Desmopressin for bleeding in non‐severe hemophilia A: Suboptimal use in a real‐world setting

Abstract

Background Desmopressin is an important treatment option in nonsevere hemophilia A because it has several benefits compared with factor (F) concentrates, including no inhibitor risk and much lower costs. Despite these advantages, data are limited on the real‐world use of desmopressin in the treatment of bleeds. Objective To describe the clinical use of desmopressin in relation to other therapeutic modalities in the treatment of bleeding episodes in patients with nonsevere hemophilia A. Methods Patients with nonsevere hemophilia A aged 12–55 years were included from the DYNAMO cohort study. Data on the desmopressin test response and treated bleeding events in the period January 2009 to July 2020 were retrospectively collected from medical files. An adequate desmopressin test response was defined based on a peak FVIII level of ≥30 IU/dl. Results A total of 248 patients with a median age of 38 years (interquartile range 25–49) were included. An adequate desmopressin test response was documented in 25% and 73% of patients with moderate and mild hemophilia, respectively. In adequate responders, 51% of bleeds were exclusively treated with FVIII concentrates, 24% exclusively with desmopressin, 21% with a combination of both and 4% with other treatments. In 54% of bleeds treated with a single dose of factor concentrates, the expected FVIII level after desmopressin exceeded the level targeted. Conclusion Most bleeds in patients with an adequate response to desmopressin are treated with factor concentrates. These findings may indicate a suboptimal use of desmopressin and that barriers to the use of desmopressin should be explored.

Desmopressin (DDAVP) use in patients with von Willebrand disease: A single‐centre retrospective review of test response and clinical outcomes

“…Moreover, a recent study suggests that there may be underutilization of DDAVP therapeutically in haemophilia. 17 and FVIII trough levels >0.50 IU/mL for surgical prophylaxis. 18 Consequently, we defined sustained response to DDAVP as VWF:Act and FVIII levels ≥ 0.5 IU/mL at 4h post-administration.…”

“…To our knowledge, this study is the first to define DDAVP‐responsiveness in a cohort of patients using various published definitions, suggesting that differences in institutional criteria could result in inconsistent treatment decisions regarding surgical prophylaxis (or management of bleeds) with DDAVP. Moreover, a recent study suggests that there may be underutilization of DDAVP therapeutically in haemophilia 17 . Consequently, this highlights the importance of reaching a consensus definition for biochemical response both for clinical and research purposes.…”

Nonsevere Hemophilia: The Need for a Renewed Focus and Improved Outcomes

“…Desmopressin (1-deamino-8-D-arginine vasopressin [DDAVP]) can produce a clinically important rise in FVIII in PWNSH and may be underused. 41 In those individuals who do not have a contraindication, a trial of DDAVP to assess responsiveness provides useful information for patient management. Nevertheless, most patients require factor concentrate under certain circumstances, for example, major trauma, surgery, head injury.…”

Peri‐operative desmopressin combined with pharmacokinetic‐guided factor VIII concentrate in non‐severe haemophilia A patients

“…Additionally, desmopressin's use is suboptimal in many patients who have an adequate FVIII:C response. 6 Both desmopressin and FVIII concentrates have certain drawbacks.…”

“…In such cases, patients are treated with FVIII concentrate. Additionally, desmopressin's use is suboptimal in many patients who have an adequate FVIII:C response 6 …”