Hemophilia Clinical Trial
Official title:
External Validation of Ultrasound as a Diagnostic Tool for Assessment of Hemophilic Arthropathy of Knees and Ankles. MRI Correlation.
The purpose of this study is to test the external validity of the systemic ultrasound protocol for data acquisition and interpretation, in order to diagnose soft tissue and osteochondral abnormalities in hemophilic children.
Status | Completed |
Enrollment | 57 |
Est. completion date | March 2014 |
Est. primary completion date | November 2013 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 7 Years to 18 Years |
Eligibility |
Inclusion Criteria: - Diagnosis of hemophilia A (factor VIII deficiency =1%) or B (factor IX deficiency =1.0%) - Cooperative patients (age of 7 to 18 years old) - Patients under 7 years old who have clinical evidence of arthropathy and imaging studies are required for clinical reasons. In these cases, the procedures may be done under sedation. - Male sex (higher prevalence in males) - History of previous ankle or knee bleed Exclusion Criteria: - Co-morbid illness such as juvenile idiopathic arthritis, muscular dystrophy, neuropathic arthropathy that cause osteoarticular findings that may obscure or confound the hemophilia-based joint findings - Non-cooperative patients - Active bleed (defined as evidence of a recent bleed with one week of the proposing imaging studies and/or musculoskeletal changes on physical examination suggestive of a bleed within one week of the proposed imaging studies). - Prior synovectomy |
Observational Model: Case-Only, Time Perspective: Prospective
Country | Name | City | State |
---|---|---|---|
Brazil | Universidade de Sao Paulo | Sao Paulo | |
Brazil | Universidade Estaduale de Campinas | Sao Paulo | |
Brazil | Universidade Federal de Sao Paulo | Sao Paulo | |
Canada | The Hospital for Sick Children | Toronto | Ontario |
Lead Sponsor | Collaborator |
---|---|
The Hospital for Sick Children | Baxter Healthcare Corporation, Federal University of São Paulo, University of Campinas, Brazil, University of Sao Paulo |
Brazil, Canada,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Ultrasound findings according to the International Prophylaxis Study Group (IPSG) scale | The readers will mark all the positive findings on a standardized spreadsheet and providing a final score for each component of the scale. Color Doppler findings will be rated as normal (grade 0), mild/moderate (grade 1) or severe (grade 2) synovial hyperemia according to an atlas that shows the intensity of hyperemia, which has been prepared by our group. | Day 1 | No |
Secondary | MRI findings according to the International Prophylaxis Study Group (IPSG) scale | The readers will interpret the MRI findings in an additive way, i.e., marking all positive findings on a standardized spreadsheet and providing a final score for the soft tissue and osteochondral components of the MRI scale. | Day 1 | No |
Secondary | X-ray findings according to the Pettersson radiographic system | Each joint will be rated on a 13-point score after the Pettersson system. | Day 1 | No |
Secondary | Physical Assessment according to the HJHS and FISH scores | Each knee, elbow and ankle joint receives a numeric score, which can be compared to itself over time to determine whether it is showing degeneration due to bleeding. FISH score is a seven item measure of disability used to evaluate change in functional independence over time. | Day 1 | No |
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