Abstract Number: PB0531
Meeting: ISTH 2021 Congress
Background: Desmopressin is used in non-severe hemophilia A to increase endogenous factor VIII (FVIII). Large inter-individual variation in desmopressin response has been recognized and therefore it may not always be effective. Due to its benefits in comparison to FVIII concentrates, including lower costs and lack of inhibitor risk, it is an important alternative treatment modality. Little is known on real world clinical use of desmopressin compared to FVIII concentrates in the treatment of bleeds.
Aims: To describe the clinical use of desmopressin and FVIII concentrate in non-severe hemophilia A.
Methods: The DYNAMO study is an international, multicentre cohort study that includes patients with non-severe hemophilia A (FVIII 2-35 IU/dL) aged 12-55 years. Data were collected on desmopressin test response and treatment of bleeding episodes over the past 10 years. Treatment product was analysed according to desmopressin response and FVIII level. Medical ethics approval and written consent was obtained (ClinicalTrials.gov:NCT03623295).
Results: We included 146 patients with non-severe hemophilia A in this preliminary analysis. The median age was 36 years (IQR 24-49) and the median FVIII level was 11 IU/dL (IQR 5-17). The desmopressin test peak response was <30 IU/dL (inadequate) in 9 (6%) patients, between 30-50 IU/dL (adequate) in 37 (25%) patients and >50 IU/dL (excellent) in 74 (51%) patients. In 26 (18%) patients the desmopressin response was unknown/not tested. Of all bleeds, 594 (74%) were treated with FVIII concentrates, 94 (12%) with desmopressin and 75 (9%) with a combination. Figure 1 and table 1 present treated bleeds per FVIII level and desmopressin response. The majority of bleeds were joint and muscle bleeds (57%) and these were mainly treated with FVIII concentrates (81%) or a combination of FVIII concentrates and desmopressin (12%).
|Desmopressin response class (n patients)||Inadequate
|Unknown/not tested (n=26)||Total
|Moderate patients, n (% of response class)||7 (78)||13 (35)||6 (8)||18 (69)||44 (30)|
|Total number of bleeds||72||162||187||381||802|
|Bleeds per patient, median (IQR)||3 (2-13)||3 (1-5)||2 (0-3)||6 (2-18)||2 (1-5)|
|Treatment of bleeds (% of total bleeds)|
|FVIII||71 (99)||85 (52)||73 (39)||365 (96)||594 (74)|
|FVIII and desmopressin||–||33 (20)||41 (22)||1 (0)||75 (9)|
|Desmopressin||–||37 (23)||54 (29)||3 (1)||94 (12)|
|Other (i.e. tranexamic acid)||1 (1)||7 (4)||19 (10)||12 (3)||39 (5)|
Conclusions: Desmopressin response was adequate or excellent in 76% of patients and was used in 21% of the bleeding episodes in the total population.
To cite this abstract in AMA style:Zwagemaker A, Kloosterman FR, Gouw SC, Beckers EAM, Boyce S, Collins P, Eikenboom J, Shapiro S, Jackson S, Kruip MJHA, Laros-Van Gorkom B, Nieuwenhuizen L, Pabinger I, Coppens M, Van der Bom JG, Castaman G, Fijnvandraat K, DYNAMO Study Group . Use of Desmopressin in the Treatment of Bleeding in Non-severe Hemophilia A [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/use-of-desmopressin-in-the-treatment-of-bleeding-in-non-severe-hemophilia-a/. Accessed December 3, 2021.
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