Treatment of severe von Willebrand disease with a high‐purity von Willebrand factor concentrate (Wilfactin®): a prospective study of 50 patients

Abstract

) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%) needed a concomitant FVIII dose. Outcome was excellent or good in 89% of the episodes. Forty-four patients underwent 108 surgical or invasive procedures. Outcome was excellent or good in 95 scheduled procedures (only VWF was infused) and 13 emergency procedures (a priming FVIII dose was co-administered with the first VWF infusion). There were no thrombotic complications and none of the 18 patients with type 3 VWD developed anti-VWF or anti-FVIII antibodies. Conclusions. This concentrate safely and effectively provides hemostasis in patients with clinically severe VWD.

Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease

“…[9][10][11][12][13] Several high purity plasma-derived VWF/FVIII products are available on the market that are effective in the treatment and prevention of bleeds in VWF patients. However, plasma-derived products are limited by plasma donor availability, and the theoretical risk of pathogen transmission cannot be excluded despite viral screening and attenuation measures that have been instituted to minimize such concerns.…”

Current issues in diagnosis and treatment of von Willebrand disease

“…Both products also contain plasma derived FVIII, although at different ratios (VWF:FVIII 2.4:1 for Humate‐P and 1:1 for Wilate). In Europe, a plasma‐derived VWF with virtually no FVIII is also available, marketed as Wilfactin 51. The latter product does pose slight challenges for emergency treatment in VWD patients whose FVIII levels are also low, in that emergency treatment likely needs to include both VWF and FVIII.…”

Principles of care for the diagnosis and treatment of von Willebrand disease

“…43 However, as post-infusion levels of FVIII:C rise slowly reaching a peak between 6 and 8 h, co-administration of a priming dose of FVIII may be required if prompt hemostasis is required in patients with baseline FVIII:C levels of 30 U/dL or lower. 44 In patients with high basal levels of FVIII, the concentrate could be useful when prolonged treatment is anticipated (e.g. secondary prophylaxis).…”