated the rate of inpatient RBC units transfused per 1000 patient-days (without exclusions), the percentage of inpatient RBC units transfused for patients with Hb ≥ 7 g/dL, and the percentage of multi-unit RBC transfusions, divided into 3 periods:

• Pre-intervention or baseline (January 1, 2014–September 30, 2014)
• During the intervention (October 1, 2014–April 30, 2015)
• Post-intervention (May 1, 2015–September 30, 2016).

There were 36,386 RBC units administered during the study period, which was 464,424 patient days.

Multi-unit transfusions decreased from 59.9% at baseline to 41.7% during the intervention period and 19.7% during the post-intervention period (P<0.0001).

Transfusions in patients with Hb values ≥ 7 g/dL decreased from 72.3% at baseline to 57.8% during the intervention period and 38.0% during the post-intervention period (P<0.0001).

The total RBC transfusion rate (units per 1000 patient-days) decreased from 89.8 at baseline to 78.1 during the intervention period and 72.7 during the post-intervention period (P<0.0001).

The estimated savings, based on a cost of $250 per RBC unit, was about $1,050,750 per year.

Photo by Elise Amendola

Researchers have reported that interventions designed to decrease the need for red blood cell (RBC) transfusions proved effective when implemented at an academic medical center, and they resulted in an annual cost savings of more than $1 million.

The interventions included educational tools, a “best practice advisory,” and changes to the computerized provider order entry system.

They enabled the medical center to reduce multi-unit transfusions by 67.1% and transfusions for patients with hemoglobin (Hb) values of at least 7 g/dL by 47.4%.

Ian Jenkins, MD, of University of California San Diego (UCSD) Health, and his colleagues reported these results in The Joint Commission Journal on Quality and Patient Safety.

This work began with a multidisciplinary team at UCSD Health reviewing the transfusion literature on clinical trials, meta-analyses, guidelines, and improvement efforts.

The team used the information gleaned from this review and implemented the following interventions in an attempt to reduce unnecessary RBC transfusions:

• Educational tools (handouts, a video, PowerPoint presentations, etc)
• Enhancements to the health system’s computerized provider order entry system (eg, changing default RBC dose from 2 units to 1 unit)
• A “best practice advisory” intended to reduce unnecessary blood product use and costs by using real-time clinical decision support, a process for providing information at the point of care to help inform decisions about a patient’s care.

To assess the impact of their interventions, the researchers evaluated data on most non-infant, inpatient RBC transfusions given at UCSD Health between January 1, 2014, and September 30, 2016.

They excluded units given to patients with gastrointestinal bleeding and units given within 12 hours of a surgical procedure. The team said they excluded these transfusions because of the higher probability of unstable blood volume or special circumstances that might justify off-protocol transfusion.

The researchers then calculated the rate of inpatient RBC units transfused per 1000 patient-days (without exclusions), the percentage of inpatient RBC units transfused for patients with Hb ≥ 7 g/dL, and the percentage of multi-unit RBC transfusions, divided into 3 periods:

• Pre-intervention or baseline (January 1, 2014–September 30, 2014)
• During the intervention (October 1, 2014–April 30, 2015)
• Post-intervention (May 1, 2015–September 30, 2016).

There were 36,386 RBC units administered during the study period, which was 464,424 patient days.

Multi-unit transfusions decreased from 59.9% at baseline to 41.7% during the intervention period and 19.7% during the post-intervention period (P<0.0001).

Transfusions in patients with Hb values ≥ 7 g/dL decreased from 72.3% at baseline to 57.8% during the intervention period and 38.0% during the post-intervention period (P<0.0001).

The total RBC transfusion rate (units per 1000 patient-days) decreased from 89.8 at baseline to 78.1 during the intervention period and 72.7 during the post-intervention period (P<0.0001).

The estimated savings, based on a cost of $250 per RBC unit, was about $1,050,750 per year.

Micrograph showing MM

A gene expression profiling (GEP) classifier can accurately identify high- and low-risk patients with multiple myeloma (MM), according to research published in Clinical Lymphoma, Myeloma and Leukemia.

The GEP classifier is known as SKY92, and researchers found they could accurately identify high-risk MM patients using SKY92 alone.

By combining SKY92 with the International Staging System (ISS), the team was able to identify low-risk MM patients as well.

“We have demonstrated the prognostic value of SKY92 not only as a marker of high risk, but also as a marker of low risk when combined with ISS,” said study author Erik H. van Beers, PhD, vice president of genomics at SkylineDx, the company that developed SKY92.

“Both validated markers may serve as the basis for the discovery of improved individualized therapies for patients with multiple myeloma.”

Dr van Beers and his colleagues compared 8 risk-assessment platforms to analyze gene expression data from 91 newly diagnosed MM patients. The patients were included in an independent dataset amassed by the Multiple Myeloma Research Foundation and the Multiple Myeloma Genomics Initiative (MMRF/MMGI).

The researchers used the GEP classifiers SKY92, UAMS70, UAMS80, IFM15, HM19, Cancer Testis Antigen, Centrosome Index, and Proliferation Index to identify high-risk patients.

Of the 8 classifiers, SKY92 identified the largest proportion (21%) of high-risk cases and also attained the highest Cox proportional hazard ratio (8.2) for overall survival (OS).

Additionally, SKY92 predicted 9 of the 13 (60%) deaths that occurred at 2 years and 16 of the 31 (52%) deaths at 5 years, a predictive rate that was higher than any of the other classifiers.

Dr van Beers and his colleagues also combined the SKY92 standard-risk classifier with the ISS to identify low-risk patients in the MMRF/MMGI cohort. This combination was recently identified as a marker for detecting low-risk MM.*

The SKY92/ISS marker identified 42% of patients as low risk, with their median OS not reached at 96 months. The low-risk classification was strongly supported by the achieved hazard ratio of 10.

“The MMRF/MMGI dataset was not part of our previous discovery*, which demonstrated that the combination of SKY92 and ISS identifies the lowest-risk patients with high accuracy and leaves the smallest proportion of patients identified as intermediate risk,” Dr van Beers said.  “Thus, the dataset remained available for independent validation—an important factor in the adoption of gene expression profiling tools.”

“We are pleased to have validated the SKY92/ISS low-risk marker by applying it to the well-characterized MMRF/MMGI cohort,” added study author Rafael Fonseca, MD, of the Mayo Clinic in Scottsdale, Arizona. “Our findings further strengthen the prognostic utility of the combination marker.”

*Kuiper R, van Duin M, van Vliet, MH, et al. Prediction of high- and low-risk multiple myeloma based on gene expression and the International Staging System. Blood. 2015;126(17):1996-2004.

Micrograph showing MM

A gene expression profiling (GEP) classifier can accurately identify high- and low-risk patients with multiple myeloma (MM), according to research published in Clinical Lymphoma, Myeloma and Leukemia.

The GEP classifier is known as SKY92, and researchers found they could accurately identify high-risk MM patients using SKY92 alone.

By combining SKY92 with the International Staging System (ISS), the team was able to identify low-risk MM patients as well.

“We have demonstrated the prognostic value of SKY92 not only as a marker of high risk, but also as a marker of low risk when combined with ISS,” said study author Erik H. van Beers, PhD, vice president of genomics at SkylineDx, the company that developed SKY92.

“Both validated markers may serve as the basis for the discovery of improved individualized therapies for patients with multiple myeloma.”

Dr van Beers and his colleagues compared 8 risk-assessment platforms to analyze gene expression data from 91 newly diagnosed MM patients. The patients were included in an independent dataset amassed by the Multiple Myeloma Research Foundation and the Multiple Myeloma Genomics Initiative (MMRF/MMGI).

The researchers used the GEP classifiers SKY92, UAMS70, UAMS80, IFM15, HM19, Cancer Testis Antigen, Centrosome Index, and Proliferation Index to identify high-risk patients.

Of the 8 classifiers, SKY92 identified the largest proportion (21%) of high-risk cases and also attained the highest Cox proportional hazard ratio (8.2) for overall survival (OS).

Additionally, SKY92 predicted 9 of the 13 (60%) deaths that occurred at 2 years and 16 of the 31 (52%) deaths at 5 years, a predictive rate that was higher than any of the other classifiers.

Dr van Beers and his colleagues also combined the SKY92 standard-risk classifier with the ISS to identify low-risk patients in the MMRF/MMGI cohort. This combination was recently identified as a marker for detecting low-risk MM.*

The SKY92/ISS marker identified 42% of patients as low risk, with their median OS not reached at 96 months. The low-risk classification was strongly supported by the achieved hazard ratio of 10.

“The MMRF/MMGI dataset was not part of our previous discovery*, which demonstrated that the combination of SKY92 and ISS identifies the lowest-risk patients with high accuracy and leaves the smallest proportion of patients identified as intermediate risk,” Dr van Beers said.  “Thus, the dataset remained available for independent validation—an important factor in the adoption of gene expression profiling tools.”

“We are pleased to have validated the SKY92/ISS low-risk marker by applying it to the well-characterized MMRF/MMGI cohort,” added study author Rafael Fonseca, MD, of the Mayo Clinic in Scottsdale, Arizona. “Our findings further strengthen the prognostic utility of the combination marker.”

*Kuiper R, van Duin M, van Vliet, MH, et al. Prediction of high- and low-risk multiple myeloma based on gene expression and the International Staging System. Blood. 2015;126(17):1996-2004.

Micrograph showing MM

A gene expression profiling (GEP) classifier can accurately identify high- and low-risk patients with multiple myeloma (MM), according to research published in Clinical Lymphoma, Myeloma and Leukemia.

The GEP classifier is known as SKY92, and researchers found they could accurately identify high-risk MM patients using SKY92 alone.

By combining SKY92 with the International Staging System (ISS), the team was able to identify low-risk MM patients as well.

“We have demonstrated the prognostic value of SKY92 not only as a marker of high risk, but also as a marker of low risk when combined with ISS,” said study author Erik H. van Beers, PhD, vice president of genomics at SkylineDx, the company that developed SKY92.

“Both validated markers may serve as the basis for the discovery of improved individualized therapies for patients with multiple myeloma.”

Dr van Beers and his colleagues compared 8 risk-assessment platforms to analyze gene expression data from 91 newly diagnosed MM patients. The patients were included in an independent dataset amassed by the Multiple Myeloma Research Foundation and the Multiple Myeloma Genomics Initiative (MMRF/MMGI).

The researchers used the GEP classifiers SKY92, UAMS70, UAMS80, IFM15, HM19, Cancer Testis Antigen, Centrosome Index, and Proliferation Index to identify high-risk patients.

Of the 8 classifiers, SKY92 identified the largest proportion (21%) of high-risk cases and also attained the highest Cox proportional hazard ratio (8.2) for overall survival (OS).

Additionally, SKY92 predicted 9 of the 13 (60%) deaths that occurred at 2 years and 16 of the 31 (52%) deaths at 5 years, a predictive rate that was higher than any of the other classifiers.

Dr van Beers and his colleagues also combined the SKY92 standard-risk classifier with the ISS to identify low-risk patients in the MMRF/MMGI cohort. This combination was recently identified as a marker for detecting low-risk MM.*

The SKY92/ISS marker identified 42% of patients as low risk, with their median OS not reached at 96 months. The low-risk classification was strongly supported by the achieved hazard ratio of 10.

“The MMRF/MMGI dataset was not part of our previous discovery*, which demonstrated that the combination of SKY92 and ISS identifies the lowest-risk patients with high accuracy and leaves the smallest proportion of patients identified as intermediate risk,” Dr van Beers said.  “Thus, the dataset remained available for independent validation—an important factor in the adoption of gene expression profiling tools.”

“We are pleased to have validated the SKY92/ISS low-risk marker by applying it to the well-characterized MMRF/MMGI cohort,” added study author Rafael Fonseca, MD, of the Mayo Clinic in Scottsdale, Arizona. “Our findings further strengthen the prognostic utility of the combination marker.”

*Kuiper R, van Duin M, van Vliet, MH, et al. Prediction of high- and low-risk multiple myeloma based on gene expression and the International Staging System. Blood. 2015;126(17):1996-2004.

Image by Erhabor Osaro

The US Food and Drug Administration (FDA) has granted fast track designation to caplacizumab, an anti-von Willebrand factor (vWF) nanobody being developed for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP).

The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.

Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the new drug application or biologics license application on a rolling basis as data become available.

Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.

About caplacizumab

Caplacizumab is a bivalent anti-vWF nanobody being developed by Ablynx. Caplacizumab works by blocking the interaction of ultra-large vWF multimers with platelets, having an immediate effect on platelet aggregation and the ensuing formation and accumulation of the micro-clots that cause the severe thrombocytopenia, tissue ischemia, and organ dysfunction that occurs in patients with aTTP.

Researchers evaluated the efficacy and safety of caplacizumab, given with standard care for aTTP, in the phase 2 TITAN trial.

The trial enrolled 75 patients with aTTP. They all received the standard of care—daily plasma exchange and immunosuppressive therapy. Thirty-six patients were randomized to receive caplacizumab as well, and 39 were randomized to placebo.

The study’s primary endpoint was time to response (platelet count normalization). Patients in the caplacizumab arm had a 39% reduction in the median time to response compared to patients in the placebo arm (P=0.005).

The rate of confirmed response was 86.1% (n=31) in the caplacizumab arm and 71.8% (n=28) in the placebo arm.

There were more relapses in the caplacizumab arm than the placebo arm—8 (22.2%) and 0, respectively. Relapse was defined as a TTP event occurring more than 30 days after the end of daily plasma exchange.

There were fewer exacerbations in the caplacizumab arm than the placebo arm—3 (8.3%) and 11 (28.2%), respectively. Exacerbation was defined as recurrent thrombocytopenia within 30 days of the end of daily plasma exchange that required re-initiation of daily exchange.

The rate of adverse events thought to be related to the study drug was 17% in the caplacizumab arm and 11% in the placebo arm. The rate of events that were possibly related was 54% and 8%, respectively.

A lower proportion of subjects in the caplacizumab arm experienced one or more major thromboembolic events or died, compared to the placebo arm—11.4% and 43.2%, respectively.

In addition, fewer caplacizumab-treated patients were refractory to treatment—5.7% vs 21.6%.

There were 2 deaths in the placebo arm, and both of those patients were refractory to treatment. There were no deaths reported in the caplacizumab arm.

Now, researchers are evaluating caplacizumab in the phase 3 HERCULES trial (NCT02553317). Results from this study are anticipated in the second half of 2017 are expected to support a planned biologics license application filing in the US in 2018.

Image by Erhabor Osaro

The US Food and Drug Administration (FDA) has granted fast track designation to caplacizumab, an anti-von Willebrand factor (vWF) nanobody being developed for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP).

The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.

Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the new drug application or biologics license application on a rolling basis as data become available.

Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.

About caplacizumab

Caplacizumab is a bivalent anti-vWF nanobody being developed by Ablynx. Caplacizumab works by blocking the interaction of ultra-large vWF multimers with platelets, having an immediate effect on platelet aggregation and the ensuing formation and accumulation of the micro-clots that cause the severe thrombocytopenia, tissue ischemia, and organ dysfunction that occurs in patients with aTTP.

Researchers evaluated the efficacy and safety of caplacizumab, given with standard care for aTTP, in the phase 2 TITAN trial.

The trial enrolled 75 patients with aTTP. They all received the standard of care—daily plasma exchange and immunosuppressive therapy. Thirty-six patients were randomized to receive caplacizumab as well, and 39 were randomized to placebo.

The study’s primary endpoint was time to response (platelet count normalization). Patients in the caplacizumab arm had a 39% reduction in the median time to response compared to patients in the placebo arm (P=0.005).

The rate of confirmed response was 86.1% (n=31) in the caplacizumab arm and 71.8% (n=28) in the placebo arm.

There were more relapses in the caplacizumab arm than the placebo arm—8 (22.2%) and 0, respectively. Relapse was defined as a TTP event occurring more than 30 days after the end of daily plasma exchange.

There were fewer exacerbations in the caplacizumab arm than the placebo arm—3 (8.3%) and 11 (28.2%), respectively. Exacerbation was defined as recurrent thrombocytopenia within 30 days of the end of daily plasma exchange that required re-initiation of daily exchange.

The rate of adverse events thought to be related to the study drug was 17% in the caplacizumab arm and 11% in the placebo arm. The rate of events that were possibly related was 54% and 8%, respectively.

A lower proportion of subjects in the caplacizumab arm experienced one or more major thromboembolic events or died, compared to the placebo arm—11.4% and 43.2%, respectively.

In addition, fewer caplacizumab-treated patients were refractory to treatment—5.7% vs 21.6%.

There were 2 deaths in the placebo arm, and both of those patients were refractory to treatment. There were no deaths reported in the caplacizumab arm.

Now, researchers are evaluating caplacizumab in the phase 3 HERCULES trial (NCT02553317). Results from this study are anticipated in the second half of 2017 are expected to support a planned biologics license application filing in the US in 2018.

Image by Erhabor Osaro

The US Food and Drug Administration (FDA) has granted fast track designation to caplacizumab, an anti-von Willebrand factor (vWF) nanobody being developed for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP).

The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.

Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the new drug application or biologics license application on a rolling basis as data become available.

Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.

About caplacizumab

Caplacizumab is a bivalent anti-vWF nanobody being developed by Ablynx. Caplacizumab works by blocking the interaction of ultra-large vWF multimers with platelets, having an immediate effect on platelet aggregation and the ensuing formation and accumulation of the micro-clots that cause the severe thrombocytopenia, tissue ischemia, and organ dysfunction that occurs in patients with aTTP.

Researchers evaluated the efficacy and safety of caplacizumab, given with standard care for aTTP, in the phase 2 TITAN trial.

The trial enrolled 75 patients with aTTP. They all received the standard of care—daily plasma exchange and immunosuppressive therapy. Thirty-six patients were randomized to receive caplacizumab as well, and 39 were randomized to placebo.

The study’s primary endpoint was time to response (platelet count normalization). Patients in the caplacizumab arm had a 39% reduction in the median time to response compared to patients in the placebo arm (P=0.005).

The rate of confirmed response was 86.1% (n=31) in the caplacizumab arm and 71.8% (n=28) in the placebo arm.

There were more relapses in the caplacizumab arm than the placebo arm—8 (22.2%) and 0, respectively. Relapse was defined as a TTP event occurring more than 30 days after the end of daily plasma exchange.

There were fewer exacerbations in the caplacizumab arm than the placebo arm—3 (8.3%) and 11 (28.2%), respectively. Exacerbation was defined as recurrent thrombocytopenia within 30 days of the end of daily plasma exchange that required re-initiation of daily exchange.

The rate of adverse events thought to be related to the study drug was 17% in the caplacizumab arm and 11% in the placebo arm. The rate of events that were possibly related was 54% and 8%, respectively.

A lower proportion of subjects in the caplacizumab arm experienced one or more major thromboembolic events or died, compared to the placebo arm—11.4% and 43.2%, respectively.

In addition, fewer caplacizumab-treated patients were refractory to treatment—5.7% vs 21.6%.

There were 2 deaths in the placebo arm, and both of those patients were refractory to treatment. There were no deaths reported in the caplacizumab arm.

Now, researchers are evaluating caplacizumab in the phase 3 HERCULES trial (NCT02553317). Results from this study are anticipated in the second half of 2017 are expected to support a planned biologics license application filing in the US in 2018.

Photo from Business Wire

Health Canada has issued a non-conditional marketing authorization for brentuximab vedotin (Adcetris).

This means the drug is now approved for use as consolidation after autologous stem cell transplant (ASCT) in patients with Hodgkin lymphoma (HL) who have an increased risk of relapse or progression.

Brentuximab vedotin previously received approval with conditions in Canada to treat HL patients who relapse after ASCT or HL patients who are not ASCT candidates and relapse after at least 2 multi-agent chemotherapy regimens.

Brentuximab vedotin also has conditional approval in Canada to treat patients with systemic anaplastic large-cell lymphoma who relapse after at least 1 multi-agent chemotherapy regimen.

Brentuximab vedotin is an antibody-drug conjugate consisting of an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E.

Brentuximab vedotin has marketing authorization in 67 countries for the treatment of relapsed or refractory HL and systemic anaplastic large-cell lymphoma.

Seattle Genetics and Takeda are jointly developing brentuximab vedotin. Seattle Genetics has US and Canadian commercialization rights, and Takeda has rights to commercialize the drug in the rest of the world.

AETHERA trial

Health Canada’s decision to extend the marketing authorization of brentuximab vedotin is based on results from the phase 3 AETHERA trial.

The trial was designed to compare brentuximab vedotin to placebo, both administered for up to 16 cycles (approximately 1 year) every 3 weeks following ASCT. Results from the trial were published in The Lancet in March 2015.

The study enrolled 329 HL patients at risk of relapse or progression—165 on the brentuximab vedotin arm and 164 on the placebo arm.

Patients were eligible for enrollment if they had a history of primary refractory HL, relapsed within a year of receiving frontline chemotherapy, and/or had disease outside of the lymph nodes at the time of pre-ASCT relapse.

Brentuximab vedotin conferred a significant increase in progression-free survival over placebo, with a hazard ratio of 0.57 (P=0.001). The median progression-free survival was 43 months for patients who received brentuximab vedotin and 24 months for those who received placebo.

The most common adverse events (≥20%), of any grade and regardless of causality, in the brentuximab vedotin arm were neutropenia (78%), peripheral sensory neuropathy (56%), thrombocytopenia (41%), anemia (27%), upper respiratory tract infection (26%), fatigue (24%), peripheral motor neuropathy (23%), nausea (22%), cough (21%), and diarrhea (20%).

The most common adverse events (≥20%), of any grade and regardless of causality, in the placebo arm were neutropenia (34%), upper respiratory tract infection (23%), and thrombocytopenia (20%).

In all, 67% of patients on the brentuximab vedotin arm experienced peripheral neuropathy. Of those patients, 85% had resolution (59%) or partial improvement (26%) in symptoms at the time of their last evaluation, with a median time to improvement of 23 weeks (range, 0.1-138).

Photo from Business Wire

Health Canada has issued a non-conditional marketing authorization for brentuximab vedotin (Adcetris).

This means the drug is now approved for use as consolidation after autologous stem cell transplant (ASCT) in patients with Hodgkin lymphoma (HL) who have an increased risk of relapse or progression.

Brentuximab vedotin previously received approval with conditions in Canada to treat HL patients who relapse after ASCT or HL patients who are not ASCT candidates and relapse after at least 2 multi-agent chemotherapy regimens.

Brentuximab vedotin also has conditional approval in Canada to treat patients with systemic anaplastic large-cell lymphoma who relapse after at least 1 multi-agent chemotherapy regimen.

Brentuximab vedotin is an antibody-drug conjugate consisting of an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E.

Brentuximab vedotin has marketing authorization in 67 countries for the treatment of relapsed or refractory HL and systemic anaplastic large-cell lymphoma.

Seattle Genetics and Takeda are jointly developing brentuximab vedotin. Seattle Genetics has US and Canadian commercialization rights, and Takeda has rights to commercialize the drug in the rest of the world.

AETHERA trial

Health Canada’s decision to extend the marketing authorization of brentuximab vedotin is based on results from the phase 3 AETHERA trial.

The trial was designed to compare brentuximab vedotin to placebo, both administered for up to 16 cycles (approximately 1 year) every 3 weeks following ASCT. Results from the trial were published in The Lancet in March 2015.

The study enrolled 329 HL patients at risk of relapse or progression—165 on the brentuximab vedotin arm and 164 on the placebo arm.

Patients were eligible for enrollment if they had a history of primary refractory HL, relapsed within a year of receiving frontline chemotherapy, and/or had disease outside of the lymph nodes at the time of pre-ASCT relapse.

Brentuximab vedotin conferred a significant increase in progression-free survival over placebo, with a hazard ratio of 0.57 (P=0.001). The median progression-free survival was 43 months for patients who received brentuximab vedotin and 24 months for those who received placebo.

The most common adverse events (≥20%), of any grade and regardless of causality, in the brentuximab vedotin arm were neutropenia (78%), peripheral sensory neuropathy (56%), thrombocytopenia (41%), anemia (27%), upper respiratory tract infection (26%), fatigue (24%), peripheral motor neuropathy (23%), nausea (22%), cough (21%), and diarrhea (20%).

The most common adverse events (≥20%), of any grade and regardless of causality, in the placebo arm were neutropenia (34%), upper respiratory tract infection (23%), and thrombocytopenia (20%).

In all, 67% of patients on the brentuximab vedotin arm experienced peripheral neuropathy. Of those patients, 85% had resolution (59%) or partial improvement (26%) in symptoms at the time of their last evaluation, with a median time to improvement of 23 weeks (range, 0.1-138).

Photo from Business Wire

Health Canada has issued a non-conditional marketing authorization for brentuximab vedotin (Adcetris).

This means the drug is now approved for use as consolidation after autologous stem cell transplant (ASCT) in patients with Hodgkin lymphoma (HL) who have an increased risk of relapse or progression.

Brentuximab vedotin previously received approval with conditions in Canada to treat HL patients who relapse after ASCT or HL patients who are not ASCT candidates and relapse after at least 2 multi-agent chemotherapy regimens.

Brentuximab vedotin also has conditional approval in Canada to treat patients with systemic anaplastic large-cell lymphoma who relapse after at least 1 multi-agent chemotherapy regimen.

Brentuximab vedotin is an antibody-drug conjugate consisting of an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E.

Brentuximab vedotin has marketing authorization in 67 countries for the treatment of relapsed or refractory HL and systemic anaplastic large-cell lymphoma.

Seattle Genetics and Takeda are jointly developing brentuximab vedotin. Seattle Genetics has US and Canadian commercialization rights, and Takeda has rights to commercialize the drug in the rest of the world.

AETHERA trial

Health Canada’s decision to extend the marketing authorization of brentuximab vedotin is based on results from the phase 3 AETHERA trial.

The trial was designed to compare brentuximab vedotin to placebo, both administered for up to 16 cycles (approximately 1 year) every 3 weeks following ASCT. Results from the trial were published in The Lancet in March 2015.

The study enrolled 329 HL patients at risk of relapse or progression—165 on the brentuximab vedotin arm and 164 on the placebo arm.

Patients were eligible for enrollment if they had a history of primary refractory HL, relapsed within a year of receiving frontline chemotherapy, and/or had disease outside of the lymph nodes at the time of pre-ASCT relapse.

Brentuximab vedotin conferred a significant increase in progression-free survival over placebo, with a hazard ratio of 0.57 (P=0.001). The median progression-free survival was 43 months for patients who received brentuximab vedotin and 24 months for those who received placebo.

The most common adverse events (≥20%), of any grade and regardless of causality, in the brentuximab vedotin arm were neutropenia (78%), peripheral sensory neuropathy (56%), thrombocytopenia (41%), anemia (27%), upper respiratory tract infection (26%), fatigue (24%), peripheral motor neuropathy (23%), nausea (22%), cough (21%), and diarrhea (20%).

The most common adverse events (≥20%), of any grade and regardless of causality, in the placebo arm were neutropenia (34%), upper respiratory tract infection (23%), and thrombocytopenia (20%).

In all, 67% of patients on the brentuximab vedotin arm experienced peripheral neuropathy. Of those patients, 85% had resolution (59%) or partial improvement (26%) in symptoms at the time of their last evaluation, with a median time to improvement of 23 weeks (range, 0.1-138).

Photo by Rhoda Baer

More Americans are viewing clinical trials in a positive light, according to a survey commissioned by Research!America and the Association of Clinical Research Organizations (ACRO).

The results of the survey, which included 1000 participants and was conducted this month, reveal a shift in public attitudes about clinical trials since a similar survey was commissioned in 2013.

Thirty-seven percent of respondents involved in the current survey said they would “very likely” enroll in a clinical trial if their doctor recommended it. This represents an 11% increase from 2013.

Eighty-four percent of current survey respondents said they are willing to share personal health information, assuming that appropriate privacy protections are in place, so researchers can better understand diseases and develop new ways to prevent, treat, and cure them. This is a 10% increase from 2013.

When asked how much they admire people who volunteer for clinical trials, 46% of current respondents said “a great deal,” which is a 9% increase from 2013.

“More and more Americans appear to recognize the value of clinical trials—a very positive sign—but stubborn barriers to participation remain in place,” said Mary Woolley, president and CEO of Research!America.

“Development of incentives to drive more discussions between patients and healthcare professionals about the importance of participating in trials could encourage both ill and healthy individuals to view this as a routine health behavior.”

“We are pleased to have joined with Research!America in commissioning this important survey and gratified that the public’s knowledge of and attitudes toward clinical trials have moved in positive directions since 2013,” said Doug Peddicord, executive director of ACRO.

“The option to participate in a clinical trial, when appropriate, should be a routine part of the healthcare encounter, and ACRO will continue to work with Research!America and others to spread that message to doctors and patients alike.”

Survey results

The survey of 1000 US adults was conducted by Zogby Analytics for Research!America and ACRO in July 2017. The margin of error is +/- 3.1 percentage points.

Ninety percent of survey respondents said clinical trials are important to advancing science, and 87% said trials are important to improving the nation’s health. Seventy-five percent said taking part in clinical trials is as valuable to the US healthcare system as giving blood.

Eighty-six percent of respondents said healthcare professionals should discuss clinical trials with patients diagnosed with a disease as part of their standard care.

And 44% of respondents said clinical trial participation should be a routine health behavior, whether a person is healthy or ill, similar to getting an annual checkup with a healthcare provider.

Men (48%) were significantly more likely than women (39%) to say trial participation should be routine. A larger percentage of 18- to 29-year-olds (53%) and 30- to 49-year olds (48%) were in favor of routine trial participation, compared to people ages 50 to 64 (38%) and those 65 and older (34%).

Eighty percent of respondents said they had heard of a clinical trial, and 18% said they or someone in their family had participated in one.

Seventy-four percent of respondents said they would participate in a clinical trial if asked by someone they trust.

However, respondents were split on whether it’s important for everyone to take part in a clinical trial if asked. Forty-four percent said it is important, 45% disagreed, and 12% were not sure.

More than half of respondents (55%) said people don’t participate in clinical trials because of lack of awareness and information, 43% said it’s because trials are viewed as “too risky,” 41% said it’s due to a lack of information about the process, 38% said it’s due to a lack of trust, and 34% said it’s due to the risk of adverse health effects.

Nearly two-thirds of respondents (64%) said a doctor or healthcare provider is a reliable source for clinical trial information.

Respondents said doctors and other healthcare providers (44%), followed by the government (23%), have the greatest responsibility in educating the public about clinical trials. However, 74% said no healthcare professional has ever talked to them about medical research.

Seventy-two percent of respondents said they would be likely to use technology such as apps and monitoring devices to share their personal health data for clinical research.

Eighty-eight percent said clinical trial participants should have access to trial results. And 47% said they would like having clinical trial information/data/results delivered through their phone.

Photo by Rhoda Baer

More Americans are viewing clinical trials in a positive light, according to a survey commissioned by Research!America and the Association of Clinical Research Organizations (ACRO).

The results of the survey, which included 1000 participants and was conducted this month, reveal a shift in public attitudes about clinical trials since a similar survey was commissioned in 2013.

Thirty-seven percent of respondents involved in the current survey said they would “very likely” enroll in a clinical trial if their doctor recommended it. This represents an 11% increase from 2013.

Eighty-four percent of current survey respondents said they are willing to share personal health information, assuming that appropriate privacy protections are in place, so researchers can better understand diseases and develop new ways to prevent, treat, and cure them. This is a 10% increase from 2013.

When asked how much they admire people who volunteer for clinical trials, 46% of current respondents said “a great deal,” which is a 9% increase from 2013.

“More and more Americans appear to recognize the value of clinical trials—a very positive sign—but stubborn barriers to participation remain in place,” said Mary Woolley, president and CEO of Research!America.

“Development of incentives to drive more discussions between patients and healthcare professionals about the importance of participating in trials could encourage both ill and healthy individuals to view this as a routine health behavior.”

“We are pleased to have joined with Research!America in commissioning this important survey and gratified that the public’s knowledge of and attitudes toward clinical trials have moved in positive directions since 2013,” said Doug Peddicord, executive director of ACRO.

“The option to participate in a clinical trial, when appropriate, should be a routine part of the healthcare encounter, and ACRO will continue to work with Research!America and others to spread that message to doctors and patients alike.”

Survey results

The survey of 1000 US adults was conducted by Zogby Analytics for Research!America and ACRO in July 2017. The margin of error is +/- 3.1 percentage points.

Ninety percent of survey respondents said clinical trials are important to advancing science, and 87% said trials are important to improving the nation’s health. Seventy-five percent said taking part in clinical trials is as valuable to the US healthcare system as giving blood.

Eighty-six percent of respondents said healthcare professionals should discuss clinical trials with patients diagnosed with a disease as part of their standard care.

And 44% of respondents said clinical trial participation should be a routine health behavior, whether a person is healthy or ill, similar to getting an annual checkup with a healthcare provider.

Men (48%) were significantly more likely than women (39%) to say trial participation should be routine. A larger percentage of 18- to 29-year-olds (53%) and 30- to 49-year olds (48%) were in favor of routine trial participation, compared to people ages 50 to 64 (38%) and those 65 and older (34%).

Eighty percent of respondents said they had heard of a clinical trial, and 18% said they or someone in their family had participated in one.

Seventy-four percent of respondents said they would participate in a clinical trial if asked by someone they trust.

However, respondents were split on whether it’s important for everyone to take part in a clinical trial if asked. Forty-four percent said it is important, 45% disagreed, and 12% were not sure.

More than half of respondents (55%) said people don’t participate in clinical trials because of lack of awareness and information, 43% said it’s because trials are viewed as “too risky,” 41% said it’s due to a lack of information about the process, 38% said it’s due to a lack of trust, and 34% said it’s due to the risk of adverse health effects.

Nearly two-thirds of respondents (64%) said a doctor or healthcare provider is a reliable source for clinical trial information.

Respondents said doctors and other healthcare providers (44%), followed by the government (23%), have the greatest responsibility in educating the public about clinical trials. However, 74% said no healthcare professional has ever talked to them about medical research.

Seventy-two percent of respondents said they would be likely to use technology such as apps and monitoring devices to share their personal health data for clinical research.

Eighty-eight percent said clinical trial participants should have access to trial results. And 47% said they would like having clinical trial information/data/results delivered through their phone.

Photo by Rhoda Baer

More Americans are viewing clinical trials in a positive light, according to a survey commissioned by Research!America and the Association of Clinical Research Organizations (ACRO).

The results of the survey, which included 1000 participants and was conducted this month, reveal a shift in public attitudes about clinical trials since a similar survey was commissioned in 2013.

Thirty-seven percent of respondents involved in the current survey said they would “very likely” enroll in a clinical trial if their doctor recommended it. This represents an 11% increase from 2013.

Eighty-four percent of current survey respondents said they are willing to share personal health information, assuming that appropriate privacy protections are in place, so researchers can better understand diseases and develop new ways to prevent, treat, and cure them. This is a 10% increase from 2013.

When asked how much they admire people who volunteer for clinical trials, 46% of current respondents said “a great deal,” which is a 9% increase from 2013.

“More and more Americans appear to recognize the value of clinical trials—a very positive sign—but stubborn barriers to participation remain in place,” said Mary Woolley, president and CEO of Research!America.

“Development of incentives to drive more discussions between patients and healthcare professionals about the importance of participating in trials could encourage both ill and healthy individuals to view this as a routine health behavior.”

“We are pleased to have joined with Research!America in commissioning this important survey and gratified that the public’s knowledge of and attitudes toward clinical trials have moved in positive directions since 2013,” said Doug Peddicord, executive director of ACRO.

“The option to participate in a clinical trial, when appropriate, should be a routine part of the healthcare encounter, and ACRO will continue to work with Research!America and others to spread that message to doctors and patients alike.”

Survey results

The survey of 1000 US adults was conducted by Zogby Analytics for Research!America and ACRO in July 2017. The margin of error is +/- 3.1 percentage points.

Ninety percent of survey respondents said clinical trials are important to advancing science, and 87% said trials are important to improving the nation’s health. Seventy-five percent said taking part in clinical trials is as valuable to the US healthcare system as giving blood.

Eighty-six percent of respondents said healthcare professionals should discuss clinical trials with patients diagnosed with a disease as part of their standard care.

And 44% of respondents said clinical trial participation should be a routine health behavior, whether a person is healthy or ill, similar to getting an annual checkup with a healthcare provider.

Men (48%) were significantly more likely than women (39%) to say trial participation should be routine. A larger percentage of 18- to 29-year-olds (53%) and 30- to 49-year olds (48%) were in favor of routine trial participation, compared to people ages 50 to 64 (38%) and those 65 and older (34%).

Eighty percent of respondents said they had heard of a clinical trial, and 18% said they or someone in their family had participated in one.

Seventy-four percent of respondents said they would participate in a clinical trial if asked by someone they trust.

However, respondents were split on whether it’s important for everyone to take part in a clinical trial if asked. Forty-four percent said it is important, 45% disagreed, and 12% were not sure.

More than half of respondents (55%) said people don’t participate in clinical trials because of lack of awareness and information, 43% said it’s because trials are viewed as “too risky,” 41% said it’s due to a lack of information about the process, 38% said it’s due to a lack of trust, and 34% said it’s due to the risk of adverse health effects.

Nearly two-thirds of respondents (64%) said a doctor or healthcare provider is a reliable source for clinical trial information.

Respondents said doctors and other healthcare providers (44%), followed by the government (23%), have the greatest responsibility in educating the public about clinical trials. However, 74% said no healthcare professional has ever talked to them about medical research.

Seventy-two percent of respondents said they would be likely to use technology such as apps and monitoring devices to share their personal health data for clinical research.

Eighty-eight percent said clinical trial participants should have access to trial results. And 47% said they would like having clinical trial information/data/results delivered through their phone.

AML cells

Researchers say they have determined which patients will respond to treatment with SY-1425, a retinoic acid receptor alpha (RARα) agonist.

The team discovered a subset of patients with acute myeloid leukemia (AML) who had a super-enhancer associated with the RARA gene, which is predictive of response to SY-1425.

The researchers also identified a subset of patients with myelodysplastic syndromes (MDS) who had high expression of the RARA gene.

And experiments showed that RARA-high MDS had a similar response to SY-1425 as that seen in AML driven by the RARA super-enhancer.

Ravindra Majeti MD, PhD, of Stanford University School of Medicine in California, and colleagues reported these findings in Cancer Discovery. Employees of Syros Pharmaceuticals, the company developing SY-1425, were also involved in this research.

In collaboration with the Majeti lab, Syros used its gene control platform to analyze 66 AML patients’ tumor samples. In this way, the researchers identified 6 distinct patient subsets based on super-enhancer profiles, including 1 enriched for a super-enhancer associated with the RARA gene.

The team found that super-enhancer profiles were strongly associated with survival outcomes, often independent of known genetic mutations in AML.

The RARA super-enhancer was associated with high expression of the RARA gene, which codes for a transcription factor targeted by SY-1425.

The RARA super-enhancer was predictive of response to SY-1425. In AML cells with high RARA expression, SY-1425 reduced proliferation and promoted differentiation.

Moreover, SY-1425 decreased tumor burden and prolonged survival in patient-derived xenograft models of AML with high RARA expression. However, there was no effect on AML cells or models with low RARA expression.

The researchers said SY-1425 induced profound transcriptional changes promoting cell differentiation in AML cells with high RARA expression, but the drug produced little to no transcriptional changes in AML cells with low RARA expression.

DHRS3 was the most strongly and rapidly induced gene in response to treatment with SY-1425. This led to the identification of DHRS3 induction as an early indicator of whether SY-1425 is affecting the targeted biology in defined subsets of AML and MDS patients. It is therefore used as a pharmacodynamic marker in the ongoing phase 2 trial of SY-1425.

In this trial, researchers are assessing the safety and efficacy of SY-1425 as a single agent in 4 AML and MDS patient populations, as well as testing SY-1425 in combination with azacitidine in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy.

AML cells

Researchers say they have determined which patients will respond to treatment with SY-1425, a retinoic acid receptor alpha (RARα) agonist.

The team discovered a subset of patients with acute myeloid leukemia (AML) who had a super-enhancer associated with the RARA gene, which is predictive of response to SY-1425.

The researchers also identified a subset of patients with myelodysplastic syndromes (MDS) who had high expression of the RARA gene.

And experiments showed that RARA-high MDS had a similar response to SY-1425 as that seen in AML driven by the RARA super-enhancer.

Ravindra Majeti MD, PhD, of Stanford University School of Medicine in California, and colleagues reported these findings in Cancer Discovery. Employees of Syros Pharmaceuticals, the company developing SY-1425, were also involved in this research.

In collaboration with the Majeti lab, Syros used its gene control platform to analyze 66 AML patients’ tumor samples. In this way, the researchers identified 6 distinct patient subsets based on super-enhancer profiles, including 1 enriched for a super-enhancer associated with the RARA gene.

The team found that super-enhancer profiles were strongly associated with survival outcomes, often independent of known genetic mutations in AML.

The RARA super-enhancer was associated with high expression of the RARA gene, which codes for a transcription factor targeted by SY-1425.

The RARA super-enhancer was predictive of response to SY-1425. In AML cells with high RARA expression, SY-1425 reduced proliferation and promoted differentiation.

Moreover, SY-1425 decreased tumor burden and prolonged survival in patient-derived xenograft models of AML with high RARA expression. However, there was no effect on AML cells or models with low RARA expression.

The researchers said SY-1425 induced profound transcriptional changes promoting cell differentiation in AML cells with high RARA expression, but the drug produced little to no transcriptional changes in AML cells with low RARA expression.

DHRS3 was the most strongly and rapidly induced gene in response to treatment with SY-1425. This led to the identification of DHRS3 induction as an early indicator of whether SY-1425 is affecting the targeted biology in defined subsets of AML and MDS patients. It is therefore used as a pharmacodynamic marker in the ongoing phase 2 trial of SY-1425.

In this trial, researchers are assessing the safety and efficacy of SY-1425 as a single agent in 4 AML and MDS patient populations, as well as testing SY-1425 in combination with azacitidine in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy.

AML cells

Researchers say they have determined which patients will respond to treatment with SY-1425, a retinoic acid receptor alpha (RARα) agonist.

The team discovered a subset of patients with acute myeloid leukemia (AML) who had a super-enhancer associated with the RARA gene, which is predictive of response to SY-1425.

The researchers also identified a subset of patients with myelodysplastic syndromes (MDS) who had high expression of the RARA gene.

And experiments showed that RARA-high MDS had a similar response to SY-1425 as that seen in AML driven by the RARA super-enhancer.

Ravindra Majeti MD, PhD, of Stanford University School of Medicine in California, and colleagues reported these findings in Cancer Discovery. Employees of Syros Pharmaceuticals, the company developing SY-1425, were also involved in this research.

In collaboration with the Majeti lab, Syros used its gene control platform to analyze 66 AML patients’ tumor samples. In this way, the researchers identified 6 distinct patient subsets based on super-enhancer profiles, including 1 enriched for a super-enhancer associated with the RARA gene.

The team found that super-enhancer profiles were strongly associated with survival outcomes, often independent of known genetic mutations in AML.

The RARA super-enhancer was associated with high expression of the RARA gene, which codes for a transcription factor targeted by SY-1425.

The RARA super-enhancer was predictive of response to SY-1425. In AML cells with high RARA expression, SY-1425 reduced proliferation and promoted differentiation.

Moreover, SY-1425 decreased tumor burden and prolonged survival in patient-derived xenograft models of AML with high RARA expression. However, there was no effect on AML cells or models with low RARA expression.

The researchers said SY-1425 induced profound transcriptional changes promoting cell differentiation in AML cells with high RARA expression, but the drug produced little to no transcriptional changes in AML cells with low RARA expression.

DHRS3 was the most strongly and rapidly induced gene in response to treatment with SY-1425. This led to the identification of DHRS3 induction as an early indicator of whether SY-1425 is affecting the targeted biology in defined subsets of AML and MDS patients. It is therefore used as a pharmacodynamic marker in the ongoing phase 2 trial of SY-1425.

In this trial, researchers are assessing the safety and efficacy of SY-1425 as a single agent in 4 AML and MDS patient populations, as well as testing SY-1425 in combination with azacitidine in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy.

Photo by Marja Helander

England and Scotland are changing some of their policies regarding blood donation, shortening the deferral periods for donors who engage in “high-risk” sexual behavior.

Wales is considering making the same changes to its blood donation policies but has not yet made a commitment to do so.

The policy changes will mean that men who have sex with men (MSM), commercial sex workers, and people with sexual partners who have a high risk of sexually transmitted infections (including those who have been sexually active in areas where HIV is common) will be able to donate blood after 3 months have passed since their last sexual activity.

At present, MSMs and individuals with high-risk sexual partners can only donate blood after 12 months have passed since their last sexual activity, and commercial sex workers are permanently banned from giving blood.

The change in deferral periods will begin to take effect in England in early 2018 and in Scotland in November 2017. Until then, the existing rules still apply.

The Advisory Committee on the Safety of Blood, Tissues and Organs (SaBTO), which advises UK ministers and health departments, recommended the aforementioned policy changes following a review of blood donor criteria and risk assessment of certain behaviors.

SaBTO also recommended shortening the deferral period for potential donors who have undergone acupuncture, piercing, tattooing, and endoscopy, as well as those with a history of non-prescribed injectable drug use.

However, this change requires changing UK legislation, in addition to a derogation from or amendments to current European Union legislation.

“We welcome the review by SaBTO and the recommendations,” said Moira Carter, Associate Director of Donor Services and Transport for the Scottish National Blood Transfusion Service.

“The updates for donor eligibility will allow more people the opportunity to give blood. The changes take into account the latest available medical and scientific evidence about the risk of acquiring infections that can be passed on in blood, along with evidence supporting the reliability of the blood screening tests we use.”

“We’re pleased that the lifetime ban on former and current sex workers has been lifted, and the deferral period is now in line with other deferrals based on sexual behavior,” said Alex Phillips, Blood Donations Policy Lead at Terrence Higgins Trust in London.

“We know from our research that the majority of sex workers take great care of their sexual health, with 98% of sex workers we asked rating their sexual health as very important, 76% having a sexual health check up every 3 months, and 98% knowing their HIV status.”

“Medical evidence is, of course, constantly and quickly being updated, so it’s important that the deferral periods are regularly reviewed in line with the latest evidence. We therefore hope that today’s changes will pave the way for more progress as further evidence becomes available.”

Photo by Marja Helander

England and Scotland are changing some of their policies regarding blood donation, shortening the deferral periods for donors who engage in “high-risk” sexual behavior.

Wales is considering making the same changes to its blood donation policies but has not yet made a commitment to do so.

The policy changes will mean that men who have sex with men (MSM), commercial sex workers, and people with sexual partners who have a high risk of sexually transmitted infections (including those who have been sexually active in areas where HIV is common) will be able to donate blood after 3 months have passed since their last sexual activity.

At present, MSMs and individuals with high-risk sexual partners can only donate blood after 12 months have passed since their last sexual activity, and commercial sex workers are permanently banned from giving blood.

The change in deferral periods will begin to take effect in England in early 2018 and in Scotland in November 2017. Until then, the existing rules still apply.

The Advisory Committee on the Safety of Blood, Tissues and Organs (SaBTO), which advises UK ministers and health departments, recommended the aforementioned policy changes following a review of blood donor criteria and risk assessment of certain behaviors.

SaBTO also recommended shortening the deferral period for potential donors who have undergone acupuncture, piercing, tattooing, and endoscopy, as well as those with a history of non-prescribed injectable drug use.

However, this change requires changing UK legislation, in addition to a derogation from or amendments to current European Union legislation.

“We welcome the review by SaBTO and the recommendations,” said Moira Carter, Associate Director of Donor Services and Transport for the Scottish National Blood Transfusion Service.

“The updates for donor eligibility will allow more people the opportunity to give blood. The changes take into account the latest available medical and scientific evidence about the risk of acquiring infections that can be passed on in blood, along with evidence supporting the reliability of the blood screening tests we use.”

“We’re pleased that the lifetime ban on former and current sex workers has been lifted, and the deferral period is now in line with other deferrals based on sexual behavior,” said Alex Phillips, Blood Donations Policy Lead at Terrence Higgins Trust in London.

“We know from our research that the majority of sex workers take great care of their sexual health, with 98% of sex workers we asked rating their sexual health as very important, 76% having a sexual health check up every 3 months, and 98% knowing their HIV status.”

“Medical evidence is, of course, constantly and quickly being updated, so it’s important that the deferral periods are regularly reviewed in line with the latest evidence. We therefore hope that today’s changes will pave the way for more progress as further evidence becomes available.”

Photo by Marja Helander

England and Scotland are changing some of their policies regarding blood donation, shortening the deferral periods for donors who engage in “high-risk” sexual behavior.

Wales is considering making the same changes to its blood donation policies but has not yet made a commitment to do so.

The policy changes will mean that men who have sex with men (MSM), commercial sex workers, and people with sexual partners who have a high risk of sexually transmitted infections (including those who have been sexually active in areas where HIV is common) will be able to donate blood after 3 months have passed since their last sexual activity.

At present, MSMs and individuals with high-risk sexual partners can only donate blood after 12 months have passed since their last sexual activity, and commercial sex workers are permanently banned from giving blood.

The change in deferral periods will begin to take effect in England in early 2018 and in Scotland in November 2017. Until then, the existing rules still apply.

The Advisory Committee on the Safety of Blood, Tissues and Organs (SaBTO), which advises UK ministers and health departments, recommended the aforementioned policy changes following a review of blood donor criteria and risk assessment of certain behaviors.

SaBTO also recommended shortening the deferral period for potential donors who have undergone acupuncture, piercing, tattooing, and endoscopy, as well as those with a history of non-prescribed injectable drug use.

However, this change requires changing UK legislation, in addition to a derogation from or amendments to current European Union legislation.

“We welcome the review by SaBTO and the recommendations,” said Moira Carter, Associate Director of Donor Services and Transport for the Scottish National Blood Transfusion Service.

“The updates for donor eligibility will allow more people the opportunity to give blood. The changes take into account the latest available medical and scientific evidence about the risk of acquiring infections that can be passed on in blood, along with evidence supporting the reliability of the blood screening tests we use.”

“We’re pleased that the lifetime ban on former and current sex workers has been lifted, and the deferral period is now in line with other deferrals based on sexual behavior,” said Alex Phillips, Blood Donations Policy Lead at Terrence Higgins Trust in London.

“We know from our research that the majority of sex workers take great care of their sexual health, with 98% of sex workers we asked rating their sexual health as very important, 76% having a sexual health check up every 3 months, and 98% knowing their HIV status.”

“Medical evidence is, of course, constantly and quickly being updated, so it’s important that the deferral periods are regularly reviewed in line with the latest evidence. We therefore hope that today’s changes will pave the way for more progress as further evidence becomes available.”