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

• Published in: European journal of haematology 2024-02, Vol.112 (2), p.286-295
• Main Authors: Reding, Mark T., Álvarez‐Román, María Teresa, Castaman, Giancarlo, Janbain, Maissaa, Matsushita, Tadashi, Meijer, Karina, Schmidt, Kathrin, Oldenburg, Johannes
• Format: Article
• Language: English
• Subjects: Adverse events; bleeding; Dosage; Drug Administration Schedule; Factor VIII - adverse effects; half‐life; Hemophilia; hemophilia A; Hemophilia A - complications; Hemophilia A - drug therapy; Humans; Prophylaxis; Prospective Studies; real‐world; Safety
• ISSN: 0902-4441; 1600-0609
• DOI: 10.1111/ejh.14114

Objectives To assess effectiveness and safety of damoctocog alfa pegol in interim analyses of the ongoing real‐world hemophilia A HEM‐POWR study. Methods HEM‐POWR (NCT03932201) is a multinational Phase 4 prospective observational study. The primary objective was annualized bleeding rate (ABR) in previously treated patients (PTPs) with hemophilia A. Secondary objectives included adverse events and number of affected joints. Results At data cut‐off (August 17, 2022), the safety analysis set included 268 patients and the full analysis set (FAS) included 161 patients. The most common dosing regimen during observation period was prophylaxis (FAS = 158/161, 98.1%) every 3–4 days (twice weekly; FAS = 78/158, 49.4%) and a median (min, max) infusion dose of 37.5 (10, 72) IU/kg. PTPs receiving prophylactic damoctocog alfa pegol have fewer infusions compared with prior treatment. Median total ABR (Q1, Q3) was 0.0 (0.0, 1.8) and mean total ABR (SD) was 2.4 (8.2). The proportion of patients with no affected joints increased between initial visit and follow‐up. No FVIII inhibitors, treatment‐related adverse events, or deaths were reported. Conclusions Damoctocog alfa pegol shows effectiveness and acceptable safety, as well as consistent utilization, in real‐world PTPs with hemophilia A, including in patients with non‐severe hemophilia and those with a history of inhibitors. Please see video for a summary of this study.