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…
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Patient treatment for hemophilia B has come a long way since the introduction of factor IX (FIX) concentrates. In a recent commentary published in the journal Blood and titled “A new era for hemophilia B treatment,” researchers from Oregon Health and Science University and Bloodworks Northwest highlighted recent advances in…
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…
Researchers at several Canadian institutions investigated the tendencies of health-related quality of life (HRQoL) among teenagers and young adults with hemophilia A (HA) and found that young adults demonstrated more joint disease and worse HRQoL when physical function and pain were considered. The research paper, “Generic and disease-specific quality of…
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…
Prophylactic hemophilia treatment can save patients with severe forms of the disease from the long-term consequences of joint damage and other complications. A report by Dr. Suchitra S. Acharya, MD, of Hofstra Northwell School of Medicine in New York, gives a detailed overview of the current state and future perspectives…
In addition to its clinical challenges, hemophilia is characterized by a major economic burden that affects patients, care givers, the healthcare system and society. In a recent report, Sheh-Li Chen, PharmD, from the University of North Carolina Medical Center, identifies the most significant areas of healthcare hemophilia management, highlighting the importance of the…
The European Medicines Agency (EMA) is considering lowering the minimum number of patients required in clinical trials of potential hemophilia treatments because of “dramatic” increases in hemophilia research and concerns about the availability of patients for rare diseases like hemophilia A. “EMA began the process after becoming concerned that the minimum…
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