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NCT Number: NCT01217255

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia B Hemorrhagic Disorders

Age range

7 year–18 year

Sex eligibility

Male

Study type

Observational

Primary location

Universidade Estadual de Campinas, UNICAMP, Campinas, São Paulo, Brazil

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.)

Treatment and study plan

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

The Hospital for Sick Children

Other

Registry information

Official study title

Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World: The Sao Paulo - Toronto Hemophilia Study

Important dates

Study start
2010
Primary completion
2013
Study completion
2013
First posted
Oct 8, 2010
Registry last updated
Jun 6, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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