What type of study is this?

This is a Human Clinical Trial study (also classified as: Pre-Registered Trial).

What question did this study set out to answer?

To assess the efficacy and safety of pdVWF/FVIII prophylaxis in children under 6 with severe VWD.

December 8, 2025BloodOpen Access

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Multicenter trial shows efficacy and safety of prophylaxis in children under 6 with severe VWD, indicating a promising treatment approach.

To assess the efficacy and safety of pdVWF/FVIII prophylaxis in children under 6 with severe VWD.
Open-label, prospective, non-controlled, multicenter phase 3 study.
Investigated 12 patients under 6 years with severe VWD.
Administered pdVWF/FVIII 2-3 times per week for 12 months.
Mean total annualized bleeding rate was 4.6 in the full analysis set.
No serious adverse events related to treatment were observed.
Pharmacokinetics were within expected ranges.

Jain et al. (2025) studied this question.

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Multicenter trial shows efficacy and safety of prophylaxis in children under 6 with severe VWD, indicating a promising treatment approach.