Hemophilia, Hemophilic Arthropathy
Conditions
Keywords
pediatrics, haemophilia, ultrasound, MRI, arthropathy
Brief summary
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.
Detailed description
Ultrasound has advantages over MRI as it is less expensive, does not require sedation and is more readily available in centres around the world. Given these characteristics of ultrasound it is an ideal imaging tool for early assessment of hemophilic joints in countries whose access to MRI is limited. Early evaluation of soft tissue changes in young hemophilic patients may lead to earlier interventions and better mid-term and long-term clinical outcomes. By this way the joint function of hemophilic patients can be preserved, saving their joints from severe arthropathy and offering them the possibility of a normal professional life in the society.
Interventions
None listed
Sponsors
University of Sao Paulo
Federal University of São Paulo
University of Campinas, Brazil
Baxter Healthcare Corporation
The Hospital for Sick Children
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
7 Years to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Ultrasound findings according to the International Prophylaxis Study Group (IPSG) scale | Day 1 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| MRI findings according to the International Prophylaxis Study Group (IPSG) scale | Day 1 | 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. |
| X-ray findings according to the Pettersson radiographic system | Day 1 | Each joint will be rated on a 13-point score after the Pettersson system. |
| Physical Assessment according to the HJHS and FISH scores | Day 1 | 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. |
Countries
Brazil, Canada
Outcome results
None listed