The European Medicines Agency (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has initiated a review of factor VIII-containing medicines, to evaluate the risk of inhibitor protein development in patients starting treatment for hemophilia A, according to a press release. This review, which will cover all medicines containing factor VIII authorized in the…

CSL Behring‘s new long-lasting recombinant factor VII single-chain therapy, Afstyla, for adults and children with hemophilia A, is now available nationwide. The drug, approved by the U.S. Food and Drug Administration (FDSA) in May, is the only single-chain product on the market specifically designed for reduction and control of…

A recent clinical trial indicates that replacement therapy with plasma-derived factor VIII is associated with lower incidence for development of inhibitors in patients with severe hemophilia A. The study,“A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A,” was published in The New England Journal of…

  The difference in disease severity between Hemophilia A and B (HA, HB) was recently assessed in the study, “Clinical, Instrumental, Serological And Histological Findings Suggest That Hemophilia B May Be Less Severe Than Hemophilia A,” and published in Haematologica. HA and HB are caused by deficient clotting proteins,…

The safety and efficacy of emicizumab in patients with severe hemophilia A (HA) were recently evaluated in a study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” published in The New England Journal Of Medicine. Hemophilia A is caused by a lack of blood clotting factor…

Chugai Pharmaceutical recently published data of a Phase I clinical study on the antibody emicizumab. The study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” was published in The New England Journal of Medicine. The study revealed possible benefits from emicizumab  for the prevention of bleeding in…

A team led by Lurie Children’s Hospital of Chicago researchers reported a case study of a newborn with combined spontaneous splenic rupture and severe hemophilia A. This baby, whose case is believed to be the first documented, was successfully treated without surgical intervention. Results from the study, “Successful medical management of a…

Researchers at the Centre Hospitalier Metropole Savoie in France identified a rare case of associated myelofibrosis (MF) and acquired hemophilia A (AHA), which they believe to be only the second case reported to date. The study, “Myelofibrosis and acquired hemophilia A: a case report,” was published in the…

Dimension Therapeutics recently revealed positive preclinical results from a novel adeno-associated virus (AAV) that delivers Factor VIII as a therapeutic approach for hemophilia A patients. The results were presented in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” at the 19th American Society of…

Last in a series. Read parts one and two. After our oldest son, Julian, was born and we took him home, the first six months went smoothly. His hemophilia diagnosis came up occasionally, but it didn’t directly affect our quality of life. We later learned that those early…