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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB0260 - Evaluating Effectiveness and Safety in the Fourth Interim Analysis of the HEM-POWR Study: A Post Hoc Analysis of Patients From Nordic Study Sites

Sunday, June 23, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall

    Presenting Author(s)

  • Pål Andrè Holme, MD, PhD, MBA

    Professor
    Department of Hematology, Oslo University Hospital, Oslo, Norway
    Oslo, Oslo, Norway

    • Co-Author(s)

  • FB

    Fariba Baghaei

    Professor
    Department of Specialist Medicine, Region Västra Götaland, Sahlgrenska University Hospital, Gothenburg, Sweden
    Gothenburg, Vastra Gotaland, Sweden

  • LP

    Lone H. Poulsen

    Professor
    The Haemophilia Center Department of Hematology Aarhus University Hospital Aarhus, Denmark
    Aarhus, Midtjylland, Denmark

  • JA

    Jan Astermark

    Professor, Senior Consultant
    Department of Translational Medicine, Lund University, Malmö, Sweden
    Malmo, Skane Lan, Sweden

Background: Damoctocog alfa pegol (BAY 94-9027) is a factor VIII extended half-life product approved for previously treated patients (PTPs) aged ≥12 years with haemophilia A (HA).

Aims: This post hoc subgroup analysis aims to assess the effectiveness and safety of damoctocog alfa pegol for a subgroup of patients from Nordic study sites.

Methods: HEM-POWR (NCT03932201) is an ongoing, Phase 4 study of PTPs with HA. Annualised bleeding rate (ABR) was the primary endpoint. Data for Danish and Swedish sites are pooled. Patients from Norway were enrolled after the data cut, thus are presented separately. The safety analysis set (SAF) included PTPs with ≥1 dose of study drug. The full analysis set (FAS) included PTPs with a documented dose. Data were collated from physician records through electronic case report form (eCRF) and patient diaries. Ethical approval and informed consent were obtained.

Results: At data cut-off (1 August 2023), for the pooled Danish/Swedish subgroup, 31 patients were included in the SAF and 27 in the FAS; 13 patients were later enrolled from Norway. Most patients were from Denmark (Table). Median (Q1, Q3) observation period was 525.0 days (342.0, 678.0) in the pooled SAF. The majority of Nordic patients across all three countries were on a twice-weekly or every 5-day damoctocog alfa pegol prophylaxis regimen at baseline. Most patients had severe disease. For Denmark and Sweden, mean (SD) total ABR prior to damoctocog alfa pegol initiation was 2.4 (3.0) and during the observation period was 0.9 (1.1) (Figure). Mean (SD) difference during observation period from prior to damoctocog alfa pegol was –1.5 (2.9). In the SAF, no study drug-related TEAEs, new inhibitors or deaths were reported.

Conclusion(s): This analysis of PTPs from Nordic study sites demonstrates the favourable effectiveness and benefit-risk profile of damoctocog alfa pegol in the real world.

Funded by Bayer.