Long-term prophylaxis against bleeds among patients with von Willebrand disease (VWD) was more cost effective than on-demand therapy, according to the results of a study presented at the EHA 2022 Hybrid Congress.

Current clinical practice guidelines recommend long-term prophylaxis with plasma-derived von Willebrand factor (VWF)/factor VIII (FVIII) in a ratio of 2,4:1 or 1:1 for patients with severe or frequent bleeds. The aim of this study was to evaluate the cost-effectiveness of long-term prophylaxis and on-demand therapy among patients with low, medium, and high annual bleed rates (ABR).

The study estimated life years (LYs), quality-adjusted life years (QALYs), and costs of treatment over a lifetime horizon by considering bleed rates and risk of joint surgery among patients with VWF in the United Kingdom (UK) and Sweden. Treatment included long-term prophylaxis or on-demand therapy with plasma-derived VWF/FVIII 2,4:1 or 1:1.

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Long-term prophylaxis was more cost-effective with incremental savings of £831,206 and more effective with an incremental QAL/y of 6.14 compared with on-demand therapy among patients with medium ABR in the UK. Similarly, in Sweden, long-term prophylaxis resulted in a cost savings of 8,841,901 kr and an incremental QALY of 6.52.

The ratio of plasma-derived VWF/FVIII that was most cost-effective for long-term prophylaxis was 2,4:1 compared with 1:1, with savings of £2,307,370 in the UK and 21,939,947 kr in Sweden, and incremental QALYs of 1.56 and 1.42, respectively.

Plasma-derived VWF/FIII 4,2:1 was also resulted in a cost saving for on-demand therapy compared with the 1:1 ratio, with a savings of £1,250,369 and 7,923,741 kr in the UK and Sweden, respectively. The QALYs were equal between product types.

The results among patients with a high ABR were similar. The authors concluded that “these results suggest that long-term prophylaxis with plasma-derived VWF/FVIII 2,4:1 is a cost-effective strategy compared with on-demand therapy, in both the UK and Sweden, for medium and high ABR VWD patients.”

Disclosures: Disclosures are not available for this presentation.


Wilson M, Castaman G, Escolar G, et al. Assessing the cost-effectiveness of long-term prophylaxis strategies in von Willebrand disease. Presented at EHA 2022; June 9-12, 2022. Abstract S297.