Clinical trial NCT01217255: Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
Official title: Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World: The Sao Paulo - Toronto Hemophilia Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Hemophilia B
Keywords: Hemophilia
Burden of Illness
Pediatric
Quality of Life
Disability
Participation
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
First posted: 2010-10-08
Enrollment: 100
Sex: MALE
Minimum age (years): 7
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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)

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.

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.)

Primary outcomes:
measure: Burden of Illness
timeFrame: 3 years
Secondary outcomes:
measure: The Activity Scale for Kids (ASK)
timeFrame: Day 1
measure: Functional Independence Scale for Hemophilia (FISH)
timeFrame: Day 1
measure: Hemophilia Joint Health Score (HJHS)
timeFrame: Day 1
measure: Radiographs
timeFrame: Day 1
measure: Canadian Haemophilia Outcomes - Kids Life Assessment Tool (CHO-KLAT)
timeFrame: Day 1
measure: Bleeding Frequency
timeFrame: Day 1
measure: Biometrics
timeFrame: Days 1
measure: Personal Factors
timeFrame: Day 1
measure: Environmental domain
timeFrame: Day 1
Other outcomes:
Locations:
city: Campinas
country: Brazil
facility: Universidade Estadual de Campinas, UNICAMP
state: Sao Paulo
city: Sao Paulo
country: Brazil
facility: Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
city: Sao Paulo
country: Brazil
facility: Universidade Federal de São Paulo (UNIFESP)
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: The Hospital for Sick Children
name: Brain M. Feldman, MD,MSc,FRCPC
role: PRINCIPAL_INVESTIGATOR
References: