Development of outcome measures in juvenile localised scleroderma
Research type
Research Study
Full title
Development of Outcome Measures in Juvenile Localised Scleroderma: Correlating Non-invasive Imaging, Clinical Skin Scores and Patient-Reported Outcome Measures.
IRAS ID
152882
Contact name
Eileen Baildam
Contact email
Sponsor organisation
Alder Hey Children's Foundation NHS Trust
Research summary
There is almost no evidence base for treatment for juvenile localised scleroderma (JLS) and clinical trials are badly needed. JLS causes severe inflammation in skin and underlying tissues leading to damage, fibrosis, loss of function, disfigurement and deformity. It is clinically difficult to distinguish active inflammatory disease from inactive fibrotic lesions. This study would investigate objective measures of disease activity and the development of outcome measures that will a) facilitate clinical treatment trials for children and b) inform treatment decisions (eg better delineation of inactive disease will allow cessation of treatment limiting potential immunosuppressive and steroid drug toxicity.
This study will use state of the art imaging techniques to assess sclerodermatous lesions and non-affected skin within the same patient, using non-invasive high frequency ultrasound (HFUS), optical coherence tomography (OCT), laser Doppler imaging (LDI), ultrasound elastography and infrared thermography (IRT). Assessment of lesions by imaging techniques will be compared to clinical examination including a clinical skin score
Each technique provides unique information. HFUS measures lesional depth and skin thickening particularly of the dermis. OCT allows better resolution of epidermal thickness; LDI assesses microcirculation whilst IRT is an indirect measure of skin perfusion. We will use the current standard clinical skin score for activity and damage in JLS (localised scleroderma cutaneous assessment tool; LoSCAT) We hypothesise that a combination of non-invasive imaging modalities along with a validated clinical score will provide a reproducible marker of disease activity for clinical practice and clinical trials. In addition, the novel approach correlating skin scores and patient reported outcomes with imaging methods will determine if use of skin scores alone in units without the full range of imaging modalities is appropriate. The novel application of these techniques will help contribute to understanding of the pathogenesis of JLS.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
14/NW/0203
Date of REC Opinion
24 Dec 2014
REC opinion
Further Information Favourable Opinion