<p>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 &gt;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 &lt;16 years reported joint impairment less often over time, but in those aged &gt;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 &gt;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 &gt;50 years.</p>

doi.org/10.1111/jth.15424, hdl.handle.net/1765/136734
Journal of Thrombosis and Haemostasis
Erasmus MC: University Medical Center Rotterdam

Shermarke Hassan, Erna C. van Balen, Cees Smit, E. P. Mauser-Bunschoten, Lize F.D. van Vulpen, Jeroen C.J. Eikenboom, … Samantha C. Gouw. (2021). Health and treatment outcomes of patients with hemophilia in the Netherlands, 1972–2019. Journal of Thrombosis and Haemostasis, 19(10), 2394–2406. doi:10.1111/jth.15424