Von Willebrand disease is an inherited bleeding disorder affecting about 0.1% of the population. Von Willebrand disease is caused by deficient or defective von Willebrand factor (VWF), which normally facilitates platelet binding to collagen at sites of injury. About 80% of women with von Willebrand disease experience heavy menstrual bleeding (>80 mL of blood loss per cycle), but there is a scarcity of data on effective treatments. Researchers in the U.S. enrolled 36 women (aged 13-45 years) with mild or moderate von Willebrand disease and heavy menstrual bleeding between 2019 and 2021. In a phase 3, open-label crossover trial, 17 women were randomized to receive intravenous recombinant VWF for two menstrual cycles and then tranexamic acid orally (the standard non-hormonal treatment) for two cycles while 19 women were randomized to receive tranexamic acid first and then recombinant VWF. Women were followed for a median of 24 weeks. Both recombinant VWF and tranexamic acid reduced heavy menstrual bleeding without serious adverse events but not to normal levels; tranexamic acid, however, reduced bleeding significantly more than recombinant VWF (p=0.039). Neither drug reduced the frequency of RBC transfusions for these patients. Nevertheless, recombinant VWF may provide another form of treatment when hormones, tranexamic acid or desmopressin are not well tolerated or effective.
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