Recent guidelines from the American Society of Hematology (ASH) and other groups conditionally recommend long-term prophylaxis to prevent excess bleeding for patients with von Willebrand disease (VWD), specifically in those with a history of severe and frequent bleeds.1 This contrasts with typical treatment for most patients with the condition, who are treated on-demand as needed for acute bleeding or trauma, sometimes with short-term prophylaxis to prevent bleeding in a surgical setting. However, information about the cost-benefit of long-term bleeding prophylaxis versus on-demand treatment has been lacking, making it difficult to come to a consensus on the best way to manage prophylaxis in all patients.2
A study presented at the European Hematology Association (EHA) 2022 Congress used data from Sweden and the U.K.3 to shed light on this issue. The researchers, including presenter Michele Wilson, PhD, of RTI Health Solutions in North Carolina, sought to analyze the cost effectiveness of on-demand treatment versus long-term bleeding prophylaxis strategies in patients with low, medium, or high rates of annual bleeds.
Dr. Wilson and colleagues compared the estimated costs in patients receiving on-demand treatment versus long-term prophylaxis; they also contrasted the estimated life years and quality-adjusted life years (QALYs) of patients under these approaches. To do this, the team analyzed the potential inpatient and outpatient resource use via country-specific sourcing. They also estimated the risk of bleeds and probability of joint surgery in these two groups using previously published prophylaxis studies. For long-term prophylaxis, estimates were made based on administering plasma-derived von Willebrand factor (VWF) and factor VIII (either in a ratio of 2.4:1 or 1:1, depending on the specific commercial product used).
In analyses using patients with medium rates of annual bleeds, the authors found long-term regimens were cost-effective when viewed from a lifetime perspective. In analyses from U.K. data (using the 2.4:1 product), the long-term approach saved an estimated £831,000 per patient, with an improvement of 6.14 QALYs.
The Swedish analysis of the same product showed similar results, with the long-term prophylaxis approach saving an estimated 8,842,000 krona and yielding an improvement of 6.52 QALYs. Results were similar in comparisons performed on patients with high annual bleed rates in both countries.
Using probabilistic sensitivity methods to quantify confidence in these analyses, long-term treatment using the 2.4:1 product was cost-effective in 95% or more of simulations in both countries. Additionally, as a result of differences in product costs, treatment via the 2.4:1 product was less expensive compared to treatment with the 1:1 product, in both on-demand and long-term prophylaxis approaches in the two countries.
The authors concluded long-term prophylaxis with plasma-derived VWF/factor VIII (2.4:1) is a cost-effective approach for patients at high or medium annual risk of bleeds. As long-term prophylaxis also appears to improve patients’ quality of life, clinicians may want to consider it even in patients who do not carry the most severe bleeding risks.
Any conflicts of interest by the authors were made public at the time of presentation.
References
- Connell NT, Flood VH, Brignardello-Petersen R, et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease. Blood Adv. 2021;5(1):301-325.
- Miesbach W, Berntorp E. Translating the success of prophylaxis in haemophilia to von Willebrand disease. Thromb Res. 2021;199:67-74.
- Wilson M, Castaman G, Escolar Ginés, et al. Assessing the cost-effectiveness of long-term prophylaxis strategies in von Willebrand disease. Abstract S297. Presented at the European Hematology Association (EHA) 2022 Congress, June 12, 2022; Vienna, Austria.
