Patients With Hemophilia A Have More Treatment Options

PATIENTS WITH HEMOPHILIA A have numerous unmet needs and often experience poor quality of life. A virtual symposium presented in conjunction with the Asembia Specialty Pharmacy Summit focused on current and emerging therapies while differentiating therapeutic advantages among current products and management strategies.

Sean DeFrates, PharmD, BCOP, discussed some of the basics of hemophilia A, including disease background, genetics, and the clinical burden. He indicated that hemophilia A starts with an initiation and amplification process, progresses to propagation, and eventually leads to inadequate amounts of thrombin and failure of hemostasis.

Complications of hemophilia A include bleeding into the joints, intramuscular bleeds, transfusion-transmitted infections, and development of inhibitors. DeFrates also underlined the burden of hemophilia A. For these reasons, treatment goals include preventing bleeding, treating events when they occur, accommodating for surgical procedures when necessary, reducing morbidity, and avoiding treatment-related harm. The clinical team employs a few tools that replace clotting factors, including plasma-derived FVIII products, recombinant FVIII products, and bypassing agents.

DeFrates stressed that prophylaxis is the cornerstone of treatment. He also discussed inhibitor development, which occurs in about 30% of patients with hemophilia A and can increase bleeding rates, disability, cost of care, and mortality. He compared and discussed treatment options in detail, starting with extended half-life factor products.

Next, Elizabeth Duruz, RPh, CSP, reviewed nonfactor therapeutic products. She delved into the clinical data for emicizumab, then discussed fitusiran and concizumab similarly. Duruz emphasized that all of the new agents can be given subcutaneously. Emicizumab offers some dosing frequency flexibility, needs no routine coagulation testing, and has not been associated with inhibitor development. Fitusiran’s long half-life allows once-monthly dosing, and is stable at room temperature. Concizumab is administered using an injection device.

The presenters indicated that novel therapies are expected to enter the clinical pipeline within the next decade. It leads to a question: Will factor replacement go by the wayside as improved therapies come to market? Until then, clinical teams are at least able to choose from a number of different strategies, which is a marked improvement from previous decades.