Simoctocog Alfa Safe, Effective for Managing Inhibitors in Hemophilia A
Researchers evaluated whether simoctocog alfa, a fourth-generation recombinant factor VIII product, is safe and effective in patients with severe hemophilia A.
Researchers evaluated whether simoctocog alfa, a fourth-generation recombinant factor VIII product, is safe and effective in patients with severe hemophilia A.
Median age at inhibitor development was 13 years, and the inhibitor-related mortality rate was 2.44 per 1000 person-years.
Long-term follow-up from the clinical trial investigating LCAR-B38M, a BCMA-targeting CAR-T, revealed that approximately three-quarters of patients achieved a complete response.
A multicenter, randomized, parallel group, double-blind, placebo-controlled trial found that canakinumab may be well tolerated by pediatric patients with sickle cell anemia.
Patients with catastrophic antiphospholipid syndrome were more likely to have positive modified Ham assays and rare germline mutations compared with other cohorts.
Mice with thalassemia with ERFE loss exhibited increased osteoblast and osteoclast activity and decreased bone formation.
Mosunetuzumab may offer a survival benefit, particularly in patients who have relapsed after the receipt of CAR-T.
Patients were randomly assigned to receive either cyclophosphamide followed by cyclosporine A or cyclosporine A plus mucophenolic acid following allogeneic HSCT.
Researchers found that the addition of eltrombopag to standard immunosuppression therapy did not provide therapeutic benefit to pediatric patients with SAA.
Though fludarabine and melphalan led to increased treatment-related mortality, the combination also led to decreased relapse incidence compared with fludarabine and busulfan.