Multi-centre national audit of juvenile localised scleroderma: describing current UK practice in disease assessment and management

Hanna Lythgoe ,Mary Brennan ,Pamela Dawson ,Samundeeswari Deepak ,Ema Long ,Flora Mcerlane ,Chandrika Bhat ,Amarpal Bilkhu ,Nadia Rafiq ,Valentina Leone ,Despina Eleftheriou ,Nick Wilkinson ,Kathryn Harrison ,Sarah Maltby ,Athimalaipet V Ramanan ,Beverley Almeida ,Satyapal Rangaraj ,Joshua Bennett ,Eleanor Heaf ,Daniel Hawley ,Giulia Varnier ,Phil Riley ,Clare Pain

doi:10.1186/s12969-018-0295-0

Abstract

ObjectiveTo describe current United Kingdom practice in assessment and management of patients with juvenile localised scleroderma (JLS) compared to Paediatric Rheumatology European Society (PRES) scleroderma working party recommendations.MethodsPatients were included if they were diagnosed with JLS and were under the care of paediatric rheumatology between 04/2015–04/2016. Retrospective data was collected in eleven UK centres using a standardised proforma and collated centrally.Results149 patients were included with a median age of 12.5 years. The outcome measures recommended by the PRES scleroderma working party were not utilised widely. The localised scleroderma cutaneous assessment tool was only used in 37.6% of patients. Screening for extracutaneous manifestations did not meet recommendations that patients with head involvement have regular screening for uveitis and baseline magnetic resonance imaging (MRI) brain: only 38.5% of these patients were ever screened for uveitis; 71.2% had a MRI brain.Systemic treatment with disease-modifying anti-rheumatic drugs (DMARDs) or biologics was widely used (96.0%). In keeping with the recommendations, 95.5% of patients were treated with methotrexate as first-line therapy. 82.6% received systemic corticosteroids and 34.2% of patients required two or more DMARDs/biologics, highlighting the significant treatment burden. Second-line treatment was mycophenolate mofetil in 89.5%.ConclusionThere is wide variation in assessment and screening of patients with JLS but a consistent approach to systemic treatment within UK paediatric rheumatology. Improved awareness of PRES recommendations is required to ensure standardised care. As recommendations are based on low level evidence and consensus opinion, further studies are needed to better define outcome measures and treatment regimens for JLS.

Highlights

The hallmark of juvenile localised scleroderma (JLS) is chronic inflammation in the skin and soft tissues leading to fibrosis and eventually atrophy
Paediatric Rheumatology European Society (PRES) working party recommendations for assessment and management of JLS [13] were used as audit standards
This work highlights the significant treatment burden to JLS patients with 96.0% requiring treatment with at least one Disease-modifying anti-rheumatic drugs (DMARD) or biologic therapy and 34.2% receiving two or more. This finding may in part be due to selection bias, as this study only reviewed patients managed by paediatric rheumatologists while those with milder disease responding to topical treatment alone may be managed by a dermatologist alone

Summary

Introduction

The hallmark of JLS is chronic inflammation in the skin and soft tissues leading to fibrosis and eventually atrophy. This process can lead to complications such as joint contractures, limb length discrepancy and facial atrophy. First line treatment for active JLS is often methotrexate, its use supported by a randomised placebo controlled trial (RCT) and some observational studies [4,5,6,7,8]. A wide range of other treatments are used off–label in refractory cases with some suggestion of benefit [10–12]. Corticosteroids are used in the majority of patients presenting or relapsing with active JLS but there is wide variation of steroids regimens used [10, 11]

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Conclusion