Safety and efficacy of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A: Results of an interventional, post‐marketing study

摘要

Abstract Introduction Damoctocog alfa pegol (BAY 94‐9027, Jivi?) is an approved extended half‐life factor VIII (FVIII) for treatment of previously treated patients with haemophilia A aged ≥12 years. We report the final results of an interventional, post‐marketing study of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A. Methods In this open‐label, interventional, post‐marketing, phase 4 trial (NCT04085458), previously FVIII‐treated patients with severe haemophilia A aged ≥18 years received damoctocog alfa pegol for ≥100 exposure days (EDs). Patients initially received 45?IU/kg every 5 days (recommended) or 40?IU/kg twice‐weekly. At Visit?3, patients’ doses could be increased, or treatment frequency adapted. The primary endpoint was FVIII inhibitor development (titre ≥.6?Bethesda units). Secondary endpoints included anti‐polyethylene glycol (PEG) antibody development, treatment‐emergent adverse events (AEs) and annualized bleeding rate (ABR). Results Overall, 36 patients were enrolled; 32 patients received treatment, of whom, 27 completed the study. No patients developed FVIII inhibitors; three tested transiently positive for low‐titre anti‐PEG antibodies without clinical relevance. Three patients reported study‐drug‐related AEs of mild or moderate intensity. Two patients discontinued the study due to AEs. No deaths occurred. Most patients (70) were treated with E5D/E7D regimens. The median (Q1;Q3) total ABR (N?=?30) was 3.0 (.0;9.0) pre‐study and 1.8?(.7;5.9) during the study. Conclusion Damoctocog alfa pegol individualized prophylaxis regimens were well‐tolerated with no immunogenicity concerns. ABRs improved following the switch from pre‐study prophylaxis to damoctocog alfa pegol prophylaxis. These results support the favourable safety and efficacy profile of damoctocog alfa pegol prophylaxis.