Hematology News

SAN DIEGO — Patients with severe hemophilia B demonstrated stable therapeutic expression of the factor IX gene over an 8-year period without late toxicities after systemic administration of a gene therapy called scAAV2/8-LP1-hFIXco, according to a study presented at ASH Annual Meeting and Exposition.

Thrombotic events may be frequent among patients with sickle cell disease, according to findings presented at the ASH Annual Meeting and Exposition.

SAN DIEGO — Use of adjuvant low-dose rituximab with plasma exchange reduced incidence of exacerbation and refractory disease among patients with acquired thrombotic thrombocytopenic purpura and severe ADAMTS13 deficiency, according to study results presented at ASH Annual Meeting and Exposition.

SAN DIEGO — An adult with sickle cell disease responded positively to autologous gene therapy, providing the first proof that this approach could be effective for sickle cell disease, according to results of a pilot study presented at ASH Annual Meeting and Exposition.

SAN DIEGO — Adults with transfusion-dependent beta-thalassemia experienced significant reductions in red blood cell transfusion burden with luspatercept, according to results of a randomized, double-blind, placebo-controlled phase 3 study presented at ASH Annual Meeting and Exposition.

SAN DIEGO — Luspatercept significantly reduced the transfusion burden compared with placebo among patients with anemia due to myelodysplastic syndrome, according to results of a randomized, double-blind, placebo-controlled phase 3 study presented during the plenary session of ASH Annual Meeting and Exposition.

SAN DIEGO — Myeloablative and haploidentical hematopoietic stem cell transplantation from parental donors that utilized CD34 enrichment and mononuclear cell addback significantly improved health-related quality of life and neurocognition of high-risk patients with sickle cell disease, according to a study presented at ASH Annual Meeting and Exposition.

SAN DIEGO — Children from sub-Saharan Africa with sickle cell anemia treated with hydroxyurea for 6 months demonstrated reductions in sickle-related clinical events, transfusions and rates of malaria and mortality, according to results of the prospective multicenter REACH trial presented during the plenary session of the ASH Annual Meeting and Exposition.

SAN DIEGO — The use of opioids to treat pain among hospitalized patients with sickle cell disease should still be considered relatively safe despite the national opioid epidemic, according to study results presented at ASH Annual Meeting and Exposition.

Immune tolerance induction with standard or extended half-life recombinant factor VIII after initial treatment with emicizumab appears safe and effective in pediatric patients with hemophilia A and active inhibitors, according to findings presented at the ASH Annual Meeting and Exposition.