Scleroderma

The EATC4Children is involved in developing outcome measures for Juvenile Localised Scleroderma (JLS) and investigating new treatments for JLS and Juvenile Systemic Scleroderma (JSS).

JLS is characterised by chronic inflammation within the skin and tissues leading to fibrosis. It can occur at any age but is more common in girls than in boys. The disease may be rare, with an incidence of 3.4 per one million children per year in the UK, however it is associated with significant complications including joint contractures, limb length discrepancy, and facial atrophy, that impact quality of life.

JSS is even rarer than JLS; less than 10% of children with scleroderma have systemic illness. Systemic sclerosis can also occur at any age and it involves the internal organs, such as the heart, lungs, or kidneys.

The EATC4Children are currently involved in three studies, detailed below.

Development of outcome measures in juvenile localised scleroderma

Currently there is almost no evidence base for treatment of JLS and clinical trials are urgently needed. This study aims to investigate measures of disease activity and the development of outcome measures that will:

  • Facilitate clinical treatment trials for children
  • Inform treatment decisions.

The study applies state of the art imaging techniques, using non-invasive high frequency ultrasound (HFUS), hyperspectral imaging (HSI), laser Doppler imaging (LDI) and infrared thermography (IRT) compared to examination including a clinical skin score and computerised skin score (for measuring surface area). This study will help set the foundation for future work and clinical trials to identify the best treatments for this rare but significant disease (CI: C. Pain).

Cohort and biomarker study in localised scleroderma of childhood

This study aims to develop the infrastructure for collecting clinical data, standardising clinical outcome measure assessments, and identifying biomarkers of disease activity/fibrosis in JLS. By combining these biomarkers with well standardised clinical measures of disease activity the hope is to identify robust outcome measures for clinical trials. This is a prospective cohort and biomarker study for children with localised scleroderma seen at Great Ormond Street Hospital NHS Foundation Trust and Alder Hey Children’s NHS Foundation Trust (CI: Despina Eleftheriou).

Elicitation of expert prior opinion for a future Bayesian randomised controlled trial for a rare inflammatory paediatric disease, Juvenile Localised Scleroderma (JLS)

There has only been one therapeutic treatment trial in JLS which examined the effectiveness of methotrexate (MTX). Mycophenolate mofetil (MMF) is an emerging alternative for MTX however there has not been a clinical trial of MMF in children with JLS. We want to perform a clinical trial comparing MTX to MMF.

Before this clinical trial can happen, there needs to be an international expert consensus meeting to establish prior opinion and knowledge. This is critical to informing a future clinical trial and how it is designed. The meeting seeks the prior opinion of experts on the relative efficacy of both MTX and MMF. Opinion will also be sought about the relevance of data from adult and/or other paediatric studies, agreeing the primary outcome and agreeing corticosteroid regimens of a future trial. (CI: C. Pain).

Dr Clare Pain: NIHR Research Scholars Programme

Dr Pain discusses the need for a clinical trial comparing MTX to MMF in the treatment of Juvenile Localised Scleroderma (JLS).

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