Validation of Ultrasound as a Diagnostic Tool for Assessment of Hemophilic Arthropathy of Knees and Ankles

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia; Hemophilic Arthropathy

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.

• Study Type: Observational
• Enrollment (Actual): 57

Contacts and Locations

Study Locations

• Brazil
  • Sao Paulo, Brazil
    • Universidade Federal de Sao Paulo
  • Sao Paulo, Brazil
    • Universidade de Sao Paulo
  • Sao Paulo, Brazil
    • Universidade Estaduale de Campinas
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Children attending the Hemophilia clinics at the participating institutions (Universidade de São Paulo, Universidade Federal de São Paulo and Universidade Estadual de Campinas), who have a history of ankle or knee bleed(s), will be recruited to take part in this study.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
All Subjects

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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

Collaborators and Investigators

• Sponsor: The Hospital for Sick Children
• Collaborators: Baxter Healthcare Corporation; University of Sao Paulo; University of Campinas, Brazil; Federal University of São Paulo
• Investigators: Principal Investigator: Andrea Doria, MD, The Hospital for Sick Children, Toronto Canada

Study record dates

Study Major Dates

• Study Start: September 1, 2010
• Primary Completion (Actual): November 1, 2013
• Study Completion (Actual): March 1, 2014

Study Registration Dates

• First Submitted: October 29, 2010
• First Submitted That Met QC Criteria: November 1, 2010
• First Posted (Estimate): November 2, 2010

Study Record Updates

• Last Update Posted (Estimate): November 9, 2016
• Last Update Submitted That Met QC Criteria: November 8, 2016
• Last Verified: November 1, 2016

More Information

Terms related to this study

• Keywords: MRI; pediatrics; ultrasound; arthropathy; haemophilia
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Musculoskeletal Diseases; Blood Coagulation Disorders; Hemophilia A; Joint Diseases
• Other Study ID Numbers: 1000010841