Presentation Details
| Fourth Interim Analysis of the HEM-POWR Study: Evaluating Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A in the United States Mark T Reding1, Beng Fuh2, Vanessa Salinas3, Maissaa Janbain4. 1University of Minnesota Medical Center, Minneapolis, MN, USA.2East Carolina University, Greenville, NC, USA.3Center for Inherited Blood Disorders, Orange, CA, USA.4Tulane School of Medicine, New Orleans, LA, USA |
Abstract
Introduction or Background: Damoctocog alfa pegol (Jivi®, BAY 94-9027), an extended half-life, factor VIII product, has been approved since 2018 for previously treated patients (PTPs) aged ≥12 years with hemophilia A (HA) in the United States (US). Prior interim analyses of the ongoing, open-label multinational, prospective cohort HEM-POWR study (NCT03932201) have demonstrated effectiveness and safety of damoctocog alfa pegol. Aims and Objectives: An interim subgroup analysis of the effectiveness and safety of damoctocog alfa pegol in PTPs from US study sites enrolled in the HEM-POWR study. Methods: HEM-POWR included PTPs with severe and non-severe HA receiving damoctocog alfa pegol by any treatment modality. The primary endpoint was annualized bleeding rate (ABR); safety was a secondary endpoint. The safety analysis set (SAF) included PTPs with ≥1 study dose in the observation period. PTPs fulfilling all inclusion criteria with a documented study dose and ≥1 documented infusion during the observation period were included in the full analysis set (FAS). Data were captured from patient diaries and physician records. Statistical analyses were explorative and descriptive. Patients provided informed consent and ethical approval was obtained. Results: At data cut-off (1 August 2023), 31 American PTPs were included in the SAF and 26 in the FAS. The median observation period was over 2.5 years (Table). In the FAS, 92.3% of patients were aged ≥18 to <65 years (24/26). The majority of patients (61.5%, 16/26) had severe HA, 19.2% (5/26) had moderate, and 7.7% (2/26) had mild disease (data missing for 3 patients); 88.5% (23/26) were on a prophylactic regimen prior to enrolment (1 missing). The most common dosing regimens were twice weekly then every 5 days at baseline (11/24, 45.8%; 8/24, 33.3%), follow-up window 1 (11/23, 47.8%; 9/23, 39.1%), and follow-up window 2 (10/23, 43.5%; 9/23, 39.1%), respectively. The most common concomitant diseases were a positive test for Hepatitis C virus (6/26, 23.1%) or HIV (5/26, 19.2%), and hypertension (5/26, 19.2%). The median (Q1, Q3); mean (SD) total ABR prior to damoctocog alfa pegol was 3.0 (1.0, 4.0); 5.3 (9.5) and during observation period was 1.1 (0.4, 2.9); 3.7 (8.0) (Figure); the mean (SD) total difference was –1.9 (11.4). The mean (SD) difference from prior to damoctocog alfa pegol to during observation period for spontaneous bleeds was –2.7 (5.5), for joint bleeds was –1.7 (11.1), and for spontaneous joint bleeds was –2.4 (5.3). In the SAF, 15/31 patients (48.4%) reported treatment-emergent adverse events (TEAEs); none were study drug-related and 1 (3.2%) led to a change of treatment regimen. There were 7 (22.6%) patients who reported serious TEAEs, 1 (3.2%) led to a change of treatment regimen. No new inhibitor development or deaths were reported. Conclusions: The results of this subgroup analysis of HEM-POWR continue to support the favorable effectiveness and safety profile of damoctocog alfa pegol in PTPs with severe and non-severe HA. The results provide insights into real-world clinical practice in the US and inform US stakeholders. Funded by Bayer.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.