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

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

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
Maria Teresa Alvarez-Román, MD, PhD

Associate Professor
Department of Hematology, University Hospital La Paz, Autonoma University of Madrid, Madrid, Spain
Madrid, Madrid, Spain
MS

Martin Sanabria

Deputy Director Hematology
Bayer, Basel, Switzerland
Basel, Basel-Stadt, Switzerland
Giancarlo Castaman, MD

Medical Director
Center for Bleeding Disorders and Coagulation, 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

Presenting Author(s)

Karina Meijer, MD, PhD (she/her/hers)

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

Co-Author(s)

KS

Kathrin Schmidt

Global Project Manager Non-interventional studies
Bayer, Berlin, Germany
Berlin, Berlin, Germany
Johannes Oldenburg, MD (he/him/his)

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 B-domain-deleted recombinant FVIII concentrate approved for use in previously treated patients (PTPs) aged ≥12 years with hemophilia A (HA). Efficacy/effectiveness and safety of damoctocog alfa pegol has been demonstrated in the pivotal PROTECT trial and the HEM-POWR study. In the US, recommended treatment regimen is twice weekly. Based on occurrence of bleeding episodes, the regimen can be adjusted to every 5 days (E5D) or further tailored individually as required.

Aims: To explore effectiveness and safety of E5D and every 7 day (E7D) prophylactic damoctocog alfa pegol in the ongoing HEM-POWR study (NCT03932201).

Methods: In this post-hoc subgroup analysis, annualized bleeding rate (ABR) was the primary endpoint; joint health and treatment emergent adverse events (TEAEs) were secondary endpoints.

Results: At data cut-off (August 17, 2022), 54 patients in the full analysis set received E5D (n=31) or E7D (n=23) regimens. In E5D subgroup, median (mean [SD]) total ABR was 0.0 (0.8 [1.2]), and the difference in ABR between observation period and prior to initiation of damoctocog alfa pegol was -1.5 (-4.3 [8.2]). For E7D group, total ABR was 0.0 (1.8 [5.5]), and the difference in ABR was -1.2 (-1.7 [2.2]) (Table 1). The number of affected joints (mean [SD]) decreased from prior to damoctocog alfa pegol initiation (E5D: 1.5 [2.0]; E7D: 1.3 [2.0]) compared with first follow-up (E5D: 0.7 [1.6]; E7D: 0.8 [1.8]) (Figure 1). In the safety analysis set, 15/51 (29.4%) E5D and 5/38 (13.2%) E7D patients experienced TEAEs, resulting in change of treatment regimen for 3 (5.9%) and 3 (7.9%) patients, respectively. There was 1 serious TEAE in each group; no study drug–related TEAEs or discontinuations were reported.

Conclusion(s): Damoctocog alfa pegol demonstrated effectiveness and an acceptable safety profile with extended dosing prophylactic regimens in PTPs with HA.

Funded by Bayer.