Best clinical practice in the treatment of juvenile systemic sclerosis: expert panel guidance - the result of the International Hamburg Consensus Meeting December 2022

ABSTRACT

Introduction

Juvenile systemic sclerosis (jSSc) is an orphan disease with a prevalence of 3 in 1,000,000 children. Currently there is only one consensus treatment guideline concerning skin, pulmonary and vascular involvement for jSSc, the jSSc SHARE (Single Hub and Access point for pediatric Rheumatology in Europe) initiative, which was based on data procured up to 2014. Therefore, an update of these guidelines, with a more recent literature and expert experience, and extension of the guidance to more aspects of the disease is needed.

Areas covered

Treatment options were reviewed, and opinions were provided for most facets of jSSc including general management, some of which differs from adult systemic sclerosis, such as the use of corticosteroids, and specific organ involvement, such as skin, musculoskeletal, pulmonary, and gastroenterology.

Expert opinion

We are suggesting the treat to target strategy to treat early to prevent cumulative disease damage in jSSc. Conclusions are derived from both expert opinion and available literature, which is mostly based on adult systemic sclerosis (aSSc), given shared pathophysiology, extrapolation of results from aSSc studies was judged reasonable.

KEYWORDS:

• gastrointestinal involvement
• juvenile systemic sclerosis
• musculoskeletal involvement
• physical therapy
• pulmonary involvement
• skin involvement
• vascular involvement
• treatment recommendations

Article highlights

• A diverse group of international jSSc experts propose treatment recommendations for juvenile systemic sclerosis (jSSc), both general guidance and regarding specific organ systems.

• Many recommendations are extrapolated from adult systemic scleroderma (SSc) data with a pediatric context.

• Both supportive care measures and medical therapies recommended for jSSc are described.

• The goal is to provide a standardized comprehensive treatment approach, which is both multidisciplinary and interdisciplinary for best care for jSSc patients.

• Implementation of recommendations should lead to overall better outcomes in jSSc.

• The pediatric rheumatology ‘treat to target’ concept should be applied to jSSc.

Declaration of interest

The meeting was supported by an unrestricted account with the following funds: Scleroderma Clinical Trial Consortium: 5000 US$, Boehringer 1000€, GSK: 1000€, MEDAC 800€, Novartis 750€, Mylan 800€, Nordic 500€. I Foeldvari is on the board of AMGEN, HEXAL, Lilly, MEDAC, Novartis and Pfizer. SC Li gets royalty payments from Wolters Kluwer for serving as a pediatric rheumatology section editor for UpToDate. P Moinzadeh received lecture fees from Actelion Pharmaceuticals, Boehringer Ingelheim and research grants from Actelion and Bayer Pharmaceuticals. JD Pauling has undertaken consultancy work and/or received personal support or speaker honoraria from Janssen, Astra Zeneca, Permeatus Inc, Boehringher-Ingelheim, IsoMab and Sojournix Pharma. Franziska Rosser time contribution was supported by HL159333. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.