Anemia

Photo by Neil Osterweil

BOSTON—Results of a pilot program suggest preoperative management of anemia can reduce transfusion rates and cut costs, but the effect on patient outcomes isn’t clear.

For this program, anemic patients received dietary guidance and supplementation prior to surgery.

This increased day-of-surgery hemoglobin levels, reduced intraoperative and postoperative transfusions, and resulted in a cost savings of more than $100,000 over the life of the program.

Christine M. Cahill, BSN, MS, RN, of the University of Rochester and Strong Memorial Hospital in Rochester, New York, presented these results at AABB 2018 (abstract PBM4-ST4-22*).

“Anemia has been thought of as a relatively benign thing our patients live with, traditionally, but what we have been finding lately is that anemia is actually more serious than we once thought and is an independent risk factor for hospitalization, readmission, increased patient length of stay, loss of function, and diminished quality of life,” Cahill said.

She added that anemia also increases the likelihood that a patient will require transfusions.

The pilot program was implemented with this in mind. The program, which ran from February 2016 through September 2017, was designed to test the feasibility of diagnosing anemia during a cardiology consult visit and implementing an anemia management plan.

During the study period, 240 patients presenting for elective cardiac surgery were screened for anemia, and 58 were diagnosed as anemic (hemoglobin <12 g/dL). These patients were referred for anemia workups, which showed that 33 patients had iron-deficiency anemia, and 25 had anemia from other causes.

Preoperative anemia management for the iron-deficient patients included oral iron for seven patients, intravenous (IV) iron with or without folate for 20 patients, and oral folate with or without vitamin B12 for five patients. One iron-deficient patient could not have surgery delayed for anemia management.

Of the iron-replete patients, one received oral iron, 17 received folate with or without B12, and seven patients were not treated for anemia.

One iron-deficient patient had a reaction to the infusion and did not receive a scheduled second dose due to the need for immediate surgery. A second patient scheduled for IV iron and folate broke an arm and therefore missed an IV infusion appointment. No other complications or reactions occurred.

Results

The researchers compared the 58 patients from the pilot program to control subjects—patients who underwent cardiac surgery from March through July 2015, matched by age, sex, and procedures.

The anemia management group received 10 red blood cell (RBC) units intraoperatively, compared to 68 intraoperative RBC units for controls. The total number of postoperative RBC units was 13 and 22, respectively.

The rate of RBC transfusions was 24% in the anemia management group and 60% in controls (P<0.0001). The average RBC units per patient was 0.4 and 2.07, respectively (P<0.0001).

Patients in the anemia management program also had significantly higher day-of-surgery hemoglobin than controls—11.01 and 10.16 g/dL, respectively (P<0.001).

The program provided an average per-patient savings in acquisition costs of $367.40, an average transfusion cost savings of $1,837, and a total cost savings of $106,546 over the life of the program.

The key to success of a similar program is “to make sure you do your homework,” Cahill said.

Specifically, she recommended feasibility studies, evaluation of the potential impact of infusions on the service, work flow analyses, and cost analyses. It’s also important to get high-level administrative support as well as buy-in from surgeons and patients, she added.

Future studies should include assessment of patient outcomes, safety, and length of intensive care unit and hospital stay, Cahill emphasized.

This study was internally funded. Cahill reported no conflicts of interest.

*The data in the presentation differ from the abstract.

Photo by Neil Osterweil

BOSTON—Results of a pilot program suggest preoperative management of anemia can reduce transfusion rates and cut costs, but the effect on patient outcomes isn’t clear.

For this program, anemic patients received dietary guidance and supplementation prior to surgery.

This increased day-of-surgery hemoglobin levels, reduced intraoperative and postoperative transfusions, and resulted in a cost savings of more than $100,000 over the life of the program.

Christine M. Cahill, BSN, MS, RN, of the University of Rochester and Strong Memorial Hospital in Rochester, New York, presented these results at AABB 2018 (abstract PBM4-ST4-22*).

“Anemia has been thought of as a relatively benign thing our patients live with, traditionally, but what we have been finding lately is that anemia is actually more serious than we once thought and is an independent risk factor for hospitalization, readmission, increased patient length of stay, loss of function, and diminished quality of life,” Cahill said.

She added that anemia also increases the likelihood that a patient will require transfusions.

The pilot program was implemented with this in mind. The program, which ran from February 2016 through September 2017, was designed to test the feasibility of diagnosing anemia during a cardiology consult visit and implementing an anemia management plan.

During the study period, 240 patients presenting for elective cardiac surgery were screened for anemia, and 58 were diagnosed as anemic (hemoglobin <12 g/dL). These patients were referred for anemia workups, which showed that 33 patients had iron-deficiency anemia, and 25 had anemia from other causes.

Preoperative anemia management for the iron-deficient patients included oral iron for seven patients, intravenous (IV) iron with or without folate for 20 patients, and oral folate with or without vitamin B12 for five patients. One iron-deficient patient could not have surgery delayed for anemia management.

Of the iron-replete patients, one received oral iron, 17 received folate with or without B12, and seven patients were not treated for anemia.

One iron-deficient patient had a reaction to the infusion and did not receive a scheduled second dose due to the need for immediate surgery. A second patient scheduled for IV iron and folate broke an arm and therefore missed an IV infusion appointment. No other complications or reactions occurred.

Results

The researchers compared the 58 patients from the pilot program to control subjects—patients who underwent cardiac surgery from March through July 2015, matched by age, sex, and procedures.

The anemia management group received 10 red blood cell (RBC) units intraoperatively, compared to 68 intraoperative RBC units for controls. The total number of postoperative RBC units was 13 and 22, respectively.

The rate of RBC transfusions was 24% in the anemia management group and 60% in controls (P<0.0001). The average RBC units per patient was 0.4 and 2.07, respectively (P<0.0001).

Patients in the anemia management program also had significantly higher day-of-surgery hemoglobin than controls—11.01 and 10.16 g/dL, respectively (P<0.001).

The program provided an average per-patient savings in acquisition costs of $367.40, an average transfusion cost savings of $1,837, and a total cost savings of $106,546 over the life of the program.

The key to success of a similar program is “to make sure you do your homework,” Cahill said.

Specifically, she recommended feasibility studies, evaluation of the potential impact of infusions on the service, work flow analyses, and cost analyses. It’s also important to get high-level administrative support as well as buy-in from surgeons and patients, she added.

Future studies should include assessment of patient outcomes, safety, and length of intensive care unit and hospital stay, Cahill emphasized.

This study was internally funded. Cahill reported no conflicts of interest.

*The data in the presentation differ from the abstract.

Photo by Neil Osterweil

BOSTON—Results of a pilot program suggest preoperative management of anemia can reduce transfusion rates and cut costs, but the effect on patient outcomes isn’t clear.

For this program, anemic patients received dietary guidance and supplementation prior to surgery.

This increased day-of-surgery hemoglobin levels, reduced intraoperative and postoperative transfusions, and resulted in a cost savings of more than $100,000 over the life of the program.

Christine M. Cahill, BSN, MS, RN, of the University of Rochester and Strong Memorial Hospital in Rochester, New York, presented these results at AABB 2018 (abstract PBM4-ST4-22*).

“Anemia has been thought of as a relatively benign thing our patients live with, traditionally, but what we have been finding lately is that anemia is actually more serious than we once thought and is an independent risk factor for hospitalization, readmission, increased patient length of stay, loss of function, and diminished quality of life,” Cahill said.

She added that anemia also increases the likelihood that a patient will require transfusions.

The pilot program was implemented with this in mind. The program, which ran from February 2016 through September 2017, was designed to test the feasibility of diagnosing anemia during a cardiology consult visit and implementing an anemia management plan.

During the study period, 240 patients presenting for elective cardiac surgery were screened for anemia, and 58 were diagnosed as anemic (hemoglobin <12 g/dL). These patients were referred for anemia workups, which showed that 33 patients had iron-deficiency anemia, and 25 had anemia from other causes.

Preoperative anemia management for the iron-deficient patients included oral iron for seven patients, intravenous (IV) iron with or without folate for 20 patients, and oral folate with or without vitamin B12 for five patients. One iron-deficient patient could not have surgery delayed for anemia management.

Of the iron-replete patients, one received oral iron, 17 received folate with or without B12, and seven patients were not treated for anemia.

One iron-deficient patient had a reaction to the infusion and did not receive a scheduled second dose due to the need for immediate surgery. A second patient scheduled for IV iron and folate broke an arm and therefore missed an IV infusion appointment. No other complications or reactions occurred.

Results

The researchers compared the 58 patients from the pilot program to control subjects—patients who underwent cardiac surgery from March through July 2015, matched by age, sex, and procedures.

The anemia management group received 10 red blood cell (RBC) units intraoperatively, compared to 68 intraoperative RBC units for controls. The total number of postoperative RBC units was 13 and 22, respectively.

The rate of RBC transfusions was 24% in the anemia management group and 60% in controls (P<0.0001). The average RBC units per patient was 0.4 and 2.07, respectively (P<0.0001).

Patients in the anemia management program also had significantly higher day-of-surgery hemoglobin than controls—11.01 and 10.16 g/dL, respectively (P<0.001).

The program provided an average per-patient savings in acquisition costs of $367.40, an average transfusion cost savings of $1,837, and a total cost savings of $106,546 over the life of the program.

The key to success of a similar program is “to make sure you do your homework,” Cahill said.

Specifically, she recommended feasibility studies, evaluation of the potential impact of infusions on the service, work flow analyses, and cost analyses. It’s also important to get high-level administrative support as well as buy-in from surgeons and patients, she added.

Future studies should include assessment of patient outcomes, safety, and length of intensive care unit and hospital stay, Cahill emphasized.

This study was internally funded. Cahill reported no conflicts of interest.

*The data in the presentation differ from the abstract.

BOSTON – A pilot anemia optimization program resulted in significant increases in day-of-surgery hemoglobin levels and reductions in RBC transfusion rates and costs in one center, but whether patient outcomes also improved is still not known.

By diagnosing anemia at the preanesthesia visit and providing anemic patients with dietary guidance and supplementation prior to cardiac surgery, blood program managers noticed a more than $360 reduction in per-patient blood-product acquisition costs, a more than $1,800 average reduction per patient in transfusion costs, and overall cost savings of more than $100,000 over 18 months, compared with historical data.

The findings were reported by Christine M. Cahill, RN, from Strong Memorial Hospital in Rochester, N.Y., and the University of Rochester (N.Y.), at AABB 2018, the annual meeting of the group formerly known as the American Association of Blood Banks.

“Anemia has been thought of as a relatively benign thing our patients live with traditionally, but what we have been finding lately is that anemia is actually more serious than we once thought, and is an independent risk factor for hospitalization, readmission, increased patient length of stay, loss of function, and diminished quality of life,” she said.

Anemia also increases the likelihood that a patient will require allogeneic transfusions and is an independent risk factor for morbidity and mortality, she added.

The pilot program, which ran from February 2016 to September 2017, was designed to test the feasibility of diagnosing anemia during a cardiology consult visit and implementing a management plan.

During the study period, 240 patients presenting for elective cardiac surgery were screened for anemia, and 58 were diagnosed as anemic, defined as a hemoglobin level of less than 12 g/dL. These patients were referred for anemia work-ups, which found that 33 patients had iron-deficient anemia and 25 had anemia from other causes. Controls were patients who underwent cardiac surgery from March to July 2015, matched by age, sex, and procedures.

Treatments for iron-deficient patients included oral iron (7 patients), intravenous iron with or without folate (20 patients), or oral folate with or without vitamin B12 (5 patients). One iron-deficient patient could not have surgery delayed for anemia management.

Of the iron-replete patients, one received oral iron and 17 received folate plus or minus vitamin B₁₂. The remaining seven iron-replete patients were not treated for anemia.

One iron-deficient patient had a reaction to the infusion and did not receive a scheduled second dose due to the need for immediate surgery. A second patient scheduled for intravenous iron and folate broke an arm and therefore missed an intravenous infusion appointment. No other complications or reactions occurred.

Intraoperative transfusion units used in the anemia management group totaled 10, compared with 68 for controls. Postoperative transfusion units used were also significantly lower following anemia management at 13 versus 122, respectively.

The rate of RBC transfusions among patients with anemia management was 24%, compared with 60% for controls (P less than .0001). Patients in the management program also had significantly higher day-of-surgery hemoglobin, at 11.01 g/dL versus 10.16 g/dL (P less than .001), and less RBC utilization, at an average 0.40 units per patient versus 2.07 for controls (P less than .0001).

The average per patient savings in acquisition costs was $367.40, the average transfusion cost saving was $1,837, and the total cost savings over the life of the pilot program was $106,546.

The keys to success for similar programs is “to make sure you do your homework,” Ms. Cahill said. Specifically, she recommended feasibility studies, evaluation of the potential impact of infusions on the service, work flow analyses, and cost analyses. It’s also important to get high-level administrative support as well as buy-in from surgeons and patients.

Future studies should include assessment of patient outcomes, safety, and length of ICU and hospital stay, she emphasized.

The study was internally funded. Ms. Cahill reported having no conflicts of interest.

SOURCE: Cahill CM et al. AABB 2018, Abstract PBM4-ST4-22.

BOSTON – A pilot anemia optimization program resulted in significant increases in day-of-surgery hemoglobin levels and reductions in RBC transfusion rates and costs in one center, but whether patient outcomes also improved is still not known.

By diagnosing anemia at the preanesthesia visit and providing anemic patients with dietary guidance and supplementation prior to cardiac surgery, blood program managers noticed a more than $360 reduction in per-patient blood-product acquisition costs, a more than $1,800 average reduction per patient in transfusion costs, and overall cost savings of more than $100,000 over 18 months, compared with historical data.

The findings were reported by Christine M. Cahill, RN, from Strong Memorial Hospital in Rochester, N.Y., and the University of Rochester (N.Y.), at AABB 2018, the annual meeting of the group formerly known as the American Association of Blood Banks.

“Anemia has been thought of as a relatively benign thing our patients live with traditionally, but what we have been finding lately is that anemia is actually more serious than we once thought, and is an independent risk factor for hospitalization, readmission, increased patient length of stay, loss of function, and diminished quality of life,” she said.

Anemia also increases the likelihood that a patient will require allogeneic transfusions and is an independent risk factor for morbidity and mortality, she added.

The pilot program, which ran from February 2016 to September 2017, was designed to test the feasibility of diagnosing anemia during a cardiology consult visit and implementing a management plan.

During the study period, 240 patients presenting for elective cardiac surgery were screened for anemia, and 58 were diagnosed as anemic, defined as a hemoglobin level of less than 12 g/dL. These patients were referred for anemia work-ups, which found that 33 patients had iron-deficient anemia and 25 had anemia from other causes. Controls were patients who underwent cardiac surgery from March to July 2015, matched by age, sex, and procedures.

Treatments for iron-deficient patients included oral iron (7 patients), intravenous iron with or without folate (20 patients), or oral folate with or without vitamin B12 (5 patients). One iron-deficient patient could not have surgery delayed for anemia management.

Of the iron-replete patients, one received oral iron and 17 received folate plus or minus vitamin B₁₂. The remaining seven iron-replete patients were not treated for anemia.

One iron-deficient patient had a reaction to the infusion and did not receive a scheduled second dose due to the need for immediate surgery. A second patient scheduled for intravenous iron and folate broke an arm and therefore missed an intravenous infusion appointment. No other complications or reactions occurred.

Intraoperative transfusion units used in the anemia management group totaled 10, compared with 68 for controls. Postoperative transfusion units used were also significantly lower following anemia management at 13 versus 122, respectively.

The rate of RBC transfusions among patients with anemia management was 24%, compared with 60% for controls (P less than .0001). Patients in the management program also had significantly higher day-of-surgery hemoglobin, at 11.01 g/dL versus 10.16 g/dL (P less than .001), and less RBC utilization, at an average 0.40 units per patient versus 2.07 for controls (P less than .0001).

The average per patient savings in acquisition costs was $367.40, the average transfusion cost saving was $1,837, and the total cost savings over the life of the pilot program was $106,546.

The keys to success for similar programs is “to make sure you do your homework,” Ms. Cahill said. Specifically, she recommended feasibility studies, evaluation of the potential impact of infusions on the service, work flow analyses, and cost analyses. It’s also important to get high-level administrative support as well as buy-in from surgeons and patients.

Future studies should include assessment of patient outcomes, safety, and length of ICU and hospital stay, she emphasized.

The study was internally funded. Ms. Cahill reported having no conflicts of interest.

SOURCE: Cahill CM et al. AABB 2018, Abstract PBM4-ST4-22.

BOSTON – A pilot anemia optimization program resulted in significant increases in day-of-surgery hemoglobin levels and reductions in RBC transfusion rates and costs in one center, but whether patient outcomes also improved is still not known.

By diagnosing anemia at the preanesthesia visit and providing anemic patients with dietary guidance and supplementation prior to cardiac surgery, blood program managers noticed a more than $360 reduction in per-patient blood-product acquisition costs, a more than $1,800 average reduction per patient in transfusion costs, and overall cost savings of more than $100,000 over 18 months, compared with historical data.

The findings were reported by Christine M. Cahill, RN, from Strong Memorial Hospital in Rochester, N.Y., and the University of Rochester (N.Y.), at AABB 2018, the annual meeting of the group formerly known as the American Association of Blood Banks.

“Anemia has been thought of as a relatively benign thing our patients live with traditionally, but what we have been finding lately is that anemia is actually more serious than we once thought, and is an independent risk factor for hospitalization, readmission, increased patient length of stay, loss of function, and diminished quality of life,” she said.

Anemia also increases the likelihood that a patient will require allogeneic transfusions and is an independent risk factor for morbidity and mortality, she added.

The pilot program, which ran from February 2016 to September 2017, was designed to test the feasibility of diagnosing anemia during a cardiology consult visit and implementing a management plan.

During the study period, 240 patients presenting for elective cardiac surgery were screened for anemia, and 58 were diagnosed as anemic, defined as a hemoglobin level of less than 12 g/dL. These patients were referred for anemia work-ups, which found that 33 patients had iron-deficient anemia and 25 had anemia from other causes. Controls were patients who underwent cardiac surgery from March to July 2015, matched by age, sex, and procedures.

Treatments for iron-deficient patients included oral iron (7 patients), intravenous iron with or without folate (20 patients), or oral folate with or without vitamin B12 (5 patients). One iron-deficient patient could not have surgery delayed for anemia management.

Of the iron-replete patients, one received oral iron and 17 received folate plus or minus vitamin B₁₂. The remaining seven iron-replete patients were not treated for anemia.

One iron-deficient patient had a reaction to the infusion and did not receive a scheduled second dose due to the need for immediate surgery. A second patient scheduled for intravenous iron and folate broke an arm and therefore missed an intravenous infusion appointment. No other complications or reactions occurred.

Intraoperative transfusion units used in the anemia management group totaled 10, compared with 68 for controls. Postoperative transfusion units used were also significantly lower following anemia management at 13 versus 122, respectively.

The rate of RBC transfusions among patients with anemia management was 24%, compared with 60% for controls (P less than .0001). Patients in the management program also had significantly higher day-of-surgery hemoglobin, at 11.01 g/dL versus 10.16 g/dL (P less than .001), and less RBC utilization, at an average 0.40 units per patient versus 2.07 for controls (P less than .0001).

The average per patient savings in acquisition costs was $367.40, the average transfusion cost saving was $1,837, and the total cost savings over the life of the pilot program was $106,546.

The keys to success for similar programs is “to make sure you do your homework,” Ms. Cahill said. Specifically, she recommended feasibility studies, evaluation of the potential impact of infusions on the service, work flow analyses, and cost analyses. It’s also important to get high-level administrative support as well as buy-in from surgeons and patients.

Future studies should include assessment of patient outcomes, safety, and length of ICU and hospital stay, she emphasized.

The study was internally funded. Ms. Cahill reported having no conflicts of interest.

SOURCE: Cahill CM et al. AABB 2018, Abstract PBM4-ST4-22.

Photo by Petr Kratochvil

An analysis of more than 2,000 U.S. patients with blood cancers revealed an average healthcare cost of almost $157,000 in the first year after diagnosis.

Costs were highest for acute leukemia patients—almost triple the average for all blood cancers.

Out-of-pocket (OOP) costs were initially highest for acute leukemia patients. However, over time, OOP costs became highest for patients with multiple myeloma.

These results are included in a report commissioned by the Leukemia & Lymphoma Society and prepared by the actuarial firm Milliman.

The report is based on data from the Truven Health MarketScan commercial claims databases.

The cost figures are drawn from data for 2,332 patients, ages 18 to 64, who were diagnosed with blood cancer in 2014 and followed through 2016. This includes the following:

• 1,468 patients with lymphoma
• 286 with chronic leukemia
• 282 with multiple myeloma
• 148 with acute leukemia
• 148 with bone marrow disorders (myelodysplastic syndromes).

The average allowed spending—the amount paid by the payer and patient combined—in the first 12 months after diagnosis was:

• $156,845 overall
• $463,414 for acute leukemia
• $213,879 for multiple myeloma
• $133,744 for bone marrow disorders
• $130,545 for lymphoma
• $88,913 for chronic leukemia.

Differences in OOP costs were smaller, although OOP spending was 32% higher for acute leukemia patients than the overall average.

Average OOP costs—which include coinsurance, copay, and deductible—in the first 12 months after diagnosis were:

• $3,877 overall
• $5,147 for acute leukemia
• $4,849 for multiple myeloma
• $3,695 for lymphoma
• $3,480 for chronic leukemia
• $3,336 for bone marrow disorders.

Although OOP costs were initially highest for acute leukemia patients, over time, costs for multiple myeloma patients became the highest.

The average OOP costs in the month of diagnosis were $1,637 for acute leukemia patients and $1,210 for multiple myeloma patients.

The total accumulated OOP costs 3 years after diagnosis were $8,797 for acute leukemia and $9,127 for multiple myeloma. For the other blood cancers, the average 3-year accumulated OOP costs were under $7,800.

The Leukemia & Lymphoma Society received support from Pfizer, Genentech, and Amgen for this work.

Photo by Petr Kratochvil

An analysis of more than 2,000 U.S. patients with blood cancers revealed an average healthcare cost of almost $157,000 in the first year after diagnosis.

Costs were highest for acute leukemia patients—almost triple the average for all blood cancers.

Out-of-pocket (OOP) costs were initially highest for acute leukemia patients. However, over time, OOP costs became highest for patients with multiple myeloma.

These results are included in a report commissioned by the Leukemia & Lymphoma Society and prepared by the actuarial firm Milliman.

The report is based on data from the Truven Health MarketScan commercial claims databases.

The cost figures are drawn from data for 2,332 patients, ages 18 to 64, who were diagnosed with blood cancer in 2014 and followed through 2016. This includes the following:

• 1,468 patients with lymphoma
• 286 with chronic leukemia
• 282 with multiple myeloma
• 148 with acute leukemia
• 148 with bone marrow disorders (myelodysplastic syndromes).

The average allowed spending—the amount paid by the payer and patient combined—in the first 12 months after diagnosis was:

• $156,845 overall
• $463,414 for acute leukemia
• $213,879 for multiple myeloma
• $133,744 for bone marrow disorders
• $130,545 for lymphoma
• $88,913 for chronic leukemia.

Differences in OOP costs were smaller, although OOP spending was 32% higher for acute leukemia patients than the overall average.

Average OOP costs—which include coinsurance, copay, and deductible—in the first 12 months after diagnosis were:

• $3,877 overall
• $5,147 for acute leukemia
• $4,849 for multiple myeloma
• $3,695 for lymphoma
• $3,480 for chronic leukemia
• $3,336 for bone marrow disorders.

Although OOP costs were initially highest for acute leukemia patients, over time, costs for multiple myeloma patients became the highest.

The average OOP costs in the month of diagnosis were $1,637 for acute leukemia patients and $1,210 for multiple myeloma patients.

The total accumulated OOP costs 3 years after diagnosis were $8,797 for acute leukemia and $9,127 for multiple myeloma. For the other blood cancers, the average 3-year accumulated OOP costs were under $7,800.

The Leukemia & Lymphoma Society received support from Pfizer, Genentech, and Amgen for this work.

Photo by Petr Kratochvil

An analysis of more than 2,000 U.S. patients with blood cancers revealed an average healthcare cost of almost $157,000 in the first year after diagnosis.

Costs were highest for acute leukemia patients—almost triple the average for all blood cancers.

Out-of-pocket (OOP) costs were initially highest for acute leukemia patients. However, over time, OOP costs became highest for patients with multiple myeloma.

These results are included in a report commissioned by the Leukemia & Lymphoma Society and prepared by the actuarial firm Milliman.

The report is based on data from the Truven Health MarketScan commercial claims databases.

The cost figures are drawn from data for 2,332 patients, ages 18 to 64, who were diagnosed with blood cancer in 2014 and followed through 2016. This includes the following:

• 1,468 patients with lymphoma
• 286 with chronic leukemia
• 282 with multiple myeloma
• 148 with acute leukemia
• 148 with bone marrow disorders (myelodysplastic syndromes).

The average allowed spending—the amount paid by the payer and patient combined—in the first 12 months after diagnosis was:

• $156,845 overall
• $463,414 for acute leukemia
• $213,879 for multiple myeloma
• $133,744 for bone marrow disorders
• $130,545 for lymphoma
• $88,913 for chronic leukemia.

Differences in OOP costs were smaller, although OOP spending was 32% higher for acute leukemia patients than the overall average.

Average OOP costs—which include coinsurance, copay, and deductible—in the first 12 months after diagnosis were:

• $3,877 overall
• $5,147 for acute leukemia
• $4,849 for multiple myeloma
• $3,695 for lymphoma
• $3,480 for chronic leukemia
• $3,336 for bone marrow disorders.

Although OOP costs were initially highest for acute leukemia patients, over time, costs for multiple myeloma patients became the highest.

The average OOP costs in the month of diagnosis were $1,637 for acute leukemia patients and $1,210 for multiple myeloma patients.

The total accumulated OOP costs 3 years after diagnosis were $8,797 for acute leukemia and $9,127 for multiple myeloma. For the other blood cancers, the average 3-year accumulated OOP costs were under $7,800.

The Leukemia & Lymphoma Society received support from Pfizer, Genentech, and Amgen for this work.

BETHESDA, MD – While there are experimental treatments such as sevuparin and gene therapy in testing for sickle cell disease (SCD), researchers said this field is lagging because of a lack of funding.

Dr_Microbe/Thinkstock

Yogen Saunthararajah, MD, of the Cleveland Clinic, described what he called a “paltry” landscape of new drugs for SCD at Sickle Cell in Focus, a conference held by the National Institutes of Health.

There are only four main approaches taken by drugs now in clinical testing for addressing the root causes of SCD, despite decades’ worth of research of the genetic and mechanistic underpinnings of this disease, he said.

“It’s pretty sad,” Dr. Saunthararajah said, referring to the quantity of efforts, not their quality.

Within days of his presentation at the conference, one of the drugs he highlighted had officially fallen out of contention. An Oct. 26 post on NIH’s Clinicaltrials.gov site said Incyte had terminated its phase 1 study of INCB059872 in SCD “due to a business decision” not to pursue this indication. Incyte confirmed that it dropped development of INCB059872 for SCD but will continue testing it for other indications, including acute myeloid leukemia (AML).

Dr. Saunthararajah said that work on another approach, using decitabine (Dacogen), for which he has done a phase 1 study, is “struggling,” because of the search for funding.

Two other approaches that Dr. Saunthararajah cited in his presentation appear to remain on track. These are gene therapy and the once-daily voxelotor treatment from Global Blood Therapeutics.

In the field of gene therapy, Sangamo Therapeutics and Sanofi’s Bioverativ in May said the Food and Drug Administration had cleared the way for them to start a phase 1/2 clinical trial for the BIVV003 product that they are developing together. This uses zinc finger nuclease (ZFN) gene-editing technology to modifying a short sequence of the BCL11A gene, with the aim of reactivating fetal hemoglobin.

Bluebird Bio’s LentiGlobin gene therapy has advanced as far as phase 3 for transfusion-dependent beta-thalassemia and phase 1/2 for SCD. In October, the European Medicines Agency accepted the company’s application for approval of LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassemia (TDT) and a non-beta⁰/beta⁰ genotype. The company has not said when it expects to file with the FDA for approval of this treatment.

In the field of oral therapies, Global Blood Therapeutics has said it’s in discussions with the FDA about a potential accelerated approval of voxelotor. The tablet is meant to inhibit the underlying mechanism that causes sickling of red blood cells. In June, the company completed a planned review of early data from its phase 3 trial, known as the HOPE study.

“On the primary endpoint (the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline), a statistically significant increase was demonstrated with voxelotor at both the 1,500-mg and 900-mg doses after 12 weeks of treatment versus placebo,” Global Blood Therapeutics said in an August regulatory filing with the Securities and Exchange Commission.

The company has said that voxelotor may meet the FDA’s standard for accelerated approval under Subpart H program.

In his presentation at the NIH conference, Tom Williams, MD, PhD, of KEMRI/Wellcome Trust Research Programme highlighted a recent review of experimental SCD treatments by Marilyn Jo Telen, MD, of Duke University, Durham, N.C. (Blood. 2016;127[7]:810-9).

Also among the drugs being tested for SCD is Modus Therapeutics’ sevuparin, which Dr. Williams described as being “a heparin-like molecule without the anticoagulant complications.”

The compound originated as a “passion project” of Mats Wahlgren, MD, PhD, of the Karolinska Institute, Stockholm, who developed it for the treatment of malaria, Dr. Williams said. The company that’s developing sevuparin, Modus Therapeutics, is moving it forward first as a treatment for SCD for commercial reasons, Dr. Williams said.

“They think it’s a better first target,” he said.

An ongoing study of sevuparin for painful crisis is expected to be completed in December, with data then expected to be released in the middle of 2019, Ellen K. Donnelly, PhD, chief executive officer of Modus Therapeutics, said in an interview. She cited a mix of scientific, medical and commercial reasons for her company’s decision to advance sevuparin in SCD.

“First, and most importantly, there is proof of clinical benefit of a similar molecule (the low-molecular-weight heparin called tinzaparin) in patients with sickle cell disease,” Dr. Donnelly said. “Unfortunately, there is a bleeding risk with tinzaparin that limits use of the agent for the treatment of sickle cell disease. Sevuparin does not have the bleeding risk and thus is a strong candidate for SCD.”

Dr. Donnelly also noted the emphasis that the FDA’s Division of Hematology Products has put on development of therapeutics for SCD as a reason for proceeding first with this indication. The agency and the American Society of Hematology in early October held a workshop of experts, physicians, patients, and industry collaborators focused on identifying new endpoints for clinical studies.

Still, she noted that commercial reasons did factor into this decision.

“[I]t is possible for a small company like Modus to take an asset all the way to market when developing a therapeutic for a rare disease given the need for fewer patients and smaller trials,” Dr. Donnelly wrote. “As you can see from www.clinicaltrials.gov, we were able to run our phase 2 study with a small number of sites. In addition, in SCD it is possible to get approval with only one or two confirmatory studies.”
 

Financial interests

Other drugs in testing for SCD include rivipansel from GlycoMimetics, for which Pfizer is leading development. The sponsors expect to complete the so-called RESET trial by 2019, according to the clinicaltrials.gov. In this study, which is intended to enroll 350 participants, patients who have vaso-occlusive crises are randomly selected for treatment with either rivipansel or placebo.

Speaking during a question-and-answer session at the NIH conference, Robert Swift, PhD, said there’s a need for inexpensive oral drugs to treat SCD. Many other options will remain beyond the finances of people living in poor countries, he said.

“We need to focus not only on the root cause, but on something that is oral and inexpensive to solve the greater sickle cell problem,” Dr. Swift said.

Large drugmakers already have hospital-based sales forces, making SCD drugs administered in this setting attractive to them, he said.

“This is partly about where money is. The drug companies are going where the money is. It’s not oral drugs to treat everybody, it’s something else,” Dr. Swift said. “So someone else is going to have to fund the basic research” into treatments that could be more broadly used.

Dr. Swift said in an interview that he has received NIH funding for developing SCD-101, an oral drug, for which a placebo-controlled crossover study is underway.

Presenters at the NIH conference, including Dr. Saunthararajah, expressed frustration about what they see as relatively little work being done on SCD despite decades of knowledge about the root causes. Like Dr. Swift, he criticized the approach taken in selecting which treatments advance in this field.

“It’s not being driven by what is the most cost effective, what the patients need the most,” Dr. Saunthararajah said. “It’s driven by what will make the most money, not just for [the] drug company, but also for the hospital and also for the physicians.”

Dr. Saunthararajah reported having patents and patent applications around decitabine/tetrahydrouridine, 5-azacytidine/tetrahydrouridine, and differentiation therapy for oncology. He has also been a consultant for EpiDestiny, Novo Nordisk, and Takeda Oncology. Dr. Williams reported having no relevant financial disclosures. Dr. Swift is a managing member of Invenux and reported equity in Mast Therapeutics and SCD Development.

This article was updated on 11/9/2018.

BETHESDA, MD – While there are experimental treatments such as sevuparin and gene therapy in testing for sickle cell disease (SCD), researchers said this field is lagging because of a lack of funding.

Dr_Microbe/Thinkstock

Yogen Saunthararajah, MD, of the Cleveland Clinic, described what he called a “paltry” landscape of new drugs for SCD at Sickle Cell in Focus, a conference held by the National Institutes of Health.

There are only four main approaches taken by drugs now in clinical testing for addressing the root causes of SCD, despite decades’ worth of research of the genetic and mechanistic underpinnings of this disease, he said.

“It’s pretty sad,” Dr. Saunthararajah said, referring to the quantity of efforts, not their quality.

Within days of his presentation at the conference, one of the drugs he highlighted had officially fallen out of contention. An Oct. 26 post on NIH’s Clinicaltrials.gov site said Incyte had terminated its phase 1 study of INCB059872 in SCD “due to a business decision” not to pursue this indication. Incyte confirmed that it dropped development of INCB059872 for SCD but will continue testing it for other indications, including acute myeloid leukemia (AML).

Dr. Saunthararajah said that work on another approach, using decitabine (Dacogen), for which he has done a phase 1 study, is “struggling,” because of the search for funding.

Two other approaches that Dr. Saunthararajah cited in his presentation appear to remain on track. These are gene therapy and the once-daily voxelotor treatment from Global Blood Therapeutics.

In the field of gene therapy, Sangamo Therapeutics and Sanofi’s Bioverativ in May said the Food and Drug Administration had cleared the way for them to start a phase 1/2 clinical trial for the BIVV003 product that they are developing together. This uses zinc finger nuclease (ZFN) gene-editing technology to modifying a short sequence of the BCL11A gene, with the aim of reactivating fetal hemoglobin.

Bluebird Bio’s LentiGlobin gene therapy has advanced as far as phase 3 for transfusion-dependent beta-thalassemia and phase 1/2 for SCD. In October, the European Medicines Agency accepted the company’s application for approval of LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassemia (TDT) and a non-beta⁰/beta⁰ genotype. The company has not said when it expects to file with the FDA for approval of this treatment.

In the field of oral therapies, Global Blood Therapeutics has said it’s in discussions with the FDA about a potential accelerated approval of voxelotor. The tablet is meant to inhibit the underlying mechanism that causes sickling of red blood cells. In June, the company completed a planned review of early data from its phase 3 trial, known as the HOPE study.

“On the primary endpoint (the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline), a statistically significant increase was demonstrated with voxelotor at both the 1,500-mg and 900-mg doses after 12 weeks of treatment versus placebo,” Global Blood Therapeutics said in an August regulatory filing with the Securities and Exchange Commission.

The company has said that voxelotor may meet the FDA’s standard for accelerated approval under Subpart H program.

In his presentation at the NIH conference, Tom Williams, MD, PhD, of KEMRI/Wellcome Trust Research Programme highlighted a recent review of experimental SCD treatments by Marilyn Jo Telen, MD, of Duke University, Durham, N.C. (Blood. 2016;127[7]:810-9).

Also among the drugs being tested for SCD is Modus Therapeutics’ sevuparin, which Dr. Williams described as being “a heparin-like molecule without the anticoagulant complications.”

The compound originated as a “passion project” of Mats Wahlgren, MD, PhD, of the Karolinska Institute, Stockholm, who developed it for the treatment of malaria, Dr. Williams said. The company that’s developing sevuparin, Modus Therapeutics, is moving it forward first as a treatment for SCD for commercial reasons, Dr. Williams said.

“They think it’s a better first target,” he said.

An ongoing study of sevuparin for painful crisis is expected to be completed in December, with data then expected to be released in the middle of 2019, Ellen K. Donnelly, PhD, chief executive officer of Modus Therapeutics, said in an interview. She cited a mix of scientific, medical and commercial reasons for her company’s decision to advance sevuparin in SCD.

“First, and most importantly, there is proof of clinical benefit of a similar molecule (the low-molecular-weight heparin called tinzaparin) in patients with sickle cell disease,” Dr. Donnelly said. “Unfortunately, there is a bleeding risk with tinzaparin that limits use of the agent for the treatment of sickle cell disease. Sevuparin does not have the bleeding risk and thus is a strong candidate for SCD.”

Dr. Donnelly also noted the emphasis that the FDA’s Division of Hematology Products has put on development of therapeutics for SCD as a reason for proceeding first with this indication. The agency and the American Society of Hematology in early October held a workshop of experts, physicians, patients, and industry collaborators focused on identifying new endpoints for clinical studies.

Still, she noted that commercial reasons did factor into this decision.

“[I]t is possible for a small company like Modus to take an asset all the way to market when developing a therapeutic for a rare disease given the need for fewer patients and smaller trials,” Dr. Donnelly wrote. “As you can see from www.clinicaltrials.gov, we were able to run our phase 2 study with a small number of sites. In addition, in SCD it is possible to get approval with only one or two confirmatory studies.”
 

Financial interests

Other drugs in testing for SCD include rivipansel from GlycoMimetics, for which Pfizer is leading development. The sponsors expect to complete the so-called RESET trial by 2019, according to the clinicaltrials.gov. In this study, which is intended to enroll 350 participants, patients who have vaso-occlusive crises are randomly selected for treatment with either rivipansel or placebo.

Speaking during a question-and-answer session at the NIH conference, Robert Swift, PhD, said there’s a need for inexpensive oral drugs to treat SCD. Many other options will remain beyond the finances of people living in poor countries, he said.

“We need to focus not only on the root cause, but on something that is oral and inexpensive to solve the greater sickle cell problem,” Dr. Swift said.

Large drugmakers already have hospital-based sales forces, making SCD drugs administered in this setting attractive to them, he said.

“This is partly about where money is. The drug companies are going where the money is. It’s not oral drugs to treat everybody, it’s something else,” Dr. Swift said. “So someone else is going to have to fund the basic research” into treatments that could be more broadly used.

Dr. Swift said in an interview that he has received NIH funding for developing SCD-101, an oral drug, for which a placebo-controlled crossover study is underway.

Presenters at the NIH conference, including Dr. Saunthararajah, expressed frustration about what they see as relatively little work being done on SCD despite decades of knowledge about the root causes. Like Dr. Swift, he criticized the approach taken in selecting which treatments advance in this field.

“It’s not being driven by what is the most cost effective, what the patients need the most,” Dr. Saunthararajah said. “It’s driven by what will make the most money, not just for [the] drug company, but also for the hospital and also for the physicians.”

Dr. Saunthararajah reported having patents and patent applications around decitabine/tetrahydrouridine, 5-azacytidine/tetrahydrouridine, and differentiation therapy for oncology. He has also been a consultant for EpiDestiny, Novo Nordisk, and Takeda Oncology. Dr. Williams reported having no relevant financial disclosures. Dr. Swift is a managing member of Invenux and reported equity in Mast Therapeutics and SCD Development.

This article was updated on 11/9/2018.

BETHESDA, MD – While there are experimental treatments such as sevuparin and gene therapy in testing for sickle cell disease (SCD), researchers said this field is lagging because of a lack of funding.

Dr_Microbe/Thinkstock

Yogen Saunthararajah, MD, of the Cleveland Clinic, described what he called a “paltry” landscape of new drugs for SCD at Sickle Cell in Focus, a conference held by the National Institutes of Health.

There are only four main approaches taken by drugs now in clinical testing for addressing the root causes of SCD, despite decades’ worth of research of the genetic and mechanistic underpinnings of this disease, he said.

“It’s pretty sad,” Dr. Saunthararajah said, referring to the quantity of efforts, not their quality.

Within days of his presentation at the conference, one of the drugs he highlighted had officially fallen out of contention. An Oct. 26 post on NIH’s Clinicaltrials.gov site said Incyte had terminated its phase 1 study of INCB059872 in SCD “due to a business decision” not to pursue this indication. Incyte confirmed that it dropped development of INCB059872 for SCD but will continue testing it for other indications, including acute myeloid leukemia (AML).

Dr. Saunthararajah said that work on another approach, using decitabine (Dacogen), for which he has done a phase 1 study, is “struggling,” because of the search for funding.

Two other approaches that Dr. Saunthararajah cited in his presentation appear to remain on track. These are gene therapy and the once-daily voxelotor treatment from Global Blood Therapeutics.

In the field of gene therapy, Sangamo Therapeutics and Sanofi’s Bioverativ in May said the Food and Drug Administration had cleared the way for them to start a phase 1/2 clinical trial for the BIVV003 product that they are developing together. This uses zinc finger nuclease (ZFN) gene-editing technology to modifying a short sequence of the BCL11A gene, with the aim of reactivating fetal hemoglobin.

Bluebird Bio’s LentiGlobin gene therapy has advanced as far as phase 3 for transfusion-dependent beta-thalassemia and phase 1/2 for SCD. In October, the European Medicines Agency accepted the company’s application for approval of LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassemia (TDT) and a non-beta⁰/beta⁰ genotype. The company has not said when it expects to file with the FDA for approval of this treatment.

In the field of oral therapies, Global Blood Therapeutics has said it’s in discussions with the FDA about a potential accelerated approval of voxelotor. The tablet is meant to inhibit the underlying mechanism that causes sickling of red blood cells. In June, the company completed a planned review of early data from its phase 3 trial, known as the HOPE study.

“On the primary endpoint (the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline), a statistically significant increase was demonstrated with voxelotor at both the 1,500-mg and 900-mg doses after 12 weeks of treatment versus placebo,” Global Blood Therapeutics said in an August regulatory filing with the Securities and Exchange Commission.

The company has said that voxelotor may meet the FDA’s standard for accelerated approval under Subpart H program.

In his presentation at the NIH conference, Tom Williams, MD, PhD, of KEMRI/Wellcome Trust Research Programme highlighted a recent review of experimental SCD treatments by Marilyn Jo Telen, MD, of Duke University, Durham, N.C. (Blood. 2016;127[7]:810-9).

Also among the drugs being tested for SCD is Modus Therapeutics’ sevuparin, which Dr. Williams described as being “a heparin-like molecule without the anticoagulant complications.”

The compound originated as a “passion project” of Mats Wahlgren, MD, PhD, of the Karolinska Institute, Stockholm, who developed it for the treatment of malaria, Dr. Williams said. The company that’s developing sevuparin, Modus Therapeutics, is moving it forward first as a treatment for SCD for commercial reasons, Dr. Williams said.

“They think it’s a better first target,” he said.

An ongoing study of sevuparin for painful crisis is expected to be completed in December, with data then expected to be released in the middle of 2019, Ellen K. Donnelly, PhD, chief executive officer of Modus Therapeutics, said in an interview. She cited a mix of scientific, medical and commercial reasons for her company’s decision to advance sevuparin in SCD.

“First, and most importantly, there is proof of clinical benefit of a similar molecule (the low-molecular-weight heparin called tinzaparin) in patients with sickle cell disease,” Dr. Donnelly said. “Unfortunately, there is a bleeding risk with tinzaparin that limits use of the agent for the treatment of sickle cell disease. Sevuparin does not have the bleeding risk and thus is a strong candidate for SCD.”

Dr. Donnelly also noted the emphasis that the FDA’s Division of Hematology Products has put on development of therapeutics for SCD as a reason for proceeding first with this indication. The agency and the American Society of Hematology in early October held a workshop of experts, physicians, patients, and industry collaborators focused on identifying new endpoints for clinical studies.

Still, she noted that commercial reasons did factor into this decision.

“[I]t is possible for a small company like Modus to take an asset all the way to market when developing a therapeutic for a rare disease given the need for fewer patients and smaller trials,” Dr. Donnelly wrote. “As you can see from www.clinicaltrials.gov, we were able to run our phase 2 study with a small number of sites. In addition, in SCD it is possible to get approval with only one or two confirmatory studies.”
 

Financial interests

Other drugs in testing for SCD include rivipansel from GlycoMimetics, for which Pfizer is leading development. The sponsors expect to complete the so-called RESET trial by 2019, according to the clinicaltrials.gov. In this study, which is intended to enroll 350 participants, patients who have vaso-occlusive crises are randomly selected for treatment with either rivipansel or placebo.

Speaking during a question-and-answer session at the NIH conference, Robert Swift, PhD, said there’s a need for inexpensive oral drugs to treat SCD. Many other options will remain beyond the finances of people living in poor countries, he said.

“We need to focus not only on the root cause, but on something that is oral and inexpensive to solve the greater sickle cell problem,” Dr. Swift said.

Large drugmakers already have hospital-based sales forces, making SCD drugs administered in this setting attractive to them, he said.

“This is partly about where money is. The drug companies are going where the money is. It’s not oral drugs to treat everybody, it’s something else,” Dr. Swift said. “So someone else is going to have to fund the basic research” into treatments that could be more broadly used.

Dr. Swift said in an interview that he has received NIH funding for developing SCD-101, an oral drug, for which a placebo-controlled crossover study is underway.

Presenters at the NIH conference, including Dr. Saunthararajah, expressed frustration about what they see as relatively little work being done on SCD despite decades of knowledge about the root causes. Like Dr. Swift, he criticized the approach taken in selecting which treatments advance in this field.

“It’s not being driven by what is the most cost effective, what the patients need the most,” Dr. Saunthararajah said. “It’s driven by what will make the most money, not just for [the] drug company, but also for the hospital and also for the physicians.”

Dr. Saunthararajah reported having patents and patent applications around decitabine/tetrahydrouridine, 5-azacytidine/tetrahydrouridine, and differentiation therapy for oncology. He has also been a consultant for EpiDestiny, Novo Nordisk, and Takeda Oncology. Dr. Williams reported having no relevant financial disclosures. Dr. Swift is a managing member of Invenux and reported equity in Mast Therapeutics and SCD Development.

This article was updated on 11/9/2018.

BETHESDA, MD. – Obtaining an extended red cell antigen profile before a patient’s first transfusion can help improve outcomes and avoid complications in highly transfused patients, according to one researcher.

“We strongly feel that you should get an extended red cell type on the first encounter” for patients facing long-term transfusion support, Connie Westhoff, PhD, of the New York Blood Center, said at Sickle Cell in Focus, a conference held by the National Institutes of Health. “This can be a one-time test. It doesn’t have to be repeated.”

She also stressed the importance of ensuring that this information travels with patients, who may be seen at various hospitals. “One of the challenges here is making this part of the patient’s electronic medical record.”

Alloimmunization has been a major concern for chronically transfused patients, so there’s a real advantage to knowing a patient’s extended red cell antigen profile before the patient develops alloantibodies following transfusion, Dr. Westhoff said. Hemolytic transfusion reactions can sometimes destroy patients’ own RBCs in addition to the transfused RBCs. Having the patient’s profile to identify the potential cause of the incompatibility and guide transfusion support in an emergency can be lifesaving.

The implementation of genotyping by DNA-based methods has brought down the cost of this screening, making it no longer a barrier in care.


Dr. Westhoff cited a study she and her colleagues published on a study typing patients who have sickle cell disease (Transfusion. 2015 Jun;55[6 Pt 2]:1388-93). In that paper, they found that DNA-based RBC typing provided improved accuracy and expanded information on RBC antigens, compared with hemagglutination methods. This led to its implementation as the primary method for extended RBC typing for patients with sickle cell disease at the Children’s Hospital of Philadelphia.

About 65%-70% of antibodies drop to levels that are not detectable by routine assays, further demonstrating the need for DNA-based methods. The drugs used to dampen the immune response in many of these patients may also hinder or impact detection of antibodies that remain at levels that continue to cause in vivo hemolysis, Dr. Westhoff said in an interview.

For patients with sickle cell disease in many Western countries, antigen matching for CEK, at a minimum, is routine. The United States is now moving in this direction.

“Modern transfusion practice is moving to knowing what antigens the patient is at risk to become immunized against. ... What antigens does the patient lack and what antibodies could the patient make,” Dr. Westhoff said in an interview. “It’s a major advantage in your transfusion service to expedite work-ups and patient care by having that extended antigen profile.”

Dr. Westhoff reported no relevant financial disclosures.

BETHESDA, MD. – Obtaining an extended red cell antigen profile before a patient’s first transfusion can help improve outcomes and avoid complications in highly transfused patients, according to one researcher.

“We strongly feel that you should get an extended red cell type on the first encounter” for patients facing long-term transfusion support, Connie Westhoff, PhD, of the New York Blood Center, said at Sickle Cell in Focus, a conference held by the National Institutes of Health. “This can be a one-time test. It doesn’t have to be repeated.”

She also stressed the importance of ensuring that this information travels with patients, who may be seen at various hospitals. “One of the challenges here is making this part of the patient’s electronic medical record.”

Alloimmunization has been a major concern for chronically transfused patients, so there’s a real advantage to knowing a patient’s extended red cell antigen profile before the patient develops alloantibodies following transfusion, Dr. Westhoff said. Hemolytic transfusion reactions can sometimes destroy patients’ own RBCs in addition to the transfused RBCs. Having the patient’s profile to identify the potential cause of the incompatibility and guide transfusion support in an emergency can be lifesaving.

The implementation of genotyping by DNA-based methods has brought down the cost of this screening, making it no longer a barrier in care.


Dr. Westhoff cited a study she and her colleagues published on a study typing patients who have sickle cell disease (Transfusion. 2015 Jun;55[6 Pt 2]:1388-93). In that paper, they found that DNA-based RBC typing provided improved accuracy and expanded information on RBC antigens, compared with hemagglutination methods. This led to its implementation as the primary method for extended RBC typing for patients with sickle cell disease at the Children’s Hospital of Philadelphia.

About 65%-70% of antibodies drop to levels that are not detectable by routine assays, further demonstrating the need for DNA-based methods. The drugs used to dampen the immune response in many of these patients may also hinder or impact detection of antibodies that remain at levels that continue to cause in vivo hemolysis, Dr. Westhoff said in an interview.

For patients with sickle cell disease in many Western countries, antigen matching for CEK, at a minimum, is routine. The United States is now moving in this direction.

“Modern transfusion practice is moving to knowing what antigens the patient is at risk to become immunized against. ... What antigens does the patient lack and what antibodies could the patient make,” Dr. Westhoff said in an interview. “It’s a major advantage in your transfusion service to expedite work-ups and patient care by having that extended antigen profile.”

Dr. Westhoff reported no relevant financial disclosures.

BETHESDA, MD. – Obtaining an extended red cell antigen profile before a patient’s first transfusion can help improve outcomes and avoid complications in highly transfused patients, according to one researcher.

“We strongly feel that you should get an extended red cell type on the first encounter” for patients facing long-term transfusion support, Connie Westhoff, PhD, of the New York Blood Center, said at Sickle Cell in Focus, a conference held by the National Institutes of Health. “This can be a one-time test. It doesn’t have to be repeated.”

She also stressed the importance of ensuring that this information travels with patients, who may be seen at various hospitals. “One of the challenges here is making this part of the patient’s electronic medical record.”

Alloimmunization has been a major concern for chronically transfused patients, so there’s a real advantage to knowing a patient’s extended red cell antigen profile before the patient develops alloantibodies following transfusion, Dr. Westhoff said. Hemolytic transfusion reactions can sometimes destroy patients’ own RBCs in addition to the transfused RBCs. Having the patient’s profile to identify the potential cause of the incompatibility and guide transfusion support in an emergency can be lifesaving.

The implementation of genotyping by DNA-based methods has brought down the cost of this screening, making it no longer a barrier in care.


Dr. Westhoff cited a study she and her colleagues published on a study typing patients who have sickle cell disease (Transfusion. 2015 Jun;55[6 Pt 2]:1388-93). In that paper, they found that DNA-based RBC typing provided improved accuracy and expanded information on RBC antigens, compared with hemagglutination methods. This led to its implementation as the primary method for extended RBC typing for patients with sickle cell disease at the Children’s Hospital of Philadelphia.

About 65%-70% of antibodies drop to levels that are not detectable by routine assays, further demonstrating the need for DNA-based methods. The drugs used to dampen the immune response in many of these patients may also hinder or impact detection of antibodies that remain at levels that continue to cause in vivo hemolysis, Dr. Westhoff said in an interview.

For patients with sickle cell disease in many Western countries, antigen matching for CEK, at a minimum, is routine. The United States is now moving in this direction.

“Modern transfusion practice is moving to knowing what antigens the patient is at risk to become immunized against. ... What antigens does the patient lack and what antibodies could the patient make,” Dr. Westhoff said in an interview. “It’s a major advantage in your transfusion service to expedite work-ups and patient care by having that extended antigen profile.”

Dr. Westhoff reported no relevant financial disclosures.

Photo courtesy of GSK

Results from the phase 3 SUPPORT trial suggest there is no role for the combination of eltrombopag and azacitidine in patients with intermediate- or high-risk myelodysplastic syndromes (MDS), according to investigators.

Adding eltrombopag to azacitidine worsened platelet recovery, reduced the overall response rate, and did not improve overall or progression-free survival when compared to azacitidine plus placebo.

Investigators had hypothesized that eltrombopag would reduce the thrombocytopenia exacerbated by azacitidine treatment.

Not only was this not the case, but the investigators observed a trend toward increased progression to acute myeloid leukemia (AML) in eltrombopag-treated patients.

An independent monitoring committee recommended the trial be terminated early.

Michael Dickinson, MBBS, of Peter MacCallum Cancer Centre and Royal Melbourne Hospital in Melbourne, Australia, and his colleagues reported results from the trial in Blood.

The investigators conducted the SUPPORT study (NCT02158936) to investigate the efficacy and safety of eltrombopag as platelet supportive care in patients with intermediate- to high-risk MDS and thrombocytopenia who were receiving azacitidine.

Study design

Investigators randomized patients 1:1 to eltrombopag or placebo in combination with azacitidine. Patients had to have at least one platelet count less than 75 x 10⁹/L within 28 days prior to the first azacitidine dose.

Patients received azacitidine subcutaneously at a dose of 75 mg/m² once daily on 7 consecutive days for 28 days.

Patients received eltrombopag or placebo at a starting dose of 200 mg/day (100 mg/day for East Asians), adjusted by 100 mg increments (50 mg increments for East Asians) to a maximum of 300 mg/day (150 mg/day for East Asians).

Patient disposition

Investigators enrolled 356 patients from 30 countries between June 2014 and December 2015, randomizing 179 patients to eltrombopag and 177 to placebo.

Patients were a median age of 70, 66% were male, 83% were white, and 47% had platelet counts between 20 and 50 x 10⁹/L.

At the start of the trial, 19% of patients (n=66) were platelet transfusion-dependent—16% (29/179) in the eltrombopag group and 21% (37/177) in the placebo group.

Treatment exposure

Patients received eltrombopag for a median of 83 days (range, 60 to 148) and placebo for a median of 149 days (range, 8 to 503).

For non-East Asian patients, the mean dose of eltrombopag was 205 mg/day (range, 65 to 293), and the mean dose of placebo was 245 mg/day (range, 107 to 316).

Sixty-eight patients (38%) in the eltrombopag arm received the recommended number of six or more azacitidine cycles, compared to 91 (51%) in the placebo arm.

Two patients in the eltrombopag arm did not receive treatment. Therefore, the safety analyses were conducted on the 177 treated patients in each arm.

Safety

The most common reason for treatment discontinuation was termination of the study. Thirty-two percent of patients in the eltrombopag cohort and 44% in the placebo cohort discontinued for this reason.

Patients had to discontinue eltrombopag or placebo if azacitidine was discontinued, and this occurred in 30% of patients in the eltrombopag group and 26% in the placebo group.

Twenty-two percent of eltrombopag-treated patients discontinued due to adverse events (AEs), compared with 14% in the placebo group.

The most common reasons for azacitidine discontinuation in the eltrombopag and placebo arms, respectively, were study termination (35% and 46%), AEs (25% and 19%), disease progression (15% and 14%), and patient decision (11% and 9%).

AEs with the greatest difference between the eltrombopag and placebo arms, respectively, were febrile neutropenia (31% and 21%), neutropenia (31% and 26%), nausea (31% and 26%), and diarrhea (25% and 14%).

Sixty-three of the 177 AEs in the eltrombopag group were suspected to be treatment-related, compared to 42 of 173 AEs in the placebo group.

One hundred twenty-eight (72%) serious AEs occurred in the eltrombopag arm, compared to 100 (56%) in the placebo arm.

Fifteen percent of the serious AEs were suspected to be related to eltrombopag treatment, compared to 4% of the serious AEs in the placebo group.

At the final analysis, 108 patients had died—57 (32%) in the eltrombopag group and 51 (29%) in the placebo group (hazard ratio [HR] 1.42; 95% CI 0.97, 2.08; nominal P=0.164).

The main causes of death were disease progression (28 in the eltrombopag group and 21 in the placebo group) and sepsis (18 in the eltrombopag group and 13 in the placebo group).

Efficacy

The study’s primary endpoint was the proportion of patients free of platelet transfusions during cycles one to four of azacitidine therapy.

At the final analysis, there were fewer patients in the eltrombopag arm than in the placebo arm who had achieved platelet transfusion independence—16% (28/179) and 31% (55/177), respectively. The odds ratio (OR) was 0.37 (95% CI 0.21, 0.65; two-sided P value=0.001).

Secondary efficacy endpoints included overall survival, disease response, duration of response, progression to AML, progression-free survival, and hematologic improvement.

The investigators pointed out that no formal statistical tests were performed for the secondary endpoints. Therefore, “statistical interpretation should be made with caution,” they wrote.

Overall response by IWG criteria occurred in 20% of patients in the eltrombopag group and 35% of those in the placebo group, according to investigator assessment (OR=0.51; 95% CI 0.30, 0.86; nominal P=0.005).

There was no significant difference in hematologic improvement, overall survival, or progression-free survival between the treatment arms.

The investigators found a higher rate of progression to AML in the eltrombopag arm than in the placebo arm—15% and 9%, respectively (OR=1.59; 95% CI 0.81, 3.14; nominal P=0.079).

They pointed out that these results contrasted with those of recent clinical studies of eltrombopag monotherapy^1,2,3,4 in patients with MDS.

“[T]he findings of this trial were unexpected,” the investigators wrote.

They hypothesized that eltrombopag inhibits the effects of azacitidine when the drugs are given concomitantly. The issue is being studied further with ongoing research, they said.

The authors acknowledged financial support for medical editorial assistance provided by Novartis Pharmaceuticals Corporation.

Dr. Dickinson disclosed relationships with Celgene, Novartis, Janssen, Pfizer, and Roche. Senior study author Uwe Platzbecker, MD, disclosed relationships with Amgen, Celgene, GlaxoSmithKline, and Novartis.

1. Platzbecker U, et al Lancet Haematol. 2015;2(10):e417-e426. DOI: https://doi.org/10.1016/S2352-3026(15)00149-0

2. Oliva EN, et al. Lancet Haematol. 2017;4(3):e127-e136. DOI: https://doi.org/10.1016/S2352-3026(17)30012-1

3. Mittelman M, et al. Lancet Haematol. 2017;5(1):e34-e43. DOI: https://doi.org/10.1016/S2352-3026(17)30228-4

4. Buckstein R. Lancet Haematol. 2015;2(10):e396-e397. DOI: https://doi.org/10.1016/S2352-3026(15)00200-8

Photo courtesy of GSK

Results from the phase 3 SUPPORT trial suggest there is no role for the combination of eltrombopag and azacitidine in patients with intermediate- or high-risk myelodysplastic syndromes (MDS), according to investigators.

Adding eltrombopag to azacitidine worsened platelet recovery, reduced the overall response rate, and did not improve overall or progression-free survival when compared to azacitidine plus placebo.

Investigators had hypothesized that eltrombopag would reduce the thrombocytopenia exacerbated by azacitidine treatment.

Not only was this not the case, but the investigators observed a trend toward increased progression to acute myeloid leukemia (AML) in eltrombopag-treated patients.

An independent monitoring committee recommended the trial be terminated early.

Michael Dickinson, MBBS, of Peter MacCallum Cancer Centre and Royal Melbourne Hospital in Melbourne, Australia, and his colleagues reported results from the trial in Blood.

The investigators conducted the SUPPORT study (NCT02158936) to investigate the efficacy and safety of eltrombopag as platelet supportive care in patients with intermediate- to high-risk MDS and thrombocytopenia who were receiving azacitidine.

Study design

Investigators randomized patients 1:1 to eltrombopag or placebo in combination with azacitidine. Patients had to have at least one platelet count less than 75 x 10⁹/L within 28 days prior to the first azacitidine dose.

Patients received azacitidine subcutaneously at a dose of 75 mg/m² once daily on 7 consecutive days for 28 days.

Patients received eltrombopag or placebo at a starting dose of 200 mg/day (100 mg/day for East Asians), adjusted by 100 mg increments (50 mg increments for East Asians) to a maximum of 300 mg/day (150 mg/day for East Asians).

Patient disposition

Investigators enrolled 356 patients from 30 countries between June 2014 and December 2015, randomizing 179 patients to eltrombopag and 177 to placebo.

Patients were a median age of 70, 66% were male, 83% were white, and 47% had platelet counts between 20 and 50 x 10⁹/L.

At the start of the trial, 19% of patients (n=66) were platelet transfusion-dependent—16% (29/179) in the eltrombopag group and 21% (37/177) in the placebo group.

Treatment exposure

Patients received eltrombopag for a median of 83 days (range, 60 to 148) and placebo for a median of 149 days (range, 8 to 503).

For non-East Asian patients, the mean dose of eltrombopag was 205 mg/day (range, 65 to 293), and the mean dose of placebo was 245 mg/day (range, 107 to 316).

Sixty-eight patients (38%) in the eltrombopag arm received the recommended number of six or more azacitidine cycles, compared to 91 (51%) in the placebo arm.

Two patients in the eltrombopag arm did not receive treatment. Therefore, the safety analyses were conducted on the 177 treated patients in each arm.

Safety

The most common reason for treatment discontinuation was termination of the study. Thirty-two percent of patients in the eltrombopag cohort and 44% in the placebo cohort discontinued for this reason.

Patients had to discontinue eltrombopag or placebo if azacitidine was discontinued, and this occurred in 30% of patients in the eltrombopag group and 26% in the placebo group.

Twenty-two percent of eltrombopag-treated patients discontinued due to adverse events (AEs), compared with 14% in the placebo group.

The most common reasons for azacitidine discontinuation in the eltrombopag and placebo arms, respectively, were study termination (35% and 46%), AEs (25% and 19%), disease progression (15% and 14%), and patient decision (11% and 9%).

AEs with the greatest difference between the eltrombopag and placebo arms, respectively, were febrile neutropenia (31% and 21%), neutropenia (31% and 26%), nausea (31% and 26%), and diarrhea (25% and 14%).

Sixty-three of the 177 AEs in the eltrombopag group were suspected to be treatment-related, compared to 42 of 173 AEs in the placebo group.

One hundred twenty-eight (72%) serious AEs occurred in the eltrombopag arm, compared to 100 (56%) in the placebo arm.

Fifteen percent of the serious AEs were suspected to be related to eltrombopag treatment, compared to 4% of the serious AEs in the placebo group.

At the final analysis, 108 patients had died—57 (32%) in the eltrombopag group and 51 (29%) in the placebo group (hazard ratio [HR] 1.42; 95% CI 0.97, 2.08; nominal P=0.164).

The main causes of death were disease progression (28 in the eltrombopag group and 21 in the placebo group) and sepsis (18 in the eltrombopag group and 13 in the placebo group).

Efficacy

The study’s primary endpoint was the proportion of patients free of platelet transfusions during cycles one to four of azacitidine therapy.

At the final analysis, there were fewer patients in the eltrombopag arm than in the placebo arm who had achieved platelet transfusion independence—16% (28/179) and 31% (55/177), respectively. The odds ratio (OR) was 0.37 (95% CI 0.21, 0.65; two-sided P value=0.001).

Secondary efficacy endpoints included overall survival, disease response, duration of response, progression to AML, progression-free survival, and hematologic improvement.

The investigators pointed out that no formal statistical tests were performed for the secondary endpoints. Therefore, “statistical interpretation should be made with caution,” they wrote.

Overall response by IWG criteria occurred in 20% of patients in the eltrombopag group and 35% of those in the placebo group, according to investigator assessment (OR=0.51; 95% CI 0.30, 0.86; nominal P=0.005).

There was no significant difference in hematologic improvement, overall survival, or progression-free survival between the treatment arms.

The investigators found a higher rate of progression to AML in the eltrombopag arm than in the placebo arm—15% and 9%, respectively (OR=1.59; 95% CI 0.81, 3.14; nominal P=0.079).

They pointed out that these results contrasted with those of recent clinical studies of eltrombopag monotherapy^1,2,3,4 in patients with MDS.

“[T]he findings of this trial were unexpected,” the investigators wrote.

They hypothesized that eltrombopag inhibits the effects of azacitidine when the drugs are given concomitantly. The issue is being studied further with ongoing research, they said.

The authors acknowledged financial support for medical editorial assistance provided by Novartis Pharmaceuticals Corporation.

Dr. Dickinson disclosed relationships with Celgene, Novartis, Janssen, Pfizer, and Roche. Senior study author Uwe Platzbecker, MD, disclosed relationships with Amgen, Celgene, GlaxoSmithKline, and Novartis.

1. Platzbecker U, et al Lancet Haematol. 2015;2(10):e417-e426. DOI: https://doi.org/10.1016/S2352-3026(15)00149-0

2. Oliva EN, et al. Lancet Haematol. 2017;4(3):e127-e136. DOI: https://doi.org/10.1016/S2352-3026(17)30012-1

3. Mittelman M, et al. Lancet Haematol. 2017;5(1):e34-e43. DOI: https://doi.org/10.1016/S2352-3026(17)30228-4

4. Buckstein R. Lancet Haematol. 2015;2(10):e396-e397. DOI: https://doi.org/10.1016/S2352-3026(15)00200-8

Photo courtesy of GSK

Results from the phase 3 SUPPORT trial suggest there is no role for the combination of eltrombopag and azacitidine in patients with intermediate- or high-risk myelodysplastic syndromes (MDS), according to investigators.

Adding eltrombopag to azacitidine worsened platelet recovery, reduced the overall response rate, and did not improve overall or progression-free survival when compared to azacitidine plus placebo.

Investigators had hypothesized that eltrombopag would reduce the thrombocytopenia exacerbated by azacitidine treatment.

Not only was this not the case, but the investigators observed a trend toward increased progression to acute myeloid leukemia (AML) in eltrombopag-treated patients.

An independent monitoring committee recommended the trial be terminated early.

Michael Dickinson, MBBS, of Peter MacCallum Cancer Centre and Royal Melbourne Hospital in Melbourne, Australia, and his colleagues reported results from the trial in Blood.

The investigators conducted the SUPPORT study (NCT02158936) to investigate the efficacy and safety of eltrombopag as platelet supportive care in patients with intermediate- to high-risk MDS and thrombocytopenia who were receiving azacitidine.

Study design

Investigators randomized patients 1:1 to eltrombopag or placebo in combination with azacitidine. Patients had to have at least one platelet count less than 75 x 10⁹/L within 28 days prior to the first azacitidine dose.

Patients received azacitidine subcutaneously at a dose of 75 mg/m² once daily on 7 consecutive days for 28 days.

Patients received eltrombopag or placebo at a starting dose of 200 mg/day (100 mg/day for East Asians), adjusted by 100 mg increments (50 mg increments for East Asians) to a maximum of 300 mg/day (150 mg/day for East Asians).

Patient disposition

Investigators enrolled 356 patients from 30 countries between June 2014 and December 2015, randomizing 179 patients to eltrombopag and 177 to placebo.

Patients were a median age of 70, 66% were male, 83% were white, and 47% had platelet counts between 20 and 50 x 10⁹/L.

At the start of the trial, 19% of patients (n=66) were platelet transfusion-dependent—16% (29/179) in the eltrombopag group and 21% (37/177) in the placebo group.

Treatment exposure

Patients received eltrombopag for a median of 83 days (range, 60 to 148) and placebo for a median of 149 days (range, 8 to 503).

For non-East Asian patients, the mean dose of eltrombopag was 205 mg/day (range, 65 to 293), and the mean dose of placebo was 245 mg/day (range, 107 to 316).

Sixty-eight patients (38%) in the eltrombopag arm received the recommended number of six or more azacitidine cycles, compared to 91 (51%) in the placebo arm.

Two patients in the eltrombopag arm did not receive treatment. Therefore, the safety analyses were conducted on the 177 treated patients in each arm.

Safety

The most common reason for treatment discontinuation was termination of the study. Thirty-two percent of patients in the eltrombopag cohort and 44% in the placebo cohort discontinued for this reason.

Patients had to discontinue eltrombopag or placebo if azacitidine was discontinued, and this occurred in 30% of patients in the eltrombopag group and 26% in the placebo group.

Twenty-two percent of eltrombopag-treated patients discontinued due to adverse events (AEs), compared with 14% in the placebo group.

The most common reasons for azacitidine discontinuation in the eltrombopag and placebo arms, respectively, were study termination (35% and 46%), AEs (25% and 19%), disease progression (15% and 14%), and patient decision (11% and 9%).

AEs with the greatest difference between the eltrombopag and placebo arms, respectively, were febrile neutropenia (31% and 21%), neutropenia (31% and 26%), nausea (31% and 26%), and diarrhea (25% and 14%).

Sixty-three of the 177 AEs in the eltrombopag group were suspected to be treatment-related, compared to 42 of 173 AEs in the placebo group.

One hundred twenty-eight (72%) serious AEs occurred in the eltrombopag arm, compared to 100 (56%) in the placebo arm.

Fifteen percent of the serious AEs were suspected to be related to eltrombopag treatment, compared to 4% of the serious AEs in the placebo group.

At the final analysis, 108 patients had died—57 (32%) in the eltrombopag group and 51 (29%) in the placebo group (hazard ratio [HR] 1.42; 95% CI 0.97, 2.08; nominal P=0.164).

The main causes of death were disease progression (28 in the eltrombopag group and 21 in the placebo group) and sepsis (18 in the eltrombopag group and 13 in the placebo group).

Efficacy

The study’s primary endpoint was the proportion of patients free of platelet transfusions during cycles one to four of azacitidine therapy.

At the final analysis, there were fewer patients in the eltrombopag arm than in the placebo arm who had achieved platelet transfusion independence—16% (28/179) and 31% (55/177), respectively. The odds ratio (OR) was 0.37 (95% CI 0.21, 0.65; two-sided P value=0.001).

Secondary efficacy endpoints included overall survival, disease response, duration of response, progression to AML, progression-free survival, and hematologic improvement.

The investigators pointed out that no formal statistical tests were performed for the secondary endpoints. Therefore, “statistical interpretation should be made with caution,” they wrote.

Overall response by IWG criteria occurred in 20% of patients in the eltrombopag group and 35% of those in the placebo group, according to investigator assessment (OR=0.51; 95% CI 0.30, 0.86; nominal P=0.005).

There was no significant difference in hematologic improvement, overall survival, or progression-free survival between the treatment arms.

The investigators found a higher rate of progression to AML in the eltrombopag arm than in the placebo arm—15% and 9%, respectively (OR=1.59; 95% CI 0.81, 3.14; nominal P=0.079).

They pointed out that these results contrasted with those of recent clinical studies of eltrombopag monotherapy^1,2,3,4 in patients with MDS.

“[T]he findings of this trial were unexpected,” the investigators wrote.

They hypothesized that eltrombopag inhibits the effects of azacitidine when the drugs are given concomitantly. The issue is being studied further with ongoing research, they said.

The authors acknowledged financial support for medical editorial assistance provided by Novartis Pharmaceuticals Corporation.

Dr. Dickinson disclosed relationships with Celgene, Novartis, Janssen, Pfizer, and Roche. Senior study author Uwe Platzbecker, MD, disclosed relationships with Amgen, Celgene, GlaxoSmithKline, and Novartis.

1. Platzbecker U, et al Lancet Haematol. 2015;2(10):e417-e426. DOI: https://doi.org/10.1016/S2352-3026(15)00149-0

2. Oliva EN, et al. Lancet Haematol. 2017;4(3):e127-e136. DOI: https://doi.org/10.1016/S2352-3026(17)30012-1

3. Mittelman M, et al. Lancet Haematol. 2017;5(1):e34-e43. DOI: https://doi.org/10.1016/S2352-3026(17)30228-4

4. Buckstein R. Lancet Haematol. 2015;2(10):e396-e397. DOI: https://doi.org/10.1016/S2352-3026(15)00200-8

PixCell Medical

The U.S. Food and Drug Administration (FDA) has granted 510(k) clearance for PixCell Medical’s HemoScreen™.

This portable hematology analyzer is used to perform a complete blood count at the point of care.

HemoScreen requires a single drop of blood and uses disposable cartridges that provide automatic sample preparation.

HemoScreen can analyze 20 standard complete blood count parameters and produces results within 5 minutes.

Study results suggested that HemoScreen provides results comparable to those of another hematology analyzer, Sysmex XE-2100. This study was published in the Journal of Clinical Pathology in 2016.

“The HemoScreen delivers lab-accurate results,” said Avishay Bransky, PhD, chief executive officer of PixCell Medical.

He added that HemoScreen “would be especially useful” in physicians’ offices, emergency rooms, intensive care units, oncology clinics, and remote locations.

HemoScreen makes use of a technology called viscoelastic focusing, which employs microfluidics and machine vision algorithms to analyze cells.

PixCell Medical

The U.S. Food and Drug Administration (FDA) has granted 510(k) clearance for PixCell Medical’s HemoScreen™.

This portable hematology analyzer is used to perform a complete blood count at the point of care.

HemoScreen requires a single drop of blood and uses disposable cartridges that provide automatic sample preparation.

HemoScreen can analyze 20 standard complete blood count parameters and produces results within 5 minutes.

Study results suggested that HemoScreen provides results comparable to those of another hematology analyzer, Sysmex XE-2100. This study was published in the Journal of Clinical Pathology in 2016.

“The HemoScreen delivers lab-accurate results,” said Avishay Bransky, PhD, chief executive officer of PixCell Medical.

He added that HemoScreen “would be especially useful” in physicians’ offices, emergency rooms, intensive care units, oncology clinics, and remote locations.

HemoScreen makes use of a technology called viscoelastic focusing, which employs microfluidics and machine vision algorithms to analyze cells.

PixCell Medical

The U.S. Food and Drug Administration (FDA) has granted 510(k) clearance for PixCell Medical’s HemoScreen™.

This portable hematology analyzer is used to perform a complete blood count at the point of care.

HemoScreen requires a single drop of blood and uses disposable cartridges that provide automatic sample preparation.

HemoScreen can analyze 20 standard complete blood count parameters and produces results within 5 minutes.

Study results suggested that HemoScreen provides results comparable to those of another hematology analyzer, Sysmex XE-2100. This study was published in the Journal of Clinical Pathology in 2016.

“The HemoScreen delivers lab-accurate results,” said Avishay Bransky, PhD, chief executive officer of PixCell Medical.

He added that HemoScreen “would be especially useful” in physicians’ offices, emergency rooms, intensive care units, oncology clinics, and remote locations.

HemoScreen makes use of a technology called viscoelastic focusing, which employs microfluidics and machine vision algorithms to analyze cells.

Photo by Bill Branson

Denosumab can be effective against osteoporosis caused by transfusion-dependent thalassemia (TDT), according to research published in Blood Advances.

Researchers found that patients who received twice-yearly injections of denosumab experienced a significant increase in bone density and reduction in bone pain.

“Not only is denosumab associated with improved bone health and reduced pain, but its ease of administration may very well make this drug superior to bisphosphonates for the treatment of osteoporosis in patients with TDT and osteoporosis,” said study author Evangelos Terpos, MD, of the National and Kapodistrian University of Athens in Greece.

For this phase 2b study, Dr. Terpos and his colleagues evaluated 63 patients with TDT and osteoporosis.

They were randomized (in a double-blinded fashion) to receive 60 mg of denosumab (n=32) or placebo (n=31) on days 0 and 180 of a 12-month period. Patients in both arms also received daily supplements of calcium and vitamin D.

Baseline characteristics were largely similar between the treatment arms.

However, the mean value of bone-specific alkaline phosphatase (bALP) was significantly lower in the placebo arm than the denosumab arm—68.48 IU/L and 85.45 IU/L, respectively (P=0.013).

And the mean value of the tartrate-resistant acid phosphatase isoform-5b (TRACP-5b) marker was significantly higher in the denosumab arm than in the placebo arm—0.42 IU/L and 0.16 IU/L, respectively (P=0.026).

Results

The researchers measured bone mineral density in the L1-L4 lumbar spine, the wrist, and the femoral neck.

At 12 months, the mean increase in L1-L4 bone mineral density was 5.92% in the denosumab arm and 2.92% in the placebo arm (P=0.043).

The mean decrease in wrist bone mineral density was -0.26% and -3.92%, respectively (P=0.035).

And the mean increase in femoral neck bone mineral density was 4.08% and 1.96%, respectively (P=0.870).

Patients in the denosumab arm had a significant reduction in bone pain at 12 months, according to the McGill-Melzack scoring system and Huskisson’s visual analog scale (P<0.001 for both).

However, there was no significant change in pain for patients in the placebo arm (P=0.356 with Huskisson’s and P=0.768 with McGill-Melzack).

At 12 months, patients in the denosumab arm had experienced a significant reduction from baseline (P<0.001 for all) in several markers of bone remodeling, including:

• Soluble receptor activator of nuclear factor kappa-B ligand (sRANKL)
• Osteoprotegerin (OPG)
• sRANKL/OPG ratio
• C-terminal crosslinking telopeptide of type I collagen (CTX)
• TRACP-5b
• bALP.

There were no significant changes in dickkopf-1 (Dkk-1), sclerostin, or osteocalcin (OC) in the denosumab arm.

In the placebo arm, patients had a significant increase from baseline in several markers of bone remodeling, including sRANKL, OPG, Dkk-1, sclerostin, CTX, TRACP-5b, and bALP (P<0.001 for all). There was no significant change from baseline in the sRANKL/OPG ratio or OC.

In all, there were 17 adverse events (AEs) in 14 patients.

There were three grade 1 AEs in the placebo arm and 11 in the denosumab arm. Most grade 1 AEs in the denosumab arm were test abnormalities, although three were not—headache, diarrhea, and fever.

There were three serious AEs in the denosumab arm as well—pleural effusion (grade 3), atrial fibrillation (grade 3), and supraventricular tachycardia (grade 4). All three of these AEs were considered unrelated to denosumab.

This study was funded by Amgen, which markets denosumab as Xgeva. The authors said they had no competing financial interests.

Photo by Bill Branson

Denosumab can be effective against osteoporosis caused by transfusion-dependent thalassemia (TDT), according to research published in Blood Advances.

Researchers found that patients who received twice-yearly injections of denosumab experienced a significant increase in bone density and reduction in bone pain.

“Not only is denosumab associated with improved bone health and reduced pain, but its ease of administration may very well make this drug superior to bisphosphonates for the treatment of osteoporosis in patients with TDT and osteoporosis,” said study author Evangelos Terpos, MD, of the National and Kapodistrian University of Athens in Greece.

For this phase 2b study, Dr. Terpos and his colleagues evaluated 63 patients with TDT and osteoporosis.

They were randomized (in a double-blinded fashion) to receive 60 mg of denosumab (n=32) or placebo (n=31) on days 0 and 180 of a 12-month period. Patients in both arms also received daily supplements of calcium and vitamin D.

Baseline characteristics were largely similar between the treatment arms.

However, the mean value of bone-specific alkaline phosphatase (bALP) was significantly lower in the placebo arm than the denosumab arm—68.48 IU/L and 85.45 IU/L, respectively (P=0.013).

And the mean value of the tartrate-resistant acid phosphatase isoform-5b (TRACP-5b) marker was significantly higher in the denosumab arm than in the placebo arm—0.42 IU/L and 0.16 IU/L, respectively (P=0.026).

Results

The researchers measured bone mineral density in the L1-L4 lumbar spine, the wrist, and the femoral neck.

At 12 months, the mean increase in L1-L4 bone mineral density was 5.92% in the denosumab arm and 2.92% in the placebo arm (P=0.043).

The mean decrease in wrist bone mineral density was -0.26% and -3.92%, respectively (P=0.035).

And the mean increase in femoral neck bone mineral density was 4.08% and 1.96%, respectively (P=0.870).

Patients in the denosumab arm had a significant reduction in bone pain at 12 months, according to the McGill-Melzack scoring system and Huskisson’s visual analog scale (P<0.001 for both).

However, there was no significant change in pain for patients in the placebo arm (P=0.356 with Huskisson’s and P=0.768 with McGill-Melzack).

At 12 months, patients in the denosumab arm had experienced a significant reduction from baseline (P<0.001 for all) in several markers of bone remodeling, including:

• Soluble receptor activator of nuclear factor kappa-B ligand (sRANKL)
• Osteoprotegerin (OPG)
• sRANKL/OPG ratio
• C-terminal crosslinking telopeptide of type I collagen (CTX)
• TRACP-5b
• bALP.

There were no significant changes in dickkopf-1 (Dkk-1), sclerostin, or osteocalcin (OC) in the denosumab arm.

In the placebo arm, patients had a significant increase from baseline in several markers of bone remodeling, including sRANKL, OPG, Dkk-1, sclerostin, CTX, TRACP-5b, and bALP (P<0.001 for all). There was no significant change from baseline in the sRANKL/OPG ratio or OC.

In all, there were 17 adverse events (AEs) in 14 patients.

There were three grade 1 AEs in the placebo arm and 11 in the denosumab arm. Most grade 1 AEs in the denosumab arm were test abnormalities, although three were not—headache, diarrhea, and fever.

There were three serious AEs in the denosumab arm as well—pleural effusion (grade 3), atrial fibrillation (grade 3), and supraventricular tachycardia (grade 4). All three of these AEs were considered unrelated to denosumab.

This study was funded by Amgen, which markets denosumab as Xgeva. The authors said they had no competing financial interests.

Photo by Bill Branson

Denosumab can be effective against osteoporosis caused by transfusion-dependent thalassemia (TDT), according to research published in Blood Advances.

Researchers found that patients who received twice-yearly injections of denosumab experienced a significant increase in bone density and reduction in bone pain.

“Not only is denosumab associated with improved bone health and reduced pain, but its ease of administration may very well make this drug superior to bisphosphonates for the treatment of osteoporosis in patients with TDT and osteoporosis,” said study author Evangelos Terpos, MD, of the National and Kapodistrian University of Athens in Greece.

For this phase 2b study, Dr. Terpos and his colleagues evaluated 63 patients with TDT and osteoporosis.

They were randomized (in a double-blinded fashion) to receive 60 mg of denosumab (n=32) or placebo (n=31) on days 0 and 180 of a 12-month period. Patients in both arms also received daily supplements of calcium and vitamin D.

Baseline characteristics were largely similar between the treatment arms.

However, the mean value of bone-specific alkaline phosphatase (bALP) was significantly lower in the placebo arm than the denosumab arm—68.48 IU/L and 85.45 IU/L, respectively (P=0.013).

And the mean value of the tartrate-resistant acid phosphatase isoform-5b (TRACP-5b) marker was significantly higher in the denosumab arm than in the placebo arm—0.42 IU/L and 0.16 IU/L, respectively (P=0.026).

Results

The researchers measured bone mineral density in the L1-L4 lumbar spine, the wrist, and the femoral neck.

At 12 months, the mean increase in L1-L4 bone mineral density was 5.92% in the denosumab arm and 2.92% in the placebo arm (P=0.043).

The mean decrease in wrist bone mineral density was -0.26% and -3.92%, respectively (P=0.035).

And the mean increase in femoral neck bone mineral density was 4.08% and 1.96%, respectively (P=0.870).

Patients in the denosumab arm had a significant reduction in bone pain at 12 months, according to the McGill-Melzack scoring system and Huskisson’s visual analog scale (P<0.001 for both).

However, there was no significant change in pain for patients in the placebo arm (P=0.356 with Huskisson’s and P=0.768 with McGill-Melzack).

At 12 months, patients in the denosumab arm had experienced a significant reduction from baseline (P<0.001 for all) in several markers of bone remodeling, including:

• Soluble receptor activator of nuclear factor kappa-B ligand (sRANKL)
• Osteoprotegerin (OPG)
• sRANKL/OPG ratio
• C-terminal crosslinking telopeptide of type I collagen (CTX)
• TRACP-5b
• bALP.

There were no significant changes in dickkopf-1 (Dkk-1), sclerostin, or osteocalcin (OC) in the denosumab arm.

In the placebo arm, patients had a significant increase from baseline in several markers of bone remodeling, including sRANKL, OPG, Dkk-1, sclerostin, CTX, TRACP-5b, and bALP (P<0.001 for all). There was no significant change from baseline in the sRANKL/OPG ratio or OC.

In all, there were 17 adverse events (AEs) in 14 patients.

There were three grade 1 AEs in the placebo arm and 11 in the denosumab arm. Most grade 1 AEs in the denosumab arm were test abnormalities, although three were not—headache, diarrhea, and fever.

There were three serious AEs in the denosumab arm as well—pleural effusion (grade 3), atrial fibrillation (grade 3), and supraventricular tachycardia (grade 4). All three of these AEs were considered unrelated to denosumab.

This study was funded by Amgen, which markets denosumab as Xgeva. The authors said they had no competing financial interests.

BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.

bubaone/DigitalVision Vectors

People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.

“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.

People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.

Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.

She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).

It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.

“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.

One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.

“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”

Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).

Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.

“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.

Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.

BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.

bubaone/DigitalVision Vectors

People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.

“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.

People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.

Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.

She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).

It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.

“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.

One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.

“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”

Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).

Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.

“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.

Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.

BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.

bubaone/DigitalVision Vectors

People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.

“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.

People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.

Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.

She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).

It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.

“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.

One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.

“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”

Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).

Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.

“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.

Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.

BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.

Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).

Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.

“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.

Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.

The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.

In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.

The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.

Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”

Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.

“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.

She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”

The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.

Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.

BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.

Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).

Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.

“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.

Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.

The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.

In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.

The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.

Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”

Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.

“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.

She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”

The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.

Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.

BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.

Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).

Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.

“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.

Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.

The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.

In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.

The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.

Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”

Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.

“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.

She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”

The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.

Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.