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    Poster Networking

    Platelet Disorders, von Willebrand Disease and Thrombotic Microangiopathies

    Poster Networking Session

    PB0840 - Efficacy of prophylaxis with a plasma-derived von Willebrand factor/ factor VIII concentrate in patients with von Willebrand disease: Subgroup analysis by age and VWD type

    Monday, June 26, 2023
    18:30 – 19:30 ET
    Room: Exhibition

      Presenting Author(s)

    • Robert F. Sidonio, Jr., MD, MSc

      Associate Professor of Pediatrics
      Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Department of Pediatrics, Emory University School of Medicine
      Atlanta, Georgia, United States

      • Co-Author(s)

    • Ana Boban, MD, PhD

      Professor of Internal Medicine
      Department of Internal Medicine, University Hospital Centre Zagreb, Zagreb University School of Medicine
      Zagreb, Zagrebacka, Croatia

    • LD

      Leonid Dubey

      Professor
      Communal Nonprofit Enterprise "Western Ukrainian Specialized Children's Medical Center" of Lviv Regional Council
      Lviv, L'vivs'ka Oblast', Ukraine

    • AI

      Adlette Inati

      Professor of Clinical Medicine
      Nini Hospital
      Tripoli, Liban-Nord, Lebanon

    • CK

      Csongor Kiss

      Professor
      University of Debrecen Clinical Center Nagyerdei Campus, Thrombosis and Haemostasis Center
      Debrecen, Hajdu-Bihar, Hungary

    • TL

      Toshko Lissitchkov

      Professor
      "Specialized Hospital for Active Treatment of Haematological Diseases" EAD
      Sofia, Sofiya, Bulgaria

    • LN

      Laszlo Nemes

      Head of Haemostasis Department
      Medical Centre of Hungarian Defence Forces
      Budapest, Budapest, Hungary

    • DN

      Dzmitry Novik

      Head of Haematology Department
      Government Agency "Republican Research Center for Radiation Medicine and Human Ecology",
      Gomel, Homyel'skaya Voblasts', Belarus

    • EP

      Elina Peteva

      Doctor
      Pediatric Clinic of Haematology and Oncology, MHAT, 'St. Marina'
      Varna, Varna, Bulgaria

    • AT

      Ali Taher

      Professor of Medicine
      Department of Internal Medicine, Division of Hematology-Oncology, American University of Beirut Medical Center
      Beirut, Beyrouth, Lebanon

    • MT

      Margarita Timofeeva

      Doctor
      Federal State Budgetary scientific institution Kirov scientific-research institute of Hematology and Blood Transfusion of Federal Medical and Biological Agency"
      Bryansk, Kirov, Russia

    • KV

      Kateryna Vilchevska

      Head of the NCCL Ohmatdet Hemostasis Pathology Center
      “National Specialized Children's Hospital “OKHMATDYT” of the Ministry of Health of Ukraine”, Hemostasis Pathology Center
      Kyiv, Kyyiv, Ukraine

    • VV

      Vladimir Vdovin

      Chief Specialist on Hemophilia-Haematology
      Morozovskaya Children's City Clinical Hospital
      Moscow, Moskva, Russia

    • SW

      Sylvia Werner

      Senior Director Clinical R& D – Haematology
      Octapharma USA
      Paramus, New Jersey, United States

    • SK

      Sigurd Franz Knaub, PhD

      Senior Vice President Clinical R&D – Haematology
      Octapharma AG
      Lachen, Schwyz, Switzerland

    • Claudia Djambas Khayat, MD, MSc (she/her/hers)

      Prof
      Hotel Dieu de France Hospital, Saint Joseph University Beirut
      Beirut, Beyrouth, Lebanon

    Background: Long-term prophylaxis is recommended for patients with von Willebrand disease (VWD) who have a history of severe, frequent bleeds. In previous clinical trials, prophylaxis with a human plasma-derived von Willebrand factor/factor VIII concentrate (wilate®; pdVWF/FVIII) reduced bleeding rates compared with previous on-demand treatment.

    Aims: To assess efficacy and safety of pdVWF/FVIII prophylaxis in patients with VWD.

    Methods: WIL-31, a prospective, non-controlled, international, phase 3 trial (NCT04052698), enrolled patients ≥6 years old of any gender with VWD type 1 (VWF:RCo < 30 IU/dL), type 2 (except 2N) or type 3. During a 6-month run-in study (WIL-29), patients received on-demand treatment and those who experienced ≥6 non-menstrual bleeds, of which ≥2 were treated with a VWF-containing product, were eligible to enter WIL-31. During WIL-31, patients received pdVWF/FVIII prophylaxis (20–40 IU/kg 2–3 times per week) for 12 months. The primary endpoint was >50% reduction in mean total annualized bleeding rate (ABR) during prophylaxis compared with on-demand treatment.

    Results: 33 patients were included in the modified full analysis set; 9 were < 12 years, 22 had VWD type 3. The primary endpoint was met (mean total ABR decreased by 84%). The median treated total and spontaneous ABRs decreased from 22.4 and 16.4 during WIL-29 to 1.0 and 0.0 during WIL-31, respectively. 18 (55%) patients had zero treated spontaneous bleeds during 12 months of prophylaxis. Median treated total ABRs decreased from 24.8 to 1.0 in type 3 patients and from 24.0 to 1.0 in patients < 12 years (Figure 1). The median weekly dose was 58 IU/kg, and at study end, 70% of patients were on twice-weekly dosing. No thrombotic events and no serious adverse events related to pdVWF/FVIII were reported.

    Conclusion(s): Prophylaxis with pdVWF/FVIII was effective and well tolerated in children and adults with VWD of all types.