While the majority of women with von Willebrand disease (VWD) will experience heavy menstrual bleeding (HMB) often associated negative impacts on overall health and quality of life, available data relevant to effective treatment is limited.

In an effort to better understand viable therapeutic options for this population, a group of researchers investigated the use of intravenously administered recombinant von Willebrand factor (rVWF) replacement therapy vs. tranexamic acid (TA), an antifibrinolytic agent that slows down the breakdown of blood clots. TA is taken orally in tablet form.

The study, “Recombinant von Willebrand factor and Tranexamic Acid for Heavy Menstrual Bleeding in Patients with Mild and Moderate von Willebrand Disease in the USA (VWDMin): A Phase 3, Open-Label, Randomised, Crossover Trial was published in The Lancet Haemtology.

Enrollment occurred at U.S. hemophilia treatment centers between February 12, 2019, and November 16, 2021. 36 female patients with mild or moderate VWD, ages 13 to 45 years. completed the study. All of the participants experienced HMB defined as a pictorial blood assessment chart (PBAC) score of more than 100 in one of the past two cycles. PBACs are widely used to assess HMB in clinical studies. 

17 participants were randomized to receive rVWF for two menstrual cycles and then TA for two cycles, while 19 women were randomized to receive TA first followed by rVWF. Investigators measured efficacy through PBAC scores and participants were followed for a median period of 24 weeks. While both therapies reduced HMB, neither was found to have corrected PBAC scores to the normal range. However, median PBAC scores were significantly lower after two cycles with TA than with rVWF, with scores of 146 vs. 213 respectively. Investigators also reported no serious adverse events or treatment-related deaths.

“These interim data suggest that recombinant VWF is not superior to tranexamic acid in reducing heavy menstrual bleeding in patients with mild or moderate von Willebrand disease, “ concluded the authors. “These findings support discussion of treatment options for heavy menstrual bleeding with patients based on their preferences and lived experience.”

The study was funded by the National Heart Lung Blood Institute of the U.S. National Institutes of Health.

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Source: docwirenews, October 20, 2023