Health and treatment outcomes of patients with hemophilia in the Netherlands, 1972–2019. (7th July 2021)

• Record Type: Journal Article
• Authors: Hassan, Shermarke; van Balen, Erna C.; Smit, Cees; Mauser‐Bunschoten, Evelien P.; van Vulpen, Lize F. D.; Eikenboom, Jeroen; Beckers, Erik A. M.; Hooimeijer, Louise; Ypma, Paula F.; Nieuwenhuizen, Laurens; Coppens, Michiel; Schols, Saskia E. M.; Leebeek, Frank W. G.; Driessens, Mariëtte H.; Rosendaal, Frits R.; van der Bom, Johanna G.; Gouw, Samantha C.
• Is Part Of: Journal of thrombosis and haemostasis. Volume 19:Number 10(2021)
• Page Start: 2394
• Page End: 2406
• Publication Date: 2021-07-07
• Subjects: bleeding -- clinical outcomes -- hemophilia -- joint damage -- quality of life; Thrombosis -- Periodicals; Hemostasis -- Periodicals; Blood coagulation disorders -- Periodicals; 616.1
• DOI: 10.1111/jth.15424
• Languages: English
• ISSNs: 1538-7933

Abstract

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.