PB1123 - Annualised Bleeding Rate and Joint Health: Second Interim Analysis of the Real-world HEM-POWR Study Evaluating the Effectiveness of Damoctocog Alfa Pegol in Patients With Haemophilia A

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

MR

Mark T. Reding

Professor and Director
Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, MN, USA
Minneapolis, Minnesota, United States

Presenting Author(s)

María Teresa Alvarez-Román, MD, PhD

Head of the Thrombosis and Haemostasis Unit
La Paz University Hospital, Madrid, Spain
Madrid, Madrid, Spain

Co-Author(s)

MS

Martin Sanabria

Deputy Director Hematology
Bayer, Basel, Switzerland
Basel, Basel-Stadt, Switzerland
GC

Giancarlo Castaman

Medical Director
Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy
Florence, Toscana, Italy
MJ

Maissaa Janbain

Associate Director
Louisiana Center for Bleeding and Clotting Disorders, Tulane University Medical Center, LA, USA
New Orleans, Louisiana, United States
TM

Tadashi Matsushita

Professor
Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan.
Nagoya, Aichi, Japan
Karina Meijer, MD, PhD

MD, PhD, Internist-haematologist, Professor
Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands
Groningen, Groningen, Netherlands
KS

Kathrin Schmidt

Global Project Manager Non-interventional studies
Bayer, Berlin, Germany
Berlin, Berlin, Germany
Johannes Oldenburg

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

Background: Damoctocog alfa pegol is an extended half-life PEGylated recombinant Factor VIII product for previously treated patients (PTPs) aged ≥12 years with haemophilia A. Efficacy and safety of damoctocog alfa pegol was demonstrated in the pivotal Phase 3 PROTECT VIII trials and their extensions.

Aims: HEM-POWR (NCT03932201) is an ongoing, multicentre Phase IV study to demonstrate the real-world effectiveness and safety of damoctocog alfa pegol in PTPs aged ≥12 years with haemophilia A. Second interim effectiveness analyses are presented.

Methods: PTPs aged ≥12 years with haemophilia A receiving damoctocog alfa pegol with any kind of treatment modality (prophylaxis, intermittent prophylaxis or on-demand) are eligible. Primary endpoints are total bleeding events and annualised bleeding rate (ABR). Data were recorded electronically and patients provided informed consent. Ethical approval was obtained for all sites.

Results: At data cut-off (31 August 2021), 162 patients were enrolled, with 78 patients included in the full analysis set (FAS; 3 mild, 9 moderate, 66 severe disease). In the FAS, mean (standard deviation [SD]) age at enrolment was 35.4 (13.6) years; 71/78 (91.0%) received prophylaxis before enrolment. The median (mean, SD) difference in ABR between the observation period and prior to damoctocog alfa pegol initiation was -0.11 (-1.01, 9.94). Additional data are summarised in Table 1. Joint health was assessed by the number of pre-treatment joint bleeds compared with the number of bleeds at the first follow-up. The number of patients with any affected joint decreased from 37/62 (59.7%) to 8/56 (14.3%). Additional data are presented in Figure 1.

Conclusion(s): These second interim data support the conclusions from the PROTECT clinical trials, demonstrating the effectiveness of damoctocog alfa pegol for reducing ABR and improving joint health in patients with mild, moderate and severe haemophilia A. This study was funded by Bayer.