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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Brian Feldman, The Hospital for Sick Children
ClinicalTrials.gov Identifier:
NCT01217255
First received: October 6, 2010
Last updated: December 17, 2013
Last verified: December 2013
  Purpose

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
Hemophilia A
Hemophilia B

Study Type: Observational
Study Design: 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

Resource links provided by NLM:


Further study details as provided by The Hospital for Sick Children:

Primary Outcome Measures:
  • Burden of Illness [ Time Frame: 3 years ] [ Designated as safety issue: No ]

    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:
  • The Activity Scale for Kids (ASK) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
    Children's self-reported activities will be measured with the Activity Scales for Kids (ASK) as part of the Activity Domain

  • Functional Independence Scale for Hemophilia (FISH) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]

    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.


  • Hemophilia Joint Health Score (HJHS) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]

    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.


  • Radiographs [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
    Radiographs will be taken of all 6 index joints. This will be done as part of the structure and function domain.

  • Canadian Haemophilia Outcomes - Kids Life Assessment Tool (CHO-KLAT) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
    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

  • Bleeding Frequency [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
    Families will be interviewed to determine bleeding frequency in the last 6 months. This factor will be incorporated into the Health condition domain.

  • Biometrics [ Time Frame: Days 1 ] [ Designated as safety issue: No ]
    The height and weight will be compared to national nomograms.

  • Personal Factors [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
    Ethnicity, level of education of the parent-guardian will be self-reported.

  • Environmental domain [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
    Whether a subject lives in a rural or urban area and household income will be recorded.


Enrollment: 100
Study Start Date: September 2010
Study Completion Date: October 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts
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

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.

  Eligibility

Ages Eligible for Study:   7 Years to 18 Years
Genders 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)

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.)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01217255

Locations
Brazil
Universidade Estadual de Campinas, UNICAMP
Campinas, Sao Paulo, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
Sao Paolo, 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
Investigators
Principal Investigator: Brain M. Feldman, MD,MSc,FRCPC The Hospital for Sick Children
  More Information

No publications provided

Responsible Party: Brian Feldman, Division Head, Rheumatology, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT01217255     History of Changes
Other Study ID Numbers: 1000020214
Study First Received: October 6, 2010
Last Updated: December 17, 2013
Health Authority: Canada: Ethics Review Committee

Keywords provided by The Hospital for Sick Children:
Hemophilia
Burden of Illness
Pediatric
Quality of Life
Disability
Participation

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

ClinicalTrials.gov processed this record on July 26, 2014