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

NCT01232634 | Completed

• 1 other identifier: 1000010841
• Study Type: observational
• Target: 57
• Locations: 2 countries
• Sites: 4

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.

Conditions

Hemophilia; Hemophilic Arthropathy

Keywords

pediatrics; haemophilia; ultrasound; MRI; arthropathy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• MRI findings according to the International Prophylaxis Study Group (IPSG) scale

  Day 1

• X-ray findings according to the Pettersson radiographic system

  Day 1

• Physical Assessment according to the HJHS and FISH scores

  Day 1

Study Arms (1)

All Subjects

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• The Hospital for Sick Children: lead
• University of Sao Paulo: collaborator
• Federal University of São Paulo: collaborator
• University of Campinas, Brazil: collaborator
• Baxter Healthcare Corporation: collaborator

Study Sites (4)

Universidade de Sao Paulo

São Paulo, Brazil

Location

Universidade Estaduale de Campinas

São Paulo, Brazil

Location

Universidade Federal de Sao Paulo

São Paulo, Brazil

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Hemophilia A; Joint Diseases

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; Musculoskeletal Diseases

Study Officials

• Andrea Doria, MD

  The Hospital for Sick Children, Toronto Canada

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinician Scientist

Study Record Dates

• First Submitted: October 29, 2010
• First Posted: November 2, 2010
• Study Start: September 1, 2010
• Primary Completion: November 1, 2013
• Study Completion: March 1, 2014
• Last Updated: November 9, 2016

Record last verified: 2016-11

Locations

CA (1); BR (3)