Efficacy and safety of a recombinant Von Willebrand Factor treatment in patients with inherited Von Willebrand Disease requiring surgical procedures

Autor:	Philippe Beurrier, Nicolas Drillaud, Marc Trossaert, Olivier Feugeas, Emmanuelle de Raucourt, Anamaria Callegarin, Claire Flaujac, Yoann Pailler, Fabienne Volot, Dominique Desprez, Vincent Cussac, Brigitte Pan-Petesch
Rok vydání:	2021
Předmět:	medicine.medical_specialty 030204 cardiovascular system & hematology Gastroenterology Loading dose Hemostatics law.invention 03 medical and health sciences 0302 clinical medicine Von Willebrand factor law hemic and lymphatic diseases Internal medicine von Willebrand Factor Von Willebrand disease Humans Medicine In patient Adverse effect Desmopressin Genetics (clinical) Retrospective Studies Hemostasis Factor VIII biology business.industry Hematology General Medicine Surgical procedures medicine.disease von Willebrand Diseases Recombinant DNA biology.protein business 030215 immunology medicine.drug
Zdroj:	Haemophilia. 27:270-276
ISSN:	1365-2516 1351-8216
Popis:	Introduction Von Willebrand Disease is a common inherited haemorrhagic disorder due to a deficiency of Von Willebrand Factor (VWF). In case of surgical procedures in patients who are not responsive or have contraindications to desmopressin, replacement therapy with VWF concentrates is indicated. Until recently, only plasma-derived VWF concentrates were available. A new recombinant VWF (rVWF) concentrate that contains no Factor VIII (FVIII) but a high amount of high molecular weight VWF multimers has been available in France since 2018. Aim Describe real-world experience of using rVWF in surgical procedures. Methods Sixty-three surgeries for 55 patients were retrospectively analysed in 7 French haemostasis centres. Results During minor surgeries, the median (range) number of infusions was 1 (1-8) with a preoperative loading dose of 35 (19-56) rVWF IU/kg and a total median dose of 37.5 IU (12-288). During major surgeries, the median (range) number of infusions was only 3 (1-14) with a median preoperative loading dose of 36 IU (12-51) rVWF IU/kg, and a total median dose of 108 IU (22-340) rVWF IU/kg. The overall clinical efficacy was qualified as excellent/good in 61 of the procedures (97%), moderate in 1 (1.5%) and poor in 1 (1.5%). There was no accumulation of VWF or FVIII during postoperative monitoring. No thromboembolic events, anti-VWF antibodies or adverse events were reported. Conclusion This French 'real-world' experience shows that a few infusions and low doses of rVWF provided effective prevention of bleeding in major and minor surgeries in inherited VWD, with no clinically significant safety concerns.
Databáze:	OpenAIRE
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