Global estimation of the bleeding episodes treatable with desmopressin in von Willebrand disease and hemophilia A

Abstract

Desmopressin increases plasma factor VIII and von Willebrand factor in non-severe hemophilia A (Hem A) and type 1 von Willebrand disease (VWD), following intravenous infusions, subcutaneous injections, or nasal spray. This medication, with almost 50 years of clinical experience and proven safety and efficacy, is often unavailable. Thus, there is a need to establish how many patients can benefit from it and how many bleeding episodes can be treated. We reviewed the literature to estimate the global prevalence of symptomatic patients with VWD type 1 and non-severe Hem A, the incidence of bleeds, and the rate of responsiveness to desmopressin. Real-world data indicate that 1.7 patients with VWD per 100,000 seek care in specialized centers. Of them, 1.15 per 100,000 have type 1 VWD and 80% are reported to respond to desmopressin. In type 1 VWD, the estimated frequency of bleeds treatable with desmopressin are 2.5 events per patient year. Non-severe cases are 45% of all Hem A cases, with a much lower incidence of bleeds than in severe disease (0.4 events / patient / year). Based on epidemiology, data on the rates of bleeding, and desmopressin responsiveness, we estimated that 84,000 people with VWD type 1 would benefit from desmopressin to treat 210,000 yearly bleeds, and that 81,000 people with non-severe Hem A can be treated yearly for 13,000 bleeds. Desmopressin is an essential therapeutic tool in non-severe Hem A and type 1 VWD that can treat more than 220,000 bleeding episodes successfully, safely, and economically, also in low-income settings with poor access to costly replacement therapies.