TY - JOUR
T1 - Desmopressin for bleeding in non-severe hemophilia A
T2 - Suboptimal use in a real-world setting
AU - Zwagemaker, Anne-Fleur
AU - Kloosterman, Fabienne R.
AU - Coppens, Michiel
AU - Gouw, Samantha C.
AU - Boyce, Sara
AU - Bagot, Catherine N.
AU - Beckers, Erik A. M.
AU - Brons, Paul
AU - Castaman, Giancarlo
AU - Eikenboom, Jeroen
AU - Jackson, Shannon
AU - Kruip, Marieke J. H. A.
AU - Leebeek, Frank W. G.
AU - Meijer, Karina
AU - Nieuwenhuizen, Laurens
AU - Pabinger, Ingrid
AU - the DYNAMO study group
AU - Fijnvandraat, Karin
N1 - Funding Information: This work was supported by a grant from Novo Nordisk. The funding sponsor had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; and preparation, review, and approval of the manuscript. The authors thank all local research staff for their help in setting up and conducting the study. Publisher Copyright: © 2022 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH).
PY - 2022/8/1
Y1 - 2022/8/1
N2 - 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.
AB - 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.
KW - desmopressin
KW - factor VIII
KW - hemophilia A
KW - hemorrhage
KW - treatment
UR - http://www.scopus.com/inward/record.url?scp=85138897318&partnerID=8YFLogxK
U2 - https://doi.org/10.1002/rth2.12777
DO - https://doi.org/10.1002/rth2.12777
M3 - Article
C2 - 36090159
SN - 2475-0379
VL - 6
JO - Research and practice in thrombosis and haemostasis
JF - Research and practice in thrombosis and haemostasis
IS - 6
M1 - e12777
ER -