A number of clinical studies have reported that bone marrow-derived cells contribute to the production of clotting factor VIII (FVIII), the molecule linked to hemophilia A. Because the current treatment for this bleeding disorder is associated with some disadvantages and risk, hematopoietic stem cell transplant (HSCT) has become an emerging therapy approach…

In hemophilia A, the most significant therapeutic complication is the development of antibodies against factor VIII (FVIII) that inhibit its coagulant activity and make the therapy ineffective. The plasma-derived porcine FVIII product represents a therapy with low levels of autoantibodies and successful management of bleeding when compared to human plasma-derived products.

CSL Behring’s rVIII-SingleChain, a novel recombinant Factor VIII (rFVIII) treatment candidate, recently demonstrated effectiveness in surgery and in the control of bleeding in patients with severe hemophilia A. The Phase 1 to Phase 3 clinical trial results, recently published in the journal Blood, also demonstrated low annualized bleeding rates in patients…

Hemophilia A patients who receive replacement therapies of factor VIII often develop inhibitors against factor concentrates which hinders the effectiveness of the therapy, increases disability, and reduces quality of life. Although immune tolerance induction (ITI) can reduce inhibitors against factor VIII concentrates, the study “Long-term course of…

Ruptured spleens are rare in newborns, usually occurring secondary to an underlying coagulation disorder, such as hemophilia, or a splenic abnormality. Here, researchers report the first case of a newborn with a spontaneous splenic rupture and severe hemophilia A who was successfully treated without surgical intervention. The case study, “…

Transplants of embryonic stem (ES) cells that secrete the human factor VIII (FVIII) may be a promising therapeutic approach for patients with hemophilia A, according to an animal study conducted by researchers at the Nara Medical University School of Medicine in Japan. The study, “Therapeutic approaches for treating hemophilia…

The association of hemophilia A (AHA) and myelofibrosis, a bone marrow disorder, is uncommon and rarely described but in a recent case study, researchers at the Centre Hospitalier Metropole Savoie, in France, reported a patient with AHA secondary to hematological malignancy whose rapid and challenging diagnosis — and timely treatment — was…

Shire recently announced that the U.S. Food and Drug Administration (FDA) has approved its Baxject III reconstitution system for Adynovate, an approved treatment for Hemophilia A. This system will allow patients and caregivers to prepare the therapy with fewer steps, minimizing the chances for possible errors. Adynovate is a recombinant Factor VIII (rFVIII) treatment with…

BioMarin Pharmaceuticals presented positive interim data from an open-label Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for severe hemophilia A. The promising results, presented at the XXXII International Congress of the World Federation of Hemophilia (WFH), showed that patients had improved and sustained clotting function. BMN 270 is designed to alter…

Biogen and Sobi  announced that they will be presenting updated and long-term data on two approved hemophilia treatments, Eloctate  (marketed as Elocta in Europe) for hemophilia A, and Alprolix for hemophilia B, at the World Federation of Hemophilia (WFH)’s 2016 World Congress next week. Specifically, data from separate Phase 3 extension studies evaluating each treatment’s safety and…