Health and treatment outcomes of patients with hemophilia in the Netherlands, 1972–2019

Shermarke Hassan, Erna C. van Balen, Cees Smit, Evelien P. Mauser-Bunschoten, Lize F.D. van Vulpen, Jeroen Eikenboom, Erik A.M. Beckers, Louise Hooimeijer, Paula F. Ypma, Laurens Nieuwenhuizen, Michiel Coppens, Saskia E.M. Schols, Frank W.G. Leebeek, Mariëtte H. Driessens, Frits R. Rosendaal, Johanna G. van der Bom, Samantha C. Gouw*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Introduction: We conducted six cross-sectional nationwide questionnaire studies among all patients with hemophilia in the Netherlands from 1972 until 2019 to assess how health outcomes have changed, with a special focus on patients >50 years of age. Methods: Data were collected on patient characteristics, treatment, (joint) bleeding, joint impairment, hospitalizations, human immunodeficiency virus and hepatitis C infections, and general health status (RAND-36). Results: In 2019, 1009 patients participated, of whom 48% had mild, 15% moderate, and 37% severe hemophilia. From 1972 to 2019, the use of prophylaxis among patients with severe hemophilia increased from 30% to 89%. Their median annual bleeding rate decreased from 25 to 2 bleeds. Patients with severe hemophilia aged <16 years reported joint impairment less often over time, but in those aged >40 years joint status did not improve. In 2019, 5% of all 1009 patients were positive for the human immunodeficiency virus. The proportion of patients with an active hepatitis C infection drastically decreased from 45% in 2001 to 2% in 2019 due to new anti-hepatitis C treatment options. Twenty-five percent had significant liver fibrosis even after successful therapy. Compared to the general male population, patients aged >50 years reported much lower scores on the RAND-36, especially on physical functioning. Discussion/Conclusion: Our study shows that increased use of prophylactic treatment and effective hepatitis C treatment have improved joint health and nearly eradicated hepatitis C infection in patients with hemophilia in the Netherlands. However, patients still suffer from hemophilia-related complications, especially patients aged >50 years.

Original languageEnglish
Pages (from-to)2394-2406
Number of pages13
JournalJournal of Thrombosis and Haemostasis
Issue number10
Early online date12 Jun 2021
Publication statusPublished - Oct 2021

Bibliographical note

Funding Information:
This study was funded by an unrestricted grant from the Dutch Ministry of Health, Welfare and Sport (Dutch: Ministerie van Volksgezondheid, Welzijn en Sport; VWS).

Funding Information:
S. Hassan, E.C. van Balen, C. Smit, E.P. Mauser, E.A.M. Beckers, L. Hooimeijer, P.F. Ypma, L. Nieuwenhuizen, S.E.M. Schols, M.H. Driessens, and F.R. Rosendaal have no conflicts of interest to disclose. L.F.D. van Vulpen received a research grant form CSL Behring, and is a consultant for Sobi and Tremeau. All fees go to the institution. J. Eikenboom received research support from CSL Behring and he has been a teacher on educational activities of Roche. M. Coppens has received financial support for research from Bayer, CSL Behring, Daiichi Sankyo, Portola/Alexion, Roche, Sanquin Blood Supply, and UniQure and consultancy or lecturing fees from Bayer, CSL Behring, Medcon International, MEDtalks, NovoNordisk, Pfizer, and Sobi. F.W.G. Leebeek received unrestricted research grants from CSL Behring, Takeda, uniQure, and Sobi; is consultant for uniQure, Novo Nordisk, Biomarin, and Takeda, of which the fees go to the institution; and has received a travel grant from Sobi. He is also a DSMB member for a study by Roche. J.G. van der Bom has been a teacher on the educational activities of Bayer. S.C. Gouw has received unrestricted research grants from Sobi.

Publisher Copyright:
© 2021 The Authors. Journal of Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis.


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