Preventive Hemophilia A Treatment Reduces Bleeding Events and Frequency of Infusions

CHICAGO — A Rush University Medical Center-led international research team has announced that a treatment to prevent bleeding episodes in children with hemophilia A also is effective for adolescents and adults.

The study was conducted as part of a comprehensive clinical study of ADVATE Antihemophilic Factor (Recombinant), Plasma/Albumin Free Method (rAHF-PFM) to compare the effectiveness of two prophylactic treatment regimens, as well as between on-demand and prophylaxis treatments, in preventing bleeding in previously treated patients with severe or moderately severe hemophilia A. It is the first study designed to generate prospective data for stringent comparisons of bleeding rates.

Hemophilia A is a rare, inherited, potentially deadly blood clotting disorder that affects 400,000 people worldwide, most of them males. Approximately one in 5,000 individuals is born with hemophilia annually. In people with hemophilia A, a protein called clotting factor VIII is either absent or present at low levels. Factor VIII replacement, such as rAHF-PFM, is considered the treatment of choice for managing hemophilia A patients who lack inhibitors (antibodies) of factor VIII.

Patients with severe disease are at particular risk for spontaneous bleeding into joints, muscles and internal organs, as well as trauma-induced bleeding following injury and surgery. Joint bleeding may occur as frequently as 20 to 30 times a year, resulting in clinically significant hemophilia-related arthritis.
“The main goal of replacement therapy is to prevent this pathology,” Valentino said.

In the Rush study, one regimen was based on common practice with every-other-day dosing. The other was customized for each individual based on the drug’s activity in the bod (pharmacokinetics, or PK) with every-third-day dosing. PK-tailored prophylaxis offers an alternative to standard prophylaxis for the prevention of bleeding.

Compared with on-demand treatment, both prophylaxis regimens significantly reduced bleeding, including spontaneous and traumatic hemorrhaging, and improved the quality of life for adolescent and adults patients. Results of the study suggest that prophylaxis is the optimal treatment for patients with severe hemophilia. Data from the study also confirm and extend the safety and effectiveness of rAHF-PFM for controlling and preventing bleeding in the management of hemophilia A.

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