Management of von Willebrand disease with a factor VIII‐poor von Willebrand factor concentrate: Results from a prospective observational post‐marketing study

Background A triple‐secured plasma‐derived von Willebrand factor (pdVWF) almost devoid of factor VIII (FVIII):WILFACTIN®, was approved in France in 2003, and then in other countries for the treatment of patients with von Willebrand disease (VWD). Objective To investigate long‐term safety and efficac...

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Bibliographic Details
Published in:Journal of thrombosis and haemostasis 2020-08, Vol.18 (8), p.1922-1933
Main Authors: Goudemand, Jenny, Bridey, Françoise, Claeyssens, Ségolène, Itzhar‐Baïkian, Nathalie, Harroche, Annie, Desprez, Dominique, Négrier, Claude, Chamouni, Pierre, Chambost, Hervé, Henriet, Céline, Susen, Sophie, Borel‐Derlon, Annie
Format: Article
Language:English
Subjects:
Bleeding
clinical trial
Coagulation factors
Disease prevention
factor VIII
Factor VIII - adverse effects
Female
France
HAEMOSTASIS
Hematology
Human health and pathology
Humans
Life Sciences
Marketing
Mucosa
Original
Patients
post‐marketing
Prophylaxis
Prospective Studies
Risk factors
Surgery
von Willebrand disease
von Willebrand Diseases - diagnosis
von Willebrand Diseases - drug therapy
Von Willebrand factor
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Summary:Background A triple‐secured plasma‐derived von Willebrand factor (pdVWF) almost devoid of factor VIII (FVIII):WILFACTIN®, was approved in France in 2003, and then in other countries for the treatment of patients with von Willebrand disease (VWD). Objective To investigate long‐term safety and efficacy of the product in real‐life over the first 5 post‐approval years. Patients/Methods This prospective, observational, national post‐marketing study (PMS) enrolled patients of all ages and VWD types. Patients were observed for up to 3 years and treated for one or more occasions. Efficacy was assessed for each major event. Breakthrough bleeding rate 3 days post‐infusion and annualized bleeding rate (ABR) were also evaluated for long‐term prophylaxis. Results Overall, 155 of 174 patients enrolled from 31 centers were eligible for efficacy assessment. Most patients (76.8%) were severely affected (VWF:RCo ≤ 15 IU/dL). They were treated for 743 bleeds and 140 surgeries including childbirth. Efficacy outcomes were excellent/good for 98.2% of 56 major surgeries and 94.0% of 67 major bleeds. Approximately 75% of 49 major mucosal bleeds were effectively managed without FVIII co‐administration. In 32 patients receiving prophylaxis, breakthrough bleeding occurred in 1.5% of infusions and median ABR was 1.0 for 20 patients treated ≥ 12 months. Excellent tolerability was confirmed with no safety concerns. No thrombotic events were observed. Conclusions Results from this PMS increase the clinical experience of a FVIII‐poor pdVWF in patients of all ages and VWD types including those with thrombotic risk factors and emphasize that giving FVIII is not always mandatory to effectively treat patients with severe VWD.
ISSN:1538-7933
1538-7836
1538-7836
DOI:10.1111/jth.14928