Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Severe and Nonsevere Patients With Hemophilia A From the Prospective, Multinational, Ongoing HEM-POWR Study.

OBJECTIVES

To assess the effectiveness and safety of damoctocog alfa pegol in patients with severe and nonsevere hemophilia A in the fifth interim analysis of the ongoing HEM-POWR study.

METHODS

HEM-POWR (NCT03932201) is a multinational, Phase 4, prospective observational study. The key objectives were the annualized bleeding rate (ABR) and adverse events.

RESULTS

At data cutoff (July 8, 2024), the safety analysis set and full analysis set (FAS) included 370 and 270 patients, respectively. In the modified FAS (patients with ≥ 90 days of bleed data), the mean (standard deviation; SD) ABR for total bleeds was 2.8 (5.9) 12 months prior to damoctocog alfa pegol initiation with the previous FVIII product and 2.1 (4.8) during the observation period. Bleed protection with damoctocog alfa pegol was maintained across disease severity, age group, BMI group, dosing regimen, and patient inhibitor history. One patient died due to spinal cord ischemia unrelated to the study drug. One patient developed a transient low-titer inhibitor, which resolved without clinical consequence.

CONCLUSIONS

This updated analysis further demonstrated the effectiveness, acceptable safety profile, and tolerability of damoctocog alfa pegol for previously treated patients with severe and nonsevere hemophilia A from adolescence to older age in a real-world setting.