PB0661 - Real-world Effectiveness and Safety of Damoctocog Alfa Pegol in Canadian Patients with Hemophilia A: Interim Results from the HEM-POWR Study

Monday, June 26, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Davide Matino, MD (he/him/his)

Assistant Professor
Department of Medicine, Division of Hematology, McMaster University, Hamilton, ON
Hamilton, Ontario, Canada

Co-Author(s)

AI

Alfonso Iorio

Professor
Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON
Hamilton, Ontario, Canada
Anthony KC. Chan, MBBS

Professor
McMaster University, McMaster Children’s Hospital, Hamilton, Canada
Hamilton, Ontario, Canada
NS

Nasrin Samji

Assistant Professor, Pediatrics
McMaster Children’s Hospital, Hamilton, Canada
Hamilton, Ontario, Canada

Background: Damoctocog alfa pegol is an extended half-life PEGylated recombinant Factor VIII product approved in Canada for previously treated patients (PTPs) aged ≥12 years with hemophilia A. Its efficacy and safety have been demonstrated previously (Reding, 2017; Reding ISTH 2022). HEM-POWR is an ongoing international study of real-world effectiveness and safety of damoctocog alfa pegol.

Aims: To explore effectiveness and safety of damoctocog alfa pegol in a subgroup of Canadian patients from the HEM-POWR study (NCT03932201).

Methods: In these subgroup analyses, primary endpoint was annualized bleeding rate (ABR). Secondary endpoints included joint health and safety. PTPs with hemophilia A receiving damoctocog alfa pegol with any treatment modality were eligible. Statistics were descriptive. Data were collated from patient e-diaries and physician records. Ethical approval was obtained for all sites.

Results: At data cut-off (17 August 2022), the full analysis set included 19 patients, including 1/19 (5.3%) pediatric patient. Patients were observed for a mean (SD) 345.9 (131.0) days. A total of 18/19 patients received prophylactic treatment prior to the study (1 missing); 14/19 (73.7%) were pre-treated with damoctocog alfa pegol (Table 1). Median (mean, SD) number of bleeds in the overall population (N=19) was 1.0; (0.6, 0.6). Total ABR, median (mean, SD) was 1.5 (1.8, 1.1) for the 6 patients available at assessment time point; overall change in ABR during the observation period compared with prior to damoctocog alfa pegol initiation was −1.9 (−5.1, 9.9) (Table 2). Treatment-emergent adverse events (TEAEs) occurred in 8/19 (42.1%) patients; 3/19 (15.8%) had serious TEAEs. There was 1 serious TEAE that led to change of treatment regimen. No study drug-related TEAEs or discontinuations were reported.

Conclusion(s): This interim analysis of HEM-POWR in Canadian patients demonstrates the real-world effectiveness of damoctocog alfa pegol prophylaxis in reducing ABR compared to prior prophylactic treatment with an acceptable safety profile.

Funded by Bayer.