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. 2021 Oct;19(10):2394-2406.
doi: 10.1111/jth.15424. Epub 2021 Jul 7.

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

Shermarke Hassan  1 , Erna C van Balen  1 , Cees Smit  1 , Evelien P Mauser-Bunschoten  2 , Lize F D van Vulpen  2 , Jeroen Eikenboom  3 , Erik A M Beckers  4 , Louise Hooimeijer  5 , Paula F Ypma  6 , Laurens Nieuwenhuizen  7 , Michiel Coppens  8 , Saskia E M Schols  9   10 , Frank W G Leebeek  11 , Mariëtte H Driessens  12 , Frits R Rosendaal  1 , Johanna G van der Bom  1   13 , Samantha C Gouw  1   14
Affiliations

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

Shermarke Hassan et al. J Thromb Haemost. 2021 Oct.

Abstract

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.

Keywords: bleeding; clinical outcomes; hemophilia; joint damage; quality of life.

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Conflict of interest statement

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.

Figures

FIGURE 1
FIGURE 1
Health and treatment outcomes over time. A, Proportion of patients with severe hemophilia on prophylactic treatment, from 1972 to 2019, stratified by age. B, Median annual bleeding rate of patients with severe hemophilia, from 1972 to 2019, stratified by age. C, Self‐reported absence of joint impairment in ankles, knees, and elbows in patients with severe hemophilia, from 1972 to 2019, stratified by age
See this image and copyright information in PMC

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