Hemophilia A Clinical Trial
Official title:
Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World: The Sao Paulo - Toronto Hemophilia Study
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.
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.
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