Extended Half-Life Prophylaxis Improves Quality of Life in Hemophilia Patients, Study Shows

The introduction of EHLs allowed hemophilia patients to live a life beyond their disease, with improved quality of life, the study showed.

Data from new studies show that hemophilia A and B patients who switched from on-demand treatment to extended half-life (EHL) prophylaxis saw a positive impact on clinical outcomes, including quality of life improvements and annual bleeding rates.

The data was from joint studies conducted by Sobi and Sanofi. It was presented this morning at the 27th Congress of the International Society on Thrombosis and Hemostasis in Australia. Armin Reininger, head of medical and scientific affairs at Sweden-based Sobi, said the clinical data supports the observation that “the introduction of EHLs has allowed for people with hemophilia to live a life beyond hemophilia, with increased quality of life and a greater ability to engage in sports and activities.”

Introducing EHL factor replacement products has led to patients switching from standard half-life products to EHLs as well as from on-demand treatment to EHL prophylaxis, the companies said this morning. The study showed that people who switched from on-demand treatment to Sobi’s Elocta (prophylactic rFVIIIFc) or Sanofi’s Alprolix (rFIXFc) saw the improvements, including improved joint health and quality of life. Elocta is a recombinant clotting factor therapy developed for hemophilia A and Alprolix is a recombinant clotting factor therapy developed for hemophilia B. Both drugs use Fc fusion technology to prolong circulation in the body.

Data presented by the two companies came from multiple studies, including the A-LONG and ASPIRE (Elocta), and B-LONG and B-YOND (Alprolix) studies. According to the data from the A-LONG and ASPIRE studies, 96% of patients who switched from on-demand to prophylaxis continued prophylaxis for more than six months with a median duration of prophylactic treatment with rFVIIIFc of 4.8 years, the companies said. Joints with pain decreased in 29% of subjects and joint health measured using modified Hemophilia Joint Health Score was improved, as was quality of life in relation to areas such as sports, leisure and physical health.

In the hemophilia B studies, results were similar, with 96% continuing prophylaxis for more than six months with a median duration of prophylactic treatment with rFIXFc of 3.6 years. Quality of life improved, in relation to areas such as sports, leisure and physical health.

Hemophilia A is a genetic bleeding disorder where missing or faulty blood clotting factor VIII prevents blood from clotting normally. Hemophilia B, which is more rare, is caused by insufficient clotting Factor IX. Prophylactic infusions of factor VIII or IX can temporarily replace the clotting factors that are needed to control bleeding and prevent new bleeding episodes. It is estimated that about one in 5,000 males are born with hemophilia A and more rarely in females. Hemophilia B occurs in about one in every 25,000 male births and more rarely in females.

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