title: Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates.
abstract: 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.
authors.forename: Annelies
authors.initials: A
authors.lastname: Nijdam
authors.forename: Wouter
authors.initials: W
authors.lastname: Foppen
authors.forename: Piet
authors.initials: P
authors.lastname: De Kleijn
authors.forename: Evelien P
authors.initials: EP
authors.lastname: Mauser-Bunschoten
authors.forename: Goris
authors.initials: G
authors.lastname: Roosendaal
authors.forename: Karin P M
authors.initials: KP
authors.lastname: van Galen
authors.forename: Roger E G
authors.initials: RE
authors.lastname: Schutgens
authors.forename: Yvonne T
authors.initials: YT
authors.lastname: van der Schouw
authors.affiliation: Kathelijn Fischer, MD, PhD, Van Creveldkliniek, University Medical Centre Utrecht, Postzone C01.425, PO Box 85500, 3508 GA Utrecht, The Netherlands, Tel.: +31 88 755 8450, Fax: +31 88 755 5438, E-mail: k.fischer@umcutrecht.nl.
authors.forename: Kathelijn
authors.initials: K
authors.lastname: Fischer
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005169:Factor VIII / Q000008:administration & dosage*; D006395:Hemarthrosis / Q000517:prevention & control*; D006467:Hemophilia A / Q000150:complications* / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control*; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D063868:Patient Outcome Assessment; D011994:Recombinant Proteins / Q000008:administration & dosage; D012646:Self Administration; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; D005169:Factor VIII
keywords: Arthropathy; X-rays; haemophilia; joint bleed; prophylaxis