Summary This study prospectively compared the effect of secondary prophylaxis to episodic treatment on target joint (TJ) range of motion (ROM), number of joint haemorrhages and new TJ development in patients with moderate or severe haemophilia. Two-hundred and eighty-six males, 17% in prophylaxis, 83% in episodic treatment group, participating in the Centers for Disease Control and Prevention's Universal Data Collection project, fulfilled inclusion criteria: age >2 years at enrolment, free of TJs at enrolment, developed at least one TJ after enrolment, and received either prophylaxis or episodic treatment continuously for two follow-up visits after TJ development. The outcomes of interest – percentage change in TJ ROM, number of joint haemorrhages and new TJ development, were modelled using multivariate linear, Poisson and logistic regression techniques respectively. Individuals who received secondary prophylaxis in comparison to episodic treatment were younger at TJ development (P < 0·01); there was no difference in the decrease in TJ ROM between the two groups (P = 0·9). Factors significantly associated with a higher rate of haemarthroses included episodic treatment, severe haemophilia, age >5 years at TJ development, obesity and inhibitor negative status. Secondary prophylaxis significantly decreased haemarthroses but was not associated with a significant improvement in TJ ROM or with new TJ development.

Summary In persons with haemophilia (PWH), repeated ankle haemarthroses lead to pain, loss of joint range of motion (ROM), and limitations in activity and participation in society. PWH are offered ankle arthrodesis (AA) to eliminate pain. In our experience, PWH are hesitant to proceed to AA due to concerns regarding gait anomalies, functional decline and complete loss of ROM. The aim of this study was to report outcomes in ROM, assistive device (AD)/wheelchair use, activity scale and work/school absenteeism for participants in the CDC’s Universal Data Collection surveillance project (UDC) pre- and post- AA. Males with haemophilia enrolled in the UDC with first report of AA (1998–2010) were selected. Descriptive statistics were calculated using data from the annual study visit pre-AA and the follow-up visit (~12–24 months) post-AA. The 68 subjects who fulfilled the criteria were: mean age 36.9 years (SD = 12.9); 85.3% white; 85.3% haemophilia A; 72% severe, 20.6% moderate; and 10.3% with inhibitor once during the study period. Mean loss in total arc of ankle motion was 17.02° (SD = 21.8, P ≤ 0.01) pre- compared to post-AA. For 61.8%, there was no change in use of AD for ambulation/mobility. For 85.3%, there was no change in use of a wheelchair. On a self-reported activity scale, 11.8% improved, 8.8% worsened and 79.4% did not change. Work/school absenteeism averaged 2.7 (SD = 6.4) pre- and 1.5 (SD = 6.4, P = 0.26) days per year post-AA. While ankle ROM was significantly reduced post-AA, for most subjects, there was no change in use of AD/wheelchair for ambulation/mobility. Physical activity was maintained and work/school absenteeism remained stable.

Introduction Recurrent joint hemarthroses due to hemophilia (Factor VIII and Factor IX deficiency) often lead to invasive orthopedic interventions to decrease frequency of bleeding and/or to alleviate pain associated with end-stage hemophilic arthropathy. Aim Identify trends in invasive orthopedic interventions among people with hemophilia who were enrolled in the Universal Data Collection (UDC) program during the period 2000–2010. Methods Data were collected from 130 hemophilia treatment centers in the United States annually during the period 2000–2010, in collaboration with the Centers for Disease Control and Prevention (CDC). The number of visits in which an invasive orthopedic intervention was reported was expressed as a proportion of the total visits in each year of the program. Invasive orthopedic interventions consisted of arthroplasty, arthrodesis, and synovectomy. Joints included in this study were the shoulder, elbow, hip, knee, and ankle. Results A 5.6% decrease in all invasive orthopedic interventions in all joints of people with hemophilia enrolled in the UDC program over the 11-year study period was observed. Conclusions These data reflect a declining trend in invasive orthopedic interventions in people with hemophilia. Further research is needed to understand the characteristics that may influence invasive orthopedic interventions.

This study shows modifiable risk factors, including management of bleeding episodes with a continuous prophylactic treatment schedule are associated with a decreased likelihood of IOIs in males with haemophilia.