What is the evidence surrounding prophylaxis in VWD (specifically type 3)?¶
Answer: Unlike hemophilia, prophylaxis is not yet universally established as standard of care in VWD. Expert consensus and guidelines suggest prophylaxis for patients with "severe and frequent" bleeding — but there is no accepted definition of "severe and frequent."
- Common indications in our practice: joint bleeding/disease, recurrent severe oral mucosal bleeding, GI bleeding (usually least responsive), and heavy menstrual bleeding. Where possible we enroll patients in clinical trials.
- Retrospective data: a small body of retrospective evidence shows prophylaxis reduces the annualized bleeding rate (ABR) versus no prophylaxis, with the greatest effect for joint bleeding (hemarthrosis) and less effect for mucosal bleeding.
- Type 3 (WIL-31 sub-analysis): plasma-derived VWF/FVIII concentrate (wilate) prophylaxis produced substantial reductions across all bleeding sites versus on-demand treatment — knee and arm muscle bleeds reduced to zero and ankle joint bleeds reduced by >90%.
For guideline and background context, see the ASH ISTH NHF WFH 2021 VWD guidelines and this Nature Reviews Disease Primers review.