The World Federation of Hemophilia (WFH) has created a universal case report form for the collection of core data on hemophilic patients in a standardized format that will include relevant diagnosis, therapies, and outcome. Previous WFH forms provided only an overview of the number of patients and access to treatment.

Researchers in India recently reported the case of child with hemophilia and traumatic intracerebellar hemorrhage, who was successfully treated with transfusions of plasma. They recommend a conservative approach as a first line of treatment, before more invasive alternatives such as surgery. The case report, “Intracerebellar haemorrage in a haemophilia child,” was published in…

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…

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…

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…

Genentech recently presented positive Phase 1/2 extension results for its investigational hemophilia A medicine, emicizumab, at the 2016 World Federation of Hemophilia (WFH) World Congress in Orlando, Florida. Emicizumab is being developed by Genentech (a Roche company) and Chugai Pharmaceutical. The latest data analysis continued to show a promising profile…

Researchers have created a model for how factor VIII replacement concentrates behave in the body of hemophilia A patients undergoing surgery, which could allow doctors to calculate more accurate and individualized doses for factor replacement treatment. The study “A Population Pharmacokinetic Model For Perioperative Dosing Of Factor VIII In Hemophilia A…

uniQure N.V. has updated the clinical data from its ongoing clinical trial of a gene therapy for the treatment of patients with hemophilia B. The results indicate sustained improvement by all patients at a low-dose, with durable levels of  Factor IX (FIX) gene activity for several weeks after treatment. The…