Tranexamic acid is not inferior to recombinant von Willebrand Factor (VWF) in alleviating heavy menstrual bleeding in patients with mild-to-moderate von Willebrand disease (VWD), according to interim results of a study published in The Lancet Hematology.
In view of the scarcity of effective therapeutics for heavy menstrual bleeding for women and girls with VWD, researchers conducted a phase 3, open-label, randomized crossover trial to compare recombinant VWF with the standard nonhormonal treatment, tranexamic acid. They recruited patients between ages 13 and 45 years with mild-to-moderate VWD who suffer from heavy menstrual bleeding (defined a pictorial blood assessment chart [PBAC] score of more than 100 in at least 1 of the past 2 menstrual cycles).
A total of 36 participants were randomly assigned in a 1:1 ratio to be in 1 of 2 crossover groups: those first receiving intravenous recombinant VWF at a dose of 40 IU/kg over 5-10 minutes on the first day of menstrual bleeding in cycles 1 and 2 and then oral tranexamic acid at a dose of 1300 mg three times daily in cycles 3 and 4 (n=17), and those that received the reverse (n=19). Participants were followed-up with once at week 16 after cycle 2 and again at week 24 after cycle 4.
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For participants assigned to receive recombinant VWF, a rescue dose of 40 IU/kg was made available to be used at their discretion.
The primary outcome was a 40-point reduction in PBAC score after 2 cycles of treatment, while secondary outcomes were safety, menstrual cycle severity and length, as well as quality of life.
The researchers reported that the primary endpoint “was not met,” indicating that neither therapy corrected PBAC to the desired range. There was no statistically significant difference in menstrual bleeding severity/frequency or quality of life between users of either therapy. Median PBAC scores were significantly lower after 2 cycles with tranexamic acid than with recombinant VWF (146; 95% CI, 117-199; vs 213; 95% CI, 152-298).
Both study drugs were well-tolerated with no reports of severe adverse events (AEs), such as excessively heavy menstrual bleeding, thrombosis, or allergic reactions. The most common grade 1-2 AEs included mucosal bleeding in 4 patients with tranexamic acid treatment vs 0 with recombinant VWF and other bleeding in 4 patients vs 2 patients, respectively.
“As quality of life and health-care use did not differ between treatment groups, and tranexamic acid is less costly per Federal Supply Schedule pricing, it appears to be a more effective approach to heavy menstrual bleeding,” the authors of the study concluded.
Disclosure
This study was funded by the National Heart Lung Blood Institute (National Institutes of Health). Please see the original reference for a full list of disclosures.
Reference
Ragni MV, Rothenberger SD, Feldman R, et al. 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. Lancet Haematol. Published online June 26, 2023. doi:10.1016/S2352-3026(23)00119-9
