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 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.
['Goudemand|Jenny|J|', 'Bridey|Françoise|F|', 'Claeyssens|Ségolène|S|', 'Itzhar-Baïkian|Nathalie|N|', 'Harroche|Annie|A|', 'Desprez|Dominique|D|', 'Négrier|Claude|C|', 'Chamouni|Pierre|P|', 'Chambost|Hervé|H|', 'Henriet|Céline|C|', 'Susen|Sophie|S|', 'Borel-Derlon|Annie|A|']
D005169:Factor VIII D005260:Female D005602:France D006801:Humans D040541:Marketing D011446:Prospective Studies D014842:von Willebrand Diseases D014841:von Willebrand Factor
von Willebrand disease von Willebrand factor post-marketing factor VIII clinical trial