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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01217255
Recruitment Status : Completed
First Posted : October 8, 2010
Last Update Posted : June 6, 2018
Information provided by (Responsible Party):
Brian Feldman, The Hospital for Sick Children

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.

Condition or disease
Hemophilia A Hemophilia B

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.

Study Type : Observational
Actual Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World: The Sao Paulo - Toronto Hemophilia Study
Study Start Date : September 2010
Actual Primary Completion Date : October 2013
Actual Study Completion Date : October 2013

Resource links provided by the National Library of Medicine

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)
Canadian Subject's
Recruited from The Hospital for Sick Children

Primary Outcome Measures :
  1. Burden of Illness [ Time Frame: 3 years ]

    Burden of disability for Brazilian and Canadian children will be be determined by analysis of the following domains:

    Structure and Function Domain (HJHS score, Biometrics,Radiographs) Activity Domain (ASK and FISH scales) Participation Domain (CHO-KLAT and PedsQL Quality of Life Questionnaire) Health Condition Domain (bleeding frequency) Personal Factors Domain (Ethnicity, level of education of parent/guardian, habitual exercise) Environmental Domain (Household income of parent/guardian)

Secondary Outcome Measures :
  1. The Activity Scale for Kids (ASK) [ Time Frame: Day 1 ]
    Children's self-reported activities will be measured with the Activity Scales for Kids (ASK) as part of the Activity Domain

  2. Functional Independence Scale for Hemophilia (FISH) [ Time Frame: Day 1 ]

    Observed activity limitations will be measured with the FISH as part of the Activity Domain.

    The FISH has been validated for use in developing countries. It consists of observed activities of daily living that are scored for quality.

  3. Hemophilia Joint Health Score (HJHS) [ Time Frame: Day 1 ]

    The HJHS is a valid and reliable scored measure of joint structural limitations as applied to the index joints.

    This score will be a measure of the Structure and function domain.

  4. Radiographs [ Time Frame: Day 1 ]
    Radiographs will be taken of all 6 index joints. This will be done as part of the structure and function domain.

  5. Canadian Haemophilia Outcomes - Kids Life Assessment Tool (CHO-KLAT) [ Time Frame: Day 1 ]
    After the completion of the pre-study cultural validation of the Brazilian translation of the CHO-KLAT scale, we will use it as a Quality of Life tool to measure the Participation domain

  6. Bleeding Frequency [ Time Frame: Day 1 ]
    Families will be interviewed to determine bleeding frequency in the last 6 months. This factor will be incorporated into the Health condition domain.

  7. Biometrics [ Time Frame: Days 1 ]
    The height and weight will be compared to national nomograms.

  8. Personal Factors [ Time Frame: Day 1 ]
    Ethnicity, level of education of the parent-guardian will be self-reported.

  9. Environmental domain [ Time Frame: Day 1 ]
    Whether a subject lives in a rural or urban area and household income will be recorded.

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Ages Eligible for Study:   7 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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)

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.)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01217255

Universidade Estadual de Campinas, UNICAMP
Campinas, Sao Paulo, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
Sao Paulo, Brazil
Universidade Federal de São Paulo (UNIFESP)
Sao Paulo, Brazil
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
The Hospital for Sick Children
Principal Investigator: Brain M. Feldman, MD,MSc,FRCPC The Hospital for Sick Children

Responsible Party: Brian Feldman, Division Head, Rheumatology, The Hospital for Sick Children Identifier: NCT01217255     History of Changes
Other Study ID Numbers: 1000020214
First Posted: October 8, 2010    Key Record Dates
Last Update Posted: June 6, 2018
Last Verified: June 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Brian Feldman, The Hospital for Sick Children:
Burden of Illness
Quality of Life

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked