Plain language summary on “Interim analyses of the multinational real-world prospective cohort HEM-POWR study evaluating the effectiveness and safety of damoctocog alfa pegol in patients with hemophilia A”

Abstract

Hemophilia A is a genetic disorder caused by severe or partial deficiency of factor VIII, an essential protein that forms blood clots. People with hemophilia can bleed during day-to-day activities, mostly occurring in joints. Controlling bleeding after injury or surgery is also challenging. Treatments, such as damoctocog alfa pegol, involve infusing factor VIII products into a vein to help blood clot, prevent (prophylaxis) or stop bleeding. Damoctocog alfa pegol has been studied in clinical trials of severe hemophilia A, but studies in normal clinical settings are needed as they better represent real-world patients. In a recent publication in the European Journal of Hematology, researchers studied the effectiveness and safety of damoctocog alfa pegol in a normal clinical setting (HEM-POWR). The primary focus was how treatment affects the number of bleeds a person has in a year, including joint bleeds. Unfavorable treatment reactions (adverse events) were also recorded; these describe how safe the treatment is. The HEM-POWR study found that people had fewer bleeds, including joint bleeds, on damoctocog alfa pegol compared to previous treatments. No new adverse events were reported. This research shows that damoctocog alfa pegol treatment is effective and safe in people with mild/moderate and severe hemophilia.