Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project

Marilyn J. Manco-Johnson, J. Michael Soucie and Joan Cox Gill for the Joint Outcomes Committee of the Universal Data Collection, US Hemophilia Treatment Center Network

Article Figures & Data


  • Figure 1.

    Trends in prophylaxis use for children and adults with severe hemophilia A from 1999 to 2010. The proportion of each participant age cohort (in decades) using continuous prophylaxis is displayed for each year of the study from 1999 to 2010.

  • Figure 2.

    Trends in mean number of joint hemorrhages in the preceding 6 months by prophylaxis status, 1999 to 2010. (A-C) The vertical axis on the right displays the mean number of joint hemorrhages per 6 months during each study year for participants using (▲) or not using (▪) continuous prophylaxis. The vertical axis on the left displays the proportion of the entire age cohort using prophylaxis for each study year (□). (A-C) Displays results for children ages 2 to 9 years (A), adolescents 10 to 19 years (B), and adults 20 to 29 years (C), respectively. Prophy, prophylaxis; Prophy +, data from patients on prophylaxis; Prophy −, data from patients not on prophylaxis.

  • Figure 3.

    Cross-sectional analyses: mean changes in bleeding rates and joint range of motion from 1999 to 2010. (A-D) Shows changes in outcome rates from 1999 to 2010 stratified by age cohort and prophylaxis usage. (A) Rates of joint hemorrhages; (B) rates of target joints; (C) total bleeding rates; and (D) percentage of normal joint ROM in participants. Patients on prophylaxis in 1999 (red); patients on prophylaxis in 2010 (blue); patients not on prophylaxis in 1999 (green); and patients not on prophylaxis in 2010 (purple).


  • Table 1.

    Baseline characteristics of 6196 males with severe hemophilia A receiving care in US hemophilia treatment centers, 1999 to 2010

    Age at first visit, y
     White, non-Hispanic388862.8
     Black, non-Hispanic96315.5
    Treatment type at first visit
     On demand388762.7
    • * Hispanic ethnicity of either black or white race.

  • Table 2.

    Multivariate analysis on the effect of prophylaxis on the repeated measures of overall joint ROM over time in 2908 US hemophilia participants aged 2 to 19 years

    Initial ROM statusRate of ROM change
    CovariatesParameter estimatePParameter estimateP
    Race: white vs others1.56<.001
    Inhibitor: yes vs no−0.46.06
    BMI: ≥85th vs <85th percentile−1.07.003−0.09.001
    Primary prophy <4 y: yes vs no0.
    Primary prophy 4 or 5 y: yes vs no0.92.340.10.29
    Secondary prophy: yes vs no0.54.460.04.36
    • Prophy, prophylaxis.