Ultrasound-mediated Therapeutic Gene Transfer for Hemophilia
Integrated efficacy and safety analysis of Phase 2 and 3 studies with glecaprevir/pibrentasvir in patients with a history of bleeding disorders and chronic hepatitis C virus genotype 1–6-infection
Objective: Hepatitis C virus (HCV) infection is a significant health problem for patients with bleeding disorders due to the absence of highly effective HCV screening of blood products prior to the early 1990s. The aim of this…
Patient Satisfaction with US Hemophilia Treatment Centers: National Trends 2017
Objective: Patient satisfaction with healthcare services is a measure of patient centeredness, influences treatment adherence, and increasingly affects reimbursement. In 2018, the US Hemophilia Treatment Center Network (USHTCN)…
Change in cost and units consumed by people with factor VIII and factor IX deficiency after switching from a standard half-life product to an extended half-life product
Objective: To examine whether a difference exists in factor consumption and cost after a person with factor VIII or factor IV switches from a standard half-life (SHL) factor product to an extended half-life (EHL) factor product…
Bypassing agent (BPA) use for the treatment of bleeds in persons with Hemophilia A (PwHA) with inhibitors before and after emicizumab prophylaxis in the HAVEN 1 study
Background: Emicizumab was approved by the FDA in 2017 for routine prophylaxis in PwHA with inhibitors. HAVEN 1, a phase III study in adolescent and adult PwHA with inhibitors, demonstrated that emicizumab prophylaxis…
rFVIIIFc for immune tolerance induction in severe hemophilia A subjects with inhibitors: a follow-up retrospective analysis
Achievement of therapeutic levels of factor VIII activity following gene transfer with valoctocogene roxaparvovec (BMN 270): Long-term efficacy and safety results in patients with severe hemophilia A
Objective: As a single gene disorder of Factor VIII (FVIII), hemophilia A (HA) is an ideal candidate for gene therapy. We present results from an ongoing Phase 1/2 study of valoctocogene roxaparvovec (BMN 270; AAV5-FVIII-SQ) gene…
BAY 94-9027 Maintains Hemostasis During Major Surgery in Adults and Adolescents With Severe Hemophilia A: PROTECT VIII Results
Objective: BAY 94-9027 is an extended–half-life recombinant factor VIII (FVIII) product. Efficacy in maintaining hemostasis during major surgery was evaluated in a subset of patients with severe hemophilia A in the phase 2/3…
Effective Protection for >5 Years With BAY 94-9027 Prophylaxis: PROTECT VIII Extension Trial Interim Results
Objective: In the PROTECT VIII phase 2/3 trial, the extended–half-life recombinant factor VIII product BAY 94-9027 provided effective bleed protection with twice-weekly, every-5th-day, and every-7th-day prophylaxis for 36 weeks.…
Long-term Benefit of BAY 81-8973 Prophylaxis in Children With Severe Hemophilia A: Interim Analysis of the LEOPOLD Kids Extension Study
Objective: BAY 81-8973 (Kovaltry®) is a full-length, unmodified recombinant human factor VIII (FVIII) for prophylaxis and treatment of bleeds in patients with hemophilia A. Safety and efficacy of BAY 81-8973 in children,…