Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates

Annelies Nijdam; Wouter Foppen; Piet de Kleijn; Evelien P. Mauser-Bunschoten; Goris Roosendaal; Karin P. M. van Galen; Roger E. G. Schutgens; Yvonne T. van der Schouw; Kathelijn Fischer

doi:10.1160/TH15-08-0637

Thrombosis and Haemostasis, Inhaltsverzeichnis

Thromb Haemost 2016; 115(05): 931-938
DOI: 10.1160/TH15-08-0637

Coagulation and Fibrinolysis

Schattauer GmbH

Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates

Annelies Nijdam

¹Van Creveldkliniek, Centre for Benign Haematology, Thrombosis and Haemostasis, University Medical Centre, Utrecht, The Netherlands

,

Wouter Foppen

²Department of Radiology, University Medical Centre, Utrecht, The Netherlands

,

Piet de Kleijn

¹Van Creveldkliniek, Centre for Benign Haematology, Thrombosis and Haemostasis, University Medical Centre, Utrecht, The Netherlands

³Department of Rehabilitation, Nursing Science and Sports, University Medical Centre, Utrecht, The Netherlands

,

Evelien P. Mauser-Bunschoten

¹Van Creveldkliniek, Centre for Benign Haematology, Thrombosis and Haemostasis, University Medical Centre, Utrecht, The Netherlands

,

Goris Roosendaal

¹Van Creveldkliniek, Centre for Benign Haematology, Thrombosis and Haemostasis, University Medical Centre, Utrecht, The Netherlands

,

Karin P. M. van Galen

¹Van Creveldkliniek, Centre for Benign Haematology, Thrombosis and Haemostasis, University Medical Centre, Utrecht, The Netherlands

,

Roger E. G. Schutgens

¹Van Creveldkliniek, Centre for Benign Haematology, Thrombosis and Haemostasis, University Medical Centre, Utrecht, The Netherlands

,

Yvonne T. van der Schouw

⁴Julius Centre for Health Sciences and Primary Care, University Medical Centre, Utrecht, The Netherlands

,

Kathelijn Fischer

¹Van Creveldkliniek, Centre for Benign Haematology, Thrombosis and Haemostasis, University Medical Centre, Utrecht, The Netherlands

⁴Julius Centre for Health Sciences and Primary Care, University Medical Centre, Utrecht, The Netherlands

› Institutsangaben

Abstract

Summary

Prophylaxis is the recommended treatment for children with severe haemophilia A, but whether prophylaxis should be continued in adulthood is still under debate. Previous studies with limited follow-up have suggested that some patients may be able to stop prophylaxis in adulthood, while maintaining good joint health. This single-centre observational cohort study examined patients with severe haemophilia A born 1970–1988 without inhibitor development, and assessed the long-term consequences of discontinuing prophylaxis. Patient-initiated changes in prophylaxis, including all switches to on-demand treatment lasting a minimum of two consecutive weeks, were recorded from the time self-infusion began until the last evaluation. Sixty-six patients were evaluated at a median age of 32.4 years: 26 of patients had stopped prophylaxis for a median of 10 years, 15 had interrupted prophylaxis and 59 had continued prophylaxis. Annual joint bleeding rate (AJBR), Haemophilia Joint Health Score (HJHS-2.1; 0–124 points), radiological Pettersson score (0–78 points) and Haemophilia Activities List score (HAL; 100–0 points) were compared between patients who stopped and patients who continued prophylaxis. Although self-reported bleeding rates and functional limitations were similar in both groups (AJBR: 1.5 vs 1.2 and HAL: 84 vs 84 for those who stopped and continued prophylaxis, respectively), objective assessment of joint status showed increased arthropathy after 10 years of on-demand treatment in patients who stopped prophylaxis compared with those who continued (HJHS: 23 vs. 14 and Pettersson: 16 vs 5, respectively; P< 0.01). These results support continuation of long-term prophylaxis in adults and demonstrate the need for objective monitoring of joint status.

Trial registration: Dutch Trial Registry number 3098; UTN U1111–1121–7069.

Keywords

Arthropathy - joint bleed - haemophilia - prophylaxis - X-rays

Volltext

Referenzen

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