Abstract Number: PB1026
Meeting: ISTH 2020 Congress
Background: Desmopressin is a synthetic hormone used therapeutically in hemophilia A for the induction of an increase of endogenous factor VIII (FVIII). The response to desmopressin shows large inter-individual variation. When effective, desmopressin is an important alternative to the use of FVIII concentrates in the treatment of bleeding. Its benefits are its lower cost and lack of inhibitor risk when compared to FVIII concentrates. Presently we lack comparative information on the use of desmopressin and FVIII concentrates in the treatment of bleeds in non-severe hemophilia A.
Aims: To analyze the use of desmopressin in the treatment of bleeds in patients with non-severe hemophilia A.
Methods: The DYNAMO study is a multicenter cohort study that includes patients with hemophilia A with a baseline FVIII level of 2-35 IU/dL. Data on desmopressin and bleeding episodes over the past 10 years were collected. The study was approved by the medical ethics committee and written consent was obtained (ClinicalTrials.gov Identifier:NCT03623295).
Results: In this preliminary analysis, we included 64 patients with non-severe hemophilia A in whom the desmopressin response was known. The median age was 31 years (IQR 23-47) and the median baseline FVIII level was 13 IU/dL (IQR 6-18). The desmopressin peak response was < 30 IU/dL (inadequate) in 8 (13%) patients, between 30-50 IU/dL (adequate) in 15 (23%) patients and >50 IU/dL excellent in 41 (64%) patients. In total, 195 bleeds required treatment, with 95 (49%) treated with FVIII concentrates, 49 (25%) with desmopressin and 45 (23%) with a combination of both (table.1). Joint and muscle bleeds occurred most frequently (N=110) and were mainly treated with FVIII concentrates (54%) or a combination of FVIII concentrates and desmopressin (30%)(figure 1).
Conclusions: Desmopressin was used either in combination or as solo treatment in 43% of all treated bleeds and should be considered as treatment option in patients with non-severe hemophilia.
|Desmopressin response||Inadequate (n=8)||Adequate (n=15)||Excellent (n=41)||Total (n=64)|
|Total number of bleeds||59||43||88||195|
|FVIII||58 (98%)||16 (37%)||21 (23%)||95 (49%)|
|FVIII + desmopressin||–||11 (26%)||34 (37%)||45 (23%)|
|Desmopressin||–||16 (37%)||33 (35%)||49 (25%)|
[Table 1. Percentage of bleeds treated with desmopressin, FVIII or a combination of both per desmopressin response category.]
To cite this abstract in AMA style:Kloosterman FR, Zwagemaker A, Beckers EAM, van der Bom JG, Coppens M, Collins P, Fischer K, Gouw SC, Hay CRM, Kruip MJHA, van der Meer FJM, Meijer K, Nieuwenhuizen L, Shapiro S, Fijnvandraat K. Use of Desmopressin in the Treatment of Bleeding in Non-severe Haemophilia A [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/use-of-desmopressin-in-the-treatment-of-bleeding-in-non-severe-haemophilia-a/. Accessed December 5, 2021.
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