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TA More Effective, Affordable Than rVWF for VWD-Related Heavy Menstrual Bleeding

December 11, 2022

Mid-January 2023

Tranexamic acid (TA) may be the more effective and affordable option for reducing heavy menstrual bleeding among women with von Willebrand disease (VWD) compared to recombinant von Willebrand factor (rVWF), according to results of a randomized crossover trial presented at the 64th ASH Annual Meeting and Exposition.

“Of women affected by VWD, more than 80% have heavy menstrual bleeding,” said study presenter Margaret V. Ragni, MD, MPH, of the University of Pittsburgh and Hemophilia Center of Western Pennsylvania. “This is a major public health burden. These women have severe iron deficiency, cognitive defects, psychological problems, and stress related to heavy menstrual bleeding.”

Although guidelines recommend treating VWD-related heavy menstrual bleeding, there is no definitive treatment and data on what to recommend are lacking.

Dr. Ragni and colleagues undertook a multicenter, crossover trial to compare outcomes related to heavy menstrual bleeding in patients treated with rVWF versus TA. Thirty-six women ages 13 to 45 with VWD and heavy menstrual bleeding completed the trial.

Patients were randomly assigned to intravenous rVWF 40 IU/kg on day one or oral TA three times daily on days one to five in each of two consecutive menstrual cycles. Group 1 was assigned rVWF for cycles one and two and then crossed over to TA in cycles three and four. Group 2 was assigned TA in cycles one and two and then crossed over to rVWF in cycles three and four. The primary endpoint was pictorial blood loss assessment chart (PBAC) score.

After two cycles with TA, PBAC was significantly reduced compared with rVWF (225 ± 207 vs. 273 ± 227; p=0.039). In addition, TA was associated with less frequent bleeding (44.4% vs. 59.7%; p=0.014). No differences in cycle severity, cycle length, quality of life, emergency room visits, iron infusion, or days lost from school or work were noted between the two treatment arms.

“With no difference in health care [use] between the arms, it boils down to cost, and rVWF costs significantly more than TA,” Dr. Ragni said. “TA is cost-saving and more effective than rVWF in this study.”

Dr. Ragni pointed out that the trial was not able to enroll the planned 60 patients it needed to fully power the analysis because of the COVID-19 pandemic and trial sites opening and closing.

“Despite not being able to get to the 60 patients, the randomized crossover design enabled successful trial completion,” she said.

Dr. Ragni noted the results point to the critical importance of discussing the best treatment option with each patient.

“We need to have a dialogue. It may be that TA is not the right drug for your patient,” Dr. Ragni said. “These results don’t mean that rVWF is ineffective. It means that women with VWD who have heavy menstrual bleeding have options.”

Any conflicts of interest declared by the authors can be found in the original abstract.


Ragni MV, Rothenberger S, Feldman R, et al. Multicenter, randomized crossover trial comparing recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in von Willebrand disease. Abstract #590. Presented at the 2022 American Society of Hematology Annual Meeting and Exposition; December 11, 2022; New Orleans, Louisiana.


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Mid-January 2023


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