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    Poster Networking

    Hemophilia and Rare Bleeding Disorders

    Poster Networking Session

    PB1296 - A prospective, non-interventional study to investigate the effectiveness of rVIII-SingleChain in patients with hemophilia A

    Tuesday, June 27, 2023
    18:30 – 19:30 ET
    Room: Exhibition

      Presenting Author(s)

    • 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

      • Co-Author(s)

    • SJ

      Sabrina Juranek

      Specialist in paediatrics and youth medicine; Central Emergency Room; Hemostaseology
      Paediatric Thrombosis and Haemostasis Unit, Dr. von Hauner Children´s Hospital LMU Munich, Germany
      Munich, Bayern, Germany

    • OK

      Olga Katsarou

      Director
      Blood Transfusion Centre, National Reference Centre for Congenital Bleeding Disorders, Laiko General Hospital
      Athens, Attiki, Greece

    • SB

      Soraya Benchikh El Fegoun

      Global Medical Director
      CSL Behring, Bern, Switzerland
      Bern, Bern, Switzerland

    • Ingrid Pabinger, MD

      Co-Head of the Thrombosis and Haemostasis Research Group
      Clinical Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
      Vienna, Wien, Austria

    Background: Hemophilia A (HA) is a rare X-linked recessive bleeding disorder characterized by a deficiency in coagulation factor VIII (FVIII), that can result in serious and life-threatening bleeding episodes. B-domain truncated recombinant FVIII (rVIII-SingleChain) is an approved therapy for bleeding in HA.

    Aims: This 12-month interim analysis assessed the effectiveness of rVIII-SingleChain during routine clinical practice across multiple centers in Europe.

    Methods: In a non-interventional, multicentre study, conducted across 6 European countries, patients with HA received rVIII-SingleChain for prophylaxis or on-demand treatment. Participating patients are to be followed up, during routine clinical visits, for up to 3 years. Primary endpoints included annualized bleeding rates (ABR), spontaneous bleeding rates (AsBR), infusion frequency, dosage and hemostatic efficacy.

    Results: Seventy-two patients from Austria (n=9), Belgium (n=4), Germany (n=43), Greece (n=12), Czech Republic (n=2) and Hungary (n=2) were enrolled in the study. Five patients discontinued (none due to lack of efficacy or safety concerns of rVIII-SingleChain). Patient characteristics and effectiveness data are outlined in Table 1 and 2, respectively.
    Overall, 49 patients were on a prophylaxis regimen, the mean interval period for prophylaxis was 2.79 days (range 1.0–7.0). The mean ABR and AsBR for patients on prophylaxis (n=45/49) were 0.82 and 0.52, respectively, and for those on an on-demand regimen (n=8/13), were 1.90 and 0.00, respectively.
    Most bleeding episodes reported across both groups were mild (n=11 and 10, respectively) located mostly in knees and ankles in the prophylaxis group, and within other locations in the on-demand group (Table 2).
    For patients with at least 1 assessment, overall hemostatic effectiveness was either good (26.3%) or excellent (31.6%).Thirteen adverse events were reported in 7 patients, none included inhibitor formation or were related to rVIII-SingleChain.

    Conclusion(s): This interim analysis shows that rVIII-SingleChain is an effective treatment option, with an excellent safety profile, for prophylaxis and on-demand regimens, in routine clinical practice.