Embolization in Hereditary Coagulopathies (EHCO)

This is a longitudinal, prospective study, which will include 30 subjects with hereditary coagulopathies, with arthropathy, chronic synovitis resulting from hemarthrosis of the elbows, knees and/or ankles followed up at the Centro de Hemofilia HCFMUSP, after approval by the ethics and research committee.

They will undergo imaging tests (X-rays and Magnetic Resonance of knee, elbow, or ankle), physical, pain, quality of life and functional assessments (Hemophilia Joint Health Score, Functional Independence Score in Hemophilia, Perimeter, Test Timed up and Go, 30 second sit and stand test, Haemophilia - Adult - Quality of Life questionnaire (HAEM-A-QoL), Knee Injury and Osteoarthritis Outcome Score, EQ-5D, numerical rating scale for pain and embolization procedure (superselective embolization of target arteries with spherical microparticles Embosphere 100-300 micrometers (Biosphere Medical, Roissy, France), until partial vascular stasis and decharacterization of pathological synovial enhancement.

These evaluations will be performed at baseline, 1, 3, 6, 12, 24, 36, 48 and 60 months after the procedure.

Study Overview

• Status: Recruiting
• Conditions: Hemophilia; Arthropathy; Synovitis; Hemarthrosis; Embolization; Clotting Factor Deficiency
• Intervention / Treatment: Device: embolization with spherical microparticles embosphere

Detailed Description

Longitudinal, prospective study, which will include 30 subjects with hereditary coagulopathies, with arthropathy, chronic synovitis resulting from hemarthrosis of the elbows, knees and/or ankles followed up at the Centro de Hemophilia do Hospital das Clinicas, Faculdade de Medicina da Universidade de São Paulo, i.e. HC-FMUSP), after approval by the ethics and research committee and registry at the clinical trials.

Patients will be evaluated by imaging (X-rays and Magnetic Resonance of the affected joint, i.e., knee, elbow, and/or ankle); physical and functional assessments (Hemophilia Joint Health Score, Functional Independence Score in Hemophilia, Perimeter of the affected joint(s), Test Timed up and Go, 30 second sit and stand test, Knee Injury and Osteoarthritis Outcome Score (KOOS); quality of life (Haemophilia - adult - quality of life questionnaires (HAEM-A-QoL), Knee Injury and Osteoarthritis Outcome Score (KOOS), EQ-5D; pain measured by numerical rating scale.

Volunteers will be submitted to embolization procedure (superselective embolization of target arteries with spherical microparticles embosphere 100-300 micrometers (Biosphere Medical, Roissy, France) of the affected joint(s), until partial vascular stasis and decharacterization of pathological synovial enhancement.

These evaluations will be performed at baseline, 1, 3, 6, 12, 24, 36, 48 and 60 months after the procedure.

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: marcia u rezende, MD PhD; Phone Number: +5511 981226282; Email: marcia.uchoa@hc.fm.usp.br
• Study Contact Backup: Name: Fabiane E Farias, PT; Phone Number: + 5511 3062-5531; Email: fabiane.elize@hc.fm.usp.br

Study Locations

• Brazil
  • São Paulo, Brazil, 05403-010
    • Recruiting
    • Instituto de Ortopedia e Traumatologia do Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo
    • Contact: Marcia U Rezende, MD, PhD; Phone Number: 551130625531; Email: marcia.uchoa@hc.fm.usp.br
    • Contact: Fabiane P Farias, PT; Phone Number: 55112661-2473; Email: fabiane.elize@hc.fm.usp.br
    • Principal Investigator: Marcia U Rezende, MD, PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patients with hereditary coagulopathies with arthropathy (i.e., chronic synovitis due to hemarthrosis), with X-ray and MRI (documenting synovitis) followed up at the Hemophilia Center HC-FMUSP.

Exclusion Criteria:

• Patients who do not complete the planned assessments
• Patients who do not accept to continue with the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Embolization; embolization procedure (superselective embolization of target arteries with spherical microparticles Embosphere 100-300 micrometers (Biosphere Medical, Roissy, France) of the affected joint, until partial vascular stasis and decharacterization of pathological synovial enhancement.	Device: embolization with spherical microparticles embosphere; embolization of affected joints with spherical microparticles embosphere

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reduction of synovium thickness by embolization	Measure (in milimeters) changes of synovial thickness in patients with hereditary coagulopathies with arthropathy due to hemarthrosis with MRI (measure synovium thickness)	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reduction of synovitis by embolization	Measure (in milimeters) changes on synovial thickness in patients with hereditary coagulopathies with arthropathy due to hemarthrosis with MRI	1, 3 12, 24, 36, 48 and 60 months
Inferior limb strength	Measure changes in muscle strength by timed up and go (by time of performance, in seconds).	1, 3, 6, 12, 24, 36, 48, 60 months
Inferior limb strength	Measure changes in muscle strength of lower limbs by 30 seconds sit to stand test (by number of repetitions)	1, 3, 6, 12, 24, 36, 48, 60 months
Balance and fall risk	Measure (in seconds, period of time to perform the test) changes in balance and risk of falls by Timed up and go test.	1, 3, 6, 12, 24, 36, 48, 60 months
Quality of life of the patient	Measure, according to the scale of the questionnaire, changes in quality of life with Haemophilia - Adult - Quality of life Questionnaires (HAEM-A-QoL, 10 dimensions, 0 to 100, being 0 the best health related quality of life)	1, 3, 6, 12, 24, 36, 48, 60 months
Quality of life of the patient	Measure, according to the scale of the questionnaire, changes in quality of life with Knee Injury and Osteoarthritis Outcome Score (KOOS, 0-100, 100 being the best quality of life).	1, 3, 6, 12, 24, 36, 48, 60 months
Quality of life of the patient	Measure, according to the scale of the questionnaire, changes in quality of life with by EQ-5D, up to 1, being 1 the best quality of life). Please note: "Since 2009, EQ-5D has been available in three versions: the three-level EQ-5D-3L; the five-level EQ-5D-5L; and a 'youth' adaptation, EQ-5D-Y. Although the EQ is in recognition of the EuroQol group name, the D refers to dimensions, the L signifies level, and the Y stands for youth, the name of the instrument should not be spelled out in this manner as this is inaccurate. Therefore, EQ-5D is not an abbreviation and is the correct term to use when referring to the instrument in general." Brooks R, Boye KS, Slaap B. EQ-5D: a plea for accurate nomenclature. J Patient Rep Outcomes. 2020 Jul 3;4(1):52. doi: 10.1186/s41687-020-00222-9. PMID: 32620995; PMCID: PMC7334333.	1, 3, 6, 12, 24, 36, 48, 60 months
Functional capacity	Measure (according to the scale of the questionnaire) changes in functional capacity with Functional Independence Score in Hemophilia (FISH score, 0 to 32, 32 being the best result)	1, 3, 6, 12, 24, 36, 48, 60 months
Functional capacity	Measure (according to the scale of the questionnaire) changes in functional capacity with Functional Independence Score by Knee Injury and Osteoarthritis Outcome Score (KOOS, 0-100, 100 being the best result)	1, 3, 6, 12, 24, 36, 48, 60 months
Physical capacity	Measure changes in physical capacity by Hemophilia Joint Health Score (HJHS), 0 to 124, 0 being the best result.	1, 3, 6, 12, 24, 36, 48, 60 months
Pain of the affected joint	Measure changes in pain by Hemophilia Joint Health Score (HJHS, 0-12, 0 being the best result)	1, 3, 6, 12, 24, 36, 48, 60 months
Pain of the affected joint	Measure changes in pain by numerical rating scale (NRS, 0-10, 0 being no pain and 10 being the worst pain possible)	1, 3, 6, 12, 24, 36, 48, 60 months
Pain of the affected joint	Measure changes in pain by Knee Injury and Osteoarthritis Outcome Score (KOOS, 0 to 100, 100 being the best result)	1, 3, 6, 12, 24, 36, 48, 60 months
Stifness	Measure (0 to 100, 100 being the best result) changes in knee stiffness by Knee Injury and Osteoarthritis Outcome Score (KOOS)	1, 3, 6, 12, 24, 36, 48, 60 months

Collaborators and Investigators

• Sponsor: University of Sao Paulo General Hospital
• Collaborators: Merit Medical Systems, Inc.
• Investigators: Principal Investigator: Fabiane E FArias, PT, IOT-HC-FMUSP (Orthopedics Institute - General Hospital- School of Medicine - University of Sao Paulo; Study Director: Andre M Assis, MD, PhD, Radiology Insititute, General Hospital, School Medicine, University of São Paulo; Study Director: Paula Villaça, MD, PhD, Hematology department, General Hospital, School of Medicine, University of Sâo Paulo

Study record dates

Study Major Dates

• Study Start (Anticipated): January 16, 2023
• Primary Completion (Anticipated): December 31, 2028
• Study Completion (Anticipated): December 31, 2029

Study Registration Dates

• First Submitted: September 28, 2022
• First Submitted That Met QC Criteria: November 16, 2022
• First Posted (Actual): November 29, 2022

Study Record Updates

• Last Update Posted (Actual): November 29, 2022
• Last Update Submitted That Met QC Criteria: November 16, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Hemorrhage; Joint Diseases; Musculoskeletal Diseases; Synovitis; Hemarthrosis
• Other Study ID Numbers: 63452822.1.0000.0068

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No