Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
Condition: Hemophilia A; Hemophilia B
MeSH terms: Hemophilia A; Hemophilia B
Study type: Observational

Summary: 
      Because of high cost, persons with hemophilia in many developing countries cannot afford
      adequate treatment. For example, many persons with hemophilia in India and China are only
      rarely treated with factor replacement in response to bleeds, and as a result many have
      developed significant arthropathy and disability. A pilot study in China estimated the mean
      Hemophilia Joint Health Score (HJHS) at 13.1 (SD 9.03) suggesting that these children had
      highly prevalent, severe joint disease. The lack of relationship between the HJHS and
      treatment history suggests overall inadequate therapy.

      The proposed study will quantify the burden of arthropathy, physical disability, and quality
      of life (QoL) in boys with hemophilia in Brazil - where comprehensive treatment is just
      beginning to be widely available. This study will also provide an opportunity to compare
      these outcomes to those observed in Canada, where the dominant therapy has become life-long
      prophylaxis.
    

Detailed description: 
      In order to quantify the burden of illness in hemophilia, and to study the response to
      different treatments, it is necessary to have quantitative outcome measures of high validity
      and reliability. The International Prophylaxis Study Group (IPSG - chair Dr. Victor
      Blanchette) was established in 2001 with the stated purpose of developing and testing outcome
      tools for this purpose. The group has developed a magnetic resonance imaging (MRI) score for
      quantifying arthropathy (representing the domain of structure and function domain in the ICF
      framework), a quantitative physical examination score, and the Hemophilia Joint Health Score
      or HJHS, to replace the older and less sensitive World Federation of Hemophilia (WFH) score
      (representing the domain of structure and function in the ICF). In addition Canadian
      investigators have also developed a quality of life measure for boys with hemophilia, the
      Canadian Hemophilia Outcomes Kids' Life Assessment Tool or CHO-KLAT (representing the domain
      of participation in the ICF).

      We will use these tools, and other validated measures, to begin to determine the burden of
      hemophilia in Brazil and compare it to the burden of disease in Canada. Additionally, we will
      use this study to demonstrate the validity of these tools in the Brazilian population.
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        Participants will be a representative (random) sample of individuals from the comprehensive
        hemophilia treatment centres at HCFMUSP and SickKids. Boys between the ages of 7 and 18
        years (inclusive) will be identified from the clinic ledgers at each of the centres. Using
        a computer generated random number list, proportionately stratified for Hemophilia A and B,
        and for moderate and severe disease (based on the baseline clinic proportions)
      
Keywords: Hemophilia; Burden of Illness; Pediatric; Quality of Life; Disability; Participation