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

NCT01217255 | Completed

• 1 other identifier: 1000020214
• Study Type: observational
• Target: 100
• Locations: 2 countries
• Sites: 4

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.

Conditions

Hemophilia A; Hemophilia B

Keywords

Hemophilia; Burden of Illness; Pediatric; Quality of Life; Disability; Participation

Outcome Measures

Primary Outcomes (1)

• Burden of Illness

  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)

  3 years

Secondary Outcomes (9)

• The Activity Scale for Kids (ASK)

  Day 1

• Functional Independence Scale for Hemophilia (FISH)

  Day 1

• Hemophilia Joint Health Score (HJHS)

  Day 1

• Radiographs

  Day 1

• Canadian Haemophilia Outcomes - Kids Life Assessment Tool (CHO-KLAT)

  Day 1

Study Arms (2)

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

Eligibility Criteria

• Age: 7 Years - 18 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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)

You may qualify if:

• 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.

You may not qualify if:

• 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.)

Sponsors & Collaborators

• The Hospital for Sick Children: lead

Study Sites (4)

Universidade Estadual de Campinas, UNICAMP

Campinas, São Paulo, Brazil

Location

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

São Paulo, Brazil

Location

Universidade Federal de São Paulo (UNIFESP)

São Paulo, Brazil

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

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

Study Officials

• Brain M. Feldman, MD,MSc,FRCPC

  The Hospital for Sick Children

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Division Head, Rheumatology

Study Record Dates

• First Submitted: October 6, 2010
• First Posted: October 8, 2010
• Study Start: September 1, 2010
• Primary Completion: October 1, 2013
• Study Completion: October 1, 2013
• Last Updated: June 6, 2018

Record last verified: 2018-06

Locations

CA (1); BR (3)