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.

Abstract

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.

• Authors: Mark T Reding, María Teresa Álvarez-Román, Giancarlo Castaman, Maissaa Janbain, Tadashi Matsushita, Karina Meijer, Kathrin Schmidt, Johannes Oldenburg
• Journal: European journal of haematology (Eur J Haematol) Vol. 112 Issue 2 Pg. 286-295 (Feb 2024) ISSN: 1600-0609 [Electronic] England
• PMID: 37876046 (Publication Type: Observational Study, Journal Article)
• Copyright: © 2023 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.

Chemical References

• Factor VIII

Topics

• Humans
• Hemophilia A (complications, drug therapy)
• Prospective Studies
• Factor VIII (adverse effects)
• Drug Administration Schedule