ABSTRACT 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. 

Real‐World Effectiveness and Safety of Damoctocog Alfa Pegol in Severe and Nonsevere Patients With Hemophilia A From the Prospective, Multinational, Ongoing <scp>HEM</scp> ‐ <scp>POWR</scp> Study

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.

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.

Introduction Damoctocog alfa pegol (BAY 94-9027) is a PEGylated, extended half-life recombinant factor VIII product, approved for treatment of previously treated patients (PTPs) aged ≥12 years with haemophilia A [1] [2]. Real-world effectiveness and safety of damoctocog alfa pegol has been previously reported in earlier analyses of the HEM-POWR study [3]. Here we present the first safety results from the updated interim analysis. 

Updated interim safety analysis of the real-world HEM-POWR study evaluating damoctocog alfa pegol in previously treated patients with haemophilia A

Interim Subgroup Analysis of the Effectiveness and Safety of Damoctocog Alfa Pegol Prophylaxis in Previously Treated Patients with Hemophilia A Treated Every 5 or Every 7 Days: Results from the Real-World Observational HEM-POWR Study

PB1263 Third Interim Subgroup Analysis of the Effectiveness and Safety of Damoctocog Alfa Pegol in Patients with Hemophilia A Treated Every 5 or Every 7 Days: Results from the Real-World HEM-POWR Study

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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.

Updated interim safety analysis of the real-world HEM-POWR study evaluating damoctocog alfa pegol in previously treated patients with haemophilia A

Interim Subgroup Analysis of the Effectiveness and Safety of Damoctocog Alfa Pegol Prophylaxis in Previously Treated Patients with Hemophilia A Treated Every 5 or Every 7 Days: Results from the Real-World Observational HEM-POWR Study

PB1263 Third Interim Subgroup Analysis of the Effectiveness and Safety of Damoctocog Alfa Pegol in Patients with Hemophilia A Treated Every 5 or Every 7 Days: Results from the Real-World HEM-POWR Study