Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World

NCT ID: NCT01217255

Last Updated: 2018-06-06

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2010-09-30
• Study Completion Date: 2013-10-31

Brief 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.

Conditions

Hemophilia A; Hemophilia B

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Brazilian Subject's

Subject's will be recruited from the Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paolo (HCFMUSP); Universidade Estadual de Campinas (UNICAMP); Universidade Federal de São Paulo (UNIFESP)

No interventions assigned to this group

Canadian Subject's

Recruited from The Hospital for Sick Children

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• Hemophilia A or B moderate or severe as determined by serum factor activity ≤ 5%
• Age 7 - 18 yrs. inclusive
• At least one parent or guardian fluent in written Portuguese or English and able to complete the study outcome questionnaires.

Exclusion Criteria

• None (Children with inhibitors will be allowed into the study for 2 reasons: i) our study aims to document the health of persons with hemophilia in a generalizable way, ii) not all children will have had inhibitor testing done.)

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

The Hospital for Sick Children

OTHER

Sponsor Role: lead

Responsible Party

Brian Feldman

Division Head, Rheumatology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Brain M. Feldman, MD,MSc,FRCPC

Role: PRINCIPAL_INVESTIGATOR

The Hospital for Sick Children

Locations

Universidade Estadual de Campinas, UNICAMP

Campinas, São Paulo, Brazil

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

São Paulo, , Brazil

Universidade Federal de São Paulo (UNIFESP)

São Paulo, , Brazil

The Hospital for Sick Children

Toronto, Ontario, Canada

Countries

Brazil; Canada

Other Identifiers

1000020214

Identifier Type: -

Identifier Source: org_study_id