Scleroderma
Conditions
Keywords
Elastography ultrasound, Scleroderma, Quality of Life, Pediatric
Brief summary
Localized scleroderma (LS) is a skin disease that can cause complications such as disfigurement and limitation of mobility. Treatment for LS should be initiated early in the disease, as late-stage scleroderma does not respond well to treatment. Objective tools for diagnosis and follow-up of treatment for LS are not available clinically and are urgently needed. A new non-invasive ultrasound technique called "elastography" measures stiffness of tissues and holds potential for providing objective measures for follow-up of treatment of LS in children and adolescents. The team plans to conduct a multicenter study in North America to determine whether elastography ultrasound can show changes in skin stiffness for a period of 9 to 12 months after initiation of treatment for LS. This novel technology may add information to the existing imperfect clinical tools, thus improving the way therapy is offered to patients with LS.
Interventions
DIAGNOSTIC_TESTElastography Ultrasound
The US imaging protocol will include three parts: (i) gray-scale; (ii) color Doppler, and (iii) Shear-wave elastography (SWE)
OTHERThe Localized Scleroderma Quality of Life Instrument (LoSQI )
The questionnaire is a validated disease-specific patient-reported outcome measure that covers different domains (skin sensations, physical functioning, body image and social support, designed for children and adolescents with localized scleroderma
Sponsors
The Hospital for Sick Children
Childhood Arthritis and Rheumatology Research Alliance
Joseph Sanzari Children's Hospital
University of Pittsburgh
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
Inclusion criteria
* Boys and girls 0-18 years of age at the time of disease onset with a new or existing diagnosis of localized scleroderma (LS) confirmed by a pediatric rheumatologist or dermatologist. * Presents with one active or inactive lesion, using the contralateral site for comparison at each visit, as long as the same lesion is evaluated at each time point. OR Presents with more than one lesion as far as both lesions are active or inactive. * Able to cooperate with clinical and US evaluation, and the parent or guardian is able to fill out interim patient history form.
Exclusion criteria
* Patients with other co-morbidities (e.g. systemic sclerosis, juvenile idiopathic arthritis, other systemic diseases). * Patients who present with one active and one inactive lesion. * Patients with lesions located in the face or head.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Skin stiffness | Baseline, 3-6m and 9-12m | Skin stiffness will be assessed using Elastography (SWE) Ultrasound, a sonographic technique that uses mechanical waves to assess tissue elasticity and display it in a quantitative manner. The stiffness of the tissue is directly proportional to its shear wave velocity. SWE will measure interval changes of skin stiffness during treatment at the target lesion and the unaffected contralateral site. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Lesion activity responsiveness | Baseline, 3-6m and 9-12m | The LoSCAT (Localized Sclerodema Cutaneous Assessment Tool scores) assesses 18 cutaneous anatomic sites, capturing both disease activity (mLoSSI - Modified Localized Skin Severity Index) and disease damage (LoSDI - Localized Scleroderma Damage Index). Scores for each site are based on the most severe score for each parameter ranging from 0 to 3. A higher score represents a worse outcome. |
| Echogenicity scores for dermis and hypodermis | Baseline, 3-6m and 9-12m | Standard ultrasound protocol. Identifying sonographic changes in the involved body site will be accomplished by comparing both echogenicity and color Doppler signal to a corresponding normal site. The score is determined by summing the absolute value of the individual parameters, with a range from 0 to 15 (maximum echogenicity score of 6, maximum vascularity score of 9). A higher score represents a worse outcome. |
| Vascularity scores for dermis and hypodermis | Baseline, 3-6m and 9-12m | Doppler Ultrasound protocol. Identifying sonographic changes in the involved body site will be accomplished by comparing both echogenicity and color Doppler signal to a corresponding normal site. The score is determined by summing the absolute value of the individual parameters, with a range from 0 to 15 (maximum echogenicity score of 6, maximum vascularity score of 9). A higher score represents a worse outcome. |
| Quality of Life | Baseline, 3-6m and 9-12m | Completiom of the LoSQI (Localized Scleroderma Quality of Life Instrument), a self-reported disease-specific questionnaire to assess the quality of life of patients (children, adolescents and young adults) with localized scleroderma. It consists of 21 items across three theoretical domains: (1) skin sensations, (2) physical functioning and musculoskeletal sequelae, and (3) body image and social support. Scores are based on the most severe score for each parameter ranging from 0 to 3. The questionnaire includes questions that comprise the Pain \& Physical Functioning subscale, and the Body Image \& Social Support subscale. Scores are summoned and converted to a scale, ranging from 41.3 to 71.6 and 43.58 to 68.42, respectively. A higher score represents a worse outcome. |
Countries
Canada
Outcome results
None listed