AHEAD International and German Studies: Effectiveness, Safety, and Quality of Life Outcomes in Patients with Hemophilia A Treated with Antihemophilic Factor (Recombinant) in a Real-World Setting Over 5 Years

The International and German AHEAD studies are prospective, observational, long-term studies that include patients with moderate or severe (factor VIII <1–5%) hemophilia A who are treated with antihemophilic factor (recombinant; ADVATE) or rurioctocog alfa pegol (ADYNOVATE/ADYNOVI) in routine clinical practice.

These ongoing studies initiated in 2011 and endpoints include joint health outcomes, annualized bleeding rate (ABR), factor consumption, and safety. As of September 2019, the International AHEAD study included 729 patients across 95 active sites in 21 countries and the German AHEAD study included 405 patients at 36 active sites.

Overall, the International cohort is younger than the German cohort (mean ± SD age, 19.0 ± 16.3 years vs. 27.7 ± 17.9 years, respectively). When patients were grouped by disease severity, a numerical difference in average Gilbert scores was observed between prophylactic and on-demand therapy in those with severe hemophilia, but not in those with moderate hemophilia. Notably, this was despite low baseline scores suggesting that these previously treated patients were not severely affected. Among patients receiving prophylaxis, 40 to 44% experienced <1 bleed per year by year 5. Few patients developed de novo FVIII inhibitors and health-related quality of life outcomes were similar during follow-up.

These interim 5-year data are consistent with previous interim reports, confirming long-term effectiveness and safety of ADVATE in patients with moderate or severe hemophilia A in clinical practice.

Publication History

Article published online:
13 November 2020