PB0261 - Investigating effectiveness and safety of extended dosing regimens of damoctocog alfa pegol in the real-world HEM-POWR study

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Johannes Oldenburg, MD

Professor, Chairman and Director
University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine
Bonn, Nordrhein-Westfalen, Germany

Co-Author(s)

María Teresa Alvarez-Román, MD, PhD

Head of the Haemostasis Unit
Department of Hematology, University Hospital La Paz, Autonoma University of Madrid, Madrid, Spain
Madrid, Madrid, Spain
Giancarlo Castaman

Medical Director Hematologist
Department of Oncology Careggi University Hospital, Florence, Italy
Florence, Toscana, Italy
MJ

Maissaa Janbain

Associate Professor
Department of Hematology/Oncology, Tulane School of Medicine, New Orleans, Louisiana, USA
New Orleans, Louisiana, United States
Tadashi Matsushita

Professor
Nagoya University Hospital, Nagoya, Japan
Nagoya, Aichi, Japan
Karina Meijer

MD, PhD, professor
Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands
Groningen, Groningen, Netherlands
KS

Kathrin Schmidt

Project Manager Non-Interventional Studies
Bayer, Berlin, Germany
Berlin, Berlin, Germany
MR

Mark T. Reding

Director
Center for Bleeding and Clotting Disorders, University of Minnesota Medical Center
Minneapolis, Minnesota, United States

Background: Damoctocog alfa pegol (BAY 94-9027) is a factor VIII (FVIII) extended half-life product approved for previously treated patients (PTPs) aged ≥12 years with haemophilia A (HA). Prophylaxis regimens include twice weekly, every 5 days (E5D) or every 7 days (E7D); dose and frequency can be adjusted based on the patient's clinical condition.

Aims: HEM-POWR (NCT03932201) is an ongoing, real-world, Phase 4 study of PTPs with HA. This post hoc analysis aims to assess effectiveness and safety of E5D and E7D regimens.

Methods: Annualised bleeding rate (ABR) was the primary endpoint and safety a secondary endpoint. The safety analysis set (SAF) included PTPs with ≥1 dose in the observation period. The full analysis set (FAS) included PTPs with a documented dose in the observation period and bleeding data. Data were collated from physician records and patient diaries. Informed consent and ethical approval was obtained.

Results: At data cut-off (1 August, 2023), 227 patients were included in the FAS. There were 40 (17.6%) patients on E5D and 33 (14.5%) on E7D regimens (Table 1). Most patients were aged ≥18 to < 65 years and had severe disease. On the previous FVIII therapy, the most common regimens were every 2 days for 37.5% of patients in E5D group and twice-weekly treatment for 24.2% in E7D group. Mean (SD) total ABR prior to damoctocog alfa pegol initiation was 4.5 (7.7) for E5D and 3.4 (5.4) for E7D regimen (Table 2). During the observation period, mean (SD) total ABR was 1.1 (1.4) for E5D and 1.6 (3.8) for E7D regimens. There were no study drug-related treatment-emergent adverse events or inhibitors. One death, unrelated to study drug, occurred in E5D group.

Conclusion(s): Results of this interim analysis indicate that effectiveness of damoctocog alfa pegol is maintained with extended dosing regimens in the real world.

Funded by Bayer.