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Efficacy of hFVIII/VWF Concentrate in Pediatric Patients with von Willebrand Disease (VWD): The French Experience

1Department of Hematology, CHU Necker, Paris, France, 2Clinical Hemostasis Unit, CHU Lyon, Lyon, France, 3CRC (Regional Hemophilia Treatment Center), CHU Bicêtre, AP-HP, Le Kremlin Bicêtre, France, 4Haemophilia Treatment Center, Hospital of Simone Veil, Eaubonne, Montmorency, France, 5CHU de Caen, Hematology Laboratory, Caen, France, 6CHRU Nancy, Vandoeuvre Les Nancy, France, 7CRC (Regional Hemophilia Treatment Center), CHU Besançon, Besançon, France, 8CSL Behring, Paris, France, 9Laboratory of Hematology, Haemophilia Center, University Hospital, Nantes, France

Abstract Number: PB0934

Meeting: ISTH 2021 Congress

Theme: Platelet Disorders, von Willebrand Disease and Thrombotic Microangiopathies » VWF and von Willebrand Factor Disorders - Clinical Conditions

Background: This French national observational study (OPALE) evaluated the use of human factor VIII (hFVIII)/von Willebrand factor (VWF) concentrate to prevent and treat bleeding episodes in patients with inherited von Willebrand disease (VWD) in real-life settings.

Aims: To determine the efficacy of hFVIII/VWF concentrate in a French pediatric population.

Methods: Data were collected May 2016 to May 2020 from patients treated with hFVIII/VWF concentrate for prophylaxis, treatment of hemorrhage or surgical bleeding.

Results: Among 116 patients enrolled in the OPALE study by 17 French centers, 19 were pediatric patients (<6 years, n=5 and ≥6–12 years, n=14), including 5 (26.3%) females. In this pediatric cohort, patients were diagnosed with type 1 (26.7%), type 2A (17.2%), type 2M (13.8%), type 2B (12.9%), type 2N (5.2%), type 2B/2N (9.5%), or type 3 (14.7%) VWD. Seven patients received prophylaxis, including four patients previously receiving prophylaxis with another VWF concentrate and two previously treated episodically; one received hFVIII/VWF concentrate as first treatment. Patients received prophylaxis 1×/week (n=2), 2×/week (n=4) or 3×/week (n=1); mean (range) dose per infusion was 85 (62–109) IU/kg, 53 (45–100) IU/kg and 31 IU/kg, respectively. Bleeding events (n=23) were reported in six patients outside of any prophylactic period; events included epistaxis (n=9) in three patients and hemarthrosis (n=2) in one patient. In addition, 9 patients received hFVIII/VWF concentrate during 10 surgical procedures including two tonsillectomies, four adenoidectomies, one tooth extraction, one postectomy, one umbilical hernia repair and one esogastroscopy. Hemostatic efficacy was rated as “excellent” or “good” by investigators for all treatment regimens (prophylaxis, episodic and surgery) in both patients <6 years and ≥6–12 years. No safety concerns have been reported in this study to date.

Conclusions: This study demonstrates that hFVIII/VWF concentrate is effective in the prevention and treatment of bleeding in this pediatric French cohort.

To cite this abstract in AMA style:

Harroche A, Rugeri L, D'Oiron R, Hassoun A, Repesse Y, Frotscher B, Fournel A, Bracquart D, Martin C, Trossaert M, Meunier S. Efficacy of hFVIII/VWF Concentrate in Pediatric Patients with von Willebrand Disease (VWD): The French Experience [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/efficacy-of-hfviii-vwf-concentrate-in-pediatric-patients-with-von-willebrand-disease-vwd-the-french-experience/. Accessed November 28, 2023.

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