American Society of Hematology, Dec. 5-8

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The 57th American Society of Hematology Annual Meeting and Exposition

The annual meeting of the American Society of Hematology was held from Dec. 5 to 8 in Orlando, Fla., and attracted approximately 22,000 participants from around the world, including hematology researchers as well as clinical practitioners and other health care professionals. The conference featured presentations focusing on the diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems.

In one study, Seth E. Karol, M.D., of the St. Jude Children's Research Hospital in Memphis, Tenn., and colleagues identified inherited genetic variants associated with the development of osteonecrosis, a chemotherapy side effect, in children treated for acute lymphoblastic leukemia who were under the age of 10 at the time of diagnosis.

The investigators performed a genome-wide association study (GWAS) involving 1,186 children (82 patients with osteonecrosis and 287 without the condition). The investigators found that patients with osteonecrosis were more likely to have genetic variants tied to BMP7, which is important for bone development, and PROX1, which is important for fat cell development.

"Variants in BMP7 and other genes important for fat cell development and bone development increase the risk of osteonecrosis in this population," Karol said. "This study improves our understanding of how osteonecrosis develops and may, in the future, lead to better diagnosis and treatment of this side effect."

One author disclosed financial ties to Sigma Tau, Jazz Pharmaceuticals, Merck, and Spectrum Pharmaceuticals.

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In another study, Mark C. Walters, M.D., of the University of California at San Francisco, and colleagues evaluated the efficacy and safety of a non-communicable virus (LentiGlobin BB305) that was developed to deliver a fully-functioning HBB gene to the blood-producing stem cells of patients with beta-thalassemia. The investigators found that five of nine patients treated with LentiGlobin BB305 did not require a transfusion for up to one year after administration. The other four patients required transfusions but the number was reduced.

"This is a clinically meaningful advance in treating beta-thalassemia major for several reasons. It appears to be safer than traditional bone marrow transplantation, which is the only proven curative treatment for thalassemia, in that none of the patients in this trial had a life-threatening complication," Walters said. "It is potentially available to all patients because it delivers a healthy copy of the globin gene to a patient's own stem cells, thus bypassing the need to find a healthy donor for a transplant. Most importantly, it can reduce and even eliminate red blood cell transfusions in patients with thalassemia major."

Several authors disclosed financial ties to pharmaceutical and medical device companies, including bluebird bio, the developer of LentiGlobin BB305.

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Wendy Landier, Ph.D., of the University of Alabama at Birmingham, and colleagues found that approximately 25 percent of patients with acute lymphoblastic leukemia are over-reporting their daily oral 6-mercaptopurine (6MP) intake during maintenance therapy.

"Over-reporting was more likely in patients who were non-adherent to 6MP, older, of minority race/ethnicity, and from households with lower paternal education," Landier said.

Using data from children enrolled in the Children's Oncology Group AALL03N1 study, the investigators evaluated the differences between self-reported and electronic monitoring of 6MP intake. In comparing self-report to electronic monitoring by study month, the investigators found that 12 percent of patients were perfect reporters (no difference between self-report and electronic monitoring for all study months) and that 24 percent over-reported their 6MP intake (compared to electronic monitoring) by five or more days per month in more than half of the study months.

"The findings suggest that subjective reporting of 6MP intake during maintenance therapy for childhood acute lymphoblastic leukemia is common, particularly in non-adherent patients, and should be used with caution by clinicians," Landier added. "We are currently developing a prediction model that will aid clinicians in identifying patients who are at increased risk for non-adherence to oral 6MP, so that interventions can be targeted to the most vulnerable patients."

Two authors disclosed financial ties to Prometheus Labs, a manufacturer of 6-mercaptopurine.

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ASH: Prasugrel Doesn't Cut Vaso-Occlusive Crisis in Sickle Cell

TUESDAY, Dec. 8, 2015 (HealthDay News) -- The rate of vaso-occlusive crisis events is not significantly lower for children and adolescents with sickle cell anemia receiving prasugrel versus placebo, according to a study published online Dec. 8 in the New England Journal of Medicine. The research was published to coincide with the annual meeting of the American Society of Hematology, held from Dec. 5 to 8 in Orlando, Fla.

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ASH: Venetoclax, Acalabrutinib Active in Relapsed CLL

TUESDAY, Dec. 8, 2015 (HealthDay News) -- Oral venetoclax and acalabrutinib are active in patients with relapsed or refractory chronic lymphocytic leukemia (CLL), according to two studies published online Dec. 7 in the New England Journal of Medicine. The research was published to coincide with the annual meeting of the American Society of Hematology, held from Dec. 5 to 8 in Orlando, Fla.

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ASH: Ibrutinib Bests Chlorambucil in Chronic Lymphocytic Leukemia

MONDAY, Dec. 7, 2015 (HealthDay News) -- For patients with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma, ibrutinib is superior to chlorambucil, according to a study published online Dec. 6 in the New England Journal of Medicine. The research was published to coincide with the annual meeting of the American Society of Hematology, held from Dec. 5 to 8 in Orlando, Fla.

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ASH: Longer Red Blood Cell Unit Storage Noninferior

MONDAY, Dec. 7, 2015 (HealthDay News) -- For children with lactic acidosis due to severe anemia, transfusion with longer-storage red blood cell (RBC) units is noninferior to shorter-storage RBC units, according to a study published online Dec. 5 in the Journal of the American Medical Association. The research was published to coincide with the annual meeting of the American Society of Hematology, held from Dec. 5 to 8 in Orlando, Fla.

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