GlobalResearch and clinical trials
Recombinant von Willebrand factor found effective and safe for up to four years in children with severe VWD
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
A phase 3 study was carried out with 25 children with severe von Willebrand disease. One of the centers was in Samsun. A total of 104 bleeding episodes in the children were successfully stopped with this treatment. Most bleeds were controlled with a single dose. No blood clots or severe allergic reactions were seen, and the effect lasted up to four years.
What does it mean for patients and families?
An option for treating bleeding in children with severe VWD has become stronger. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes. Treatment decisions are made with the doctor.
Source
Scientific paper: Efficacy and safety of recombinant von Willebrand factor in on-demand treatment of children with von Willebrand disease: up to 4 years of phase 3/3b follow-up. Research and Practice in Thrombosis and Haemostasis, 2026. DOI: 10.1016/j.rpth.2026.106817 · PubMed 42633448
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: von Willebrand disease (VWD)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.