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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01217255
First Posted: October 8, 2010
Last Update Posted: December 19, 2013
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.
Information provided by (Responsible Party):
Brian Feldman, The Hospital for Sick Children
October 6, 2010
October 8, 2010
December 19, 2013
September 2010
October 2013   (Final data collection date for primary outcome measure)
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)

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, Biometetrics,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)

Complete list of historical versions of study NCT01217255 on ClinicalTrials.gov Archive Site
  • 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
  • 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.

  • 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.

  • 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.
  • 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
  • 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.
  • Biometrics [ Time Frame: Days 1 ]
    The height and weight will be compared to national nomograms.
  • Personal Factors [ Time Frame: Day 1 ]
    Ethnicity, level of education of the parent-guardian will be self-reported.
  • Environmental domain [ Time Frame: Day 1 ]
    Whether a subject lives in a rural or urban area and household income will be recorded.
  • 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
  • Functional Independence Scale for Hemophilia (FISH) [ Time Frame: Day 1 ]

    Observed activity limitationswill 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 ]

    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 ]
    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 ]
    After the completion of the pre-study cultural validition 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 ]
    Families will be interview to determine bleeding frequency in the last 6 months. This factor will be incorporated into the Health condition domain.
  • Biometrics [ Time Frame: Days 1 ]
    The height, weight and BMI will be compared to national nomograms.
  • Personal Factors [ Time Frame: Day 1 ]
    Ethnicity, level of education of the parent-guardian will be self-reported.
  • Environmental domain [ Time Frame: Day 1 ]
    Whether a subject lives in a rural or urban area and household income will be recorded.
Not Provided
Not Provided
 
Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
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.

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Probability Sample
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)
  • Hemophilia A
  • Hemophilia B
Not Provided
  • 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
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
100
October 2013
October 2013   (Final data collection date for primary outcome measure)

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.)
Sexes Eligible for Study: Male
7 Years to 18 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
Brazil,   Canada
 
 
NCT01217255
1000020214
No
Not Provided
Not Provided
Brian Feldman, The Hospital for Sick Children
The Hospital for Sick Children
Not Provided
Principal Investigator: Brain M. Feldman, MD,MSc,FRCPC The Hospital for Sick Children
The Hospital for Sick Children
December 2013