Paediatric Rheumatology - Special Articles
Domains and outcome measures for the assessment of digital vasculopathy and Raynaud's phenomenon in adult and juvenile systemic sclerosis: a scoping literature review
V. Maniscalco1, N. Vasquez-Canizares2, J. Lemon3, L. Stead4, V. Leone5, K. Clarke6, F. Zulian7, S.C. Li8, M. Twilt9, A. Adrovic10, S. Appenzeller11, E. Marrani12, M. Çakan13, E. Del Giudice14, M. Cattalini15, F. Tirelli16, L.A. Robinson17, S. Sampath18, G. Mastrangelo19, G. Simonini20, C.E. Pain21
- Rheumatology Unit, ERN-ReCONNET Center, Meyer Children’s Hospital IRCCS, Florence, Italy.
- Department of Pediatrics, Division of Pediatric Rheumatology, Children's Hospital at Montefiore/Albert Einstein College of Medicine, Bronx, NY, USA.
- Department of Development Paediatrics, Alder Hey Children’s NHS Foundation Trust, Eaton Road, Liverpool, UK.
- Department of Rheumatology, Alder Hey Children’s NHS Foundation Trust, Liverpool, UK.
- Department of Paediatrics, Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione-IRCCS ISMETT, University of Pittsburgh Medical Centre (UPMC), Palermo, Italy.
- Department of Rheumatology, Alder Hey Children’s NHS Foundation Trust, Liverpool, UK.
- Division of Paediatric Rheumatology, Department of Women and Children Health, University of Padova, Italy.
- Hackensack Meridian School of Medicine, Joseph M. Sanzari Children’s Hospital, Hackensack, NJ, USA.
- Department of Pediatrics, Alberta Children’s Hospital, Cumming School of Medicine, University of Calgary, Alberta, Canada.
- Department of Paediatric Rheumatology, Marmara University Pendik Training and Research Hospital Istanbul, Turkey.
- Department of Orthopaedics, Rheumatology and Traumatology, Universidade Estadual de Campinas, Brazil.
- Rheumatology Unit, ERN-ReCONNET Center, Meyer Children’s Hospital IRCCS, Florence, Italy.
- Zeynep Kamil Women and Children’s Diseases Training and Research Hospital, Department of Paediatric Rheumatology, Istanbul, Turkey.
- Paediatrics and Neonatology Unit, Maternal-Child Department, Santa Maria Goretti Hospital, Sapienza University of Rome, Latina, Italy.
- Pediatrics Clinic, University of Brescia and ASST Spedali Civili di Brescia, ERN-RITA Centre, Brescia, Italy.
- Division of Paediatric Rheumatology, Department of Women and Children Health, University of Padova, Italy.
- Division of Pediatric Rheumatology, Hospital for Special Surgery, New York, NY, USA.
- Department of Paediatric Rheumatology, Great North Children’s Hospital, Newcastle upon Tyne NHS Foundation Trust, Newcastle-upon-Tyne, UK.
- Division of Paediatric Rheumatology, Department of Women and Children Health, University of Padova, Italy.
- Rheumatology Unit, ERN-ReCONNET Center, Meyer Children’s Hospital IRCCS, Florence, Italy.
- Department of Rheumatology, Alder Hey Children’s NHS Foundation Trust, Liverpool; and Department of Women’s and Children’s Health, Institute of Life Course and Medical Sciences, University of Liverpool, UK. clare.pain@alderhey.nhs.uk
on behalf of the International Juvenile Systemic Sclerosis Outcome Group (IJOG) initiative, and the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Scleroderma Working Group
CER20283
2026 Vol.44, N°8
PI 1645, PF 1652
Paediatric Rheumatology - Special Articles
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PMID: 42635152 [PubMed]
Received: 30/06/2026
Accepted : 22/07/2026
In Press: 19/08/2026
Published: 19/08/2026
Abstract
OBJECTIVES:
Juvenile systemic sclerosis (jSSc) is a rare but severe paediatric rheumatic disease associated with substantial morbidity. Digital vasculopathy is nearly universal and includes Raynaud’s phenomenon (RP), digital ulcers (DU) and critical ischemia. Despite advances in adult systemic sclerosis (SSc), progress in jSSc remains limited. This scoping review aimed to identify outcome measures used to assess digital vasculopathy in SSc and jSSc to support core outcome set development for jSSc.
METHODS:
A scoping review was conducted following PRISMA-ScR guidelines. Medline, Embase, Web of Science, and CENTRAL were searched (1994–2024) for prospective studies reporting outcomes in digital vasculopathy domains (DU, RP, microvascular involvement, telangiectasia).
RESULTS:
Of 46,002 records, 108 studies were included; 105 (97.2%) involved adults only. Fifty distinct outcome measures were identified, including 35 clinician-reported outcomes (ClinROs) and 15 patient-reported outcomes (PROs). In the DU domain, 12 ClinROs and 5 PROs were identified, with ClinROs most frequently used as primary trial endpoints. In RP, 1 ClinRO and 9 PROs were identified; PROs predominated as primary outcomes. The microvascular domain included 16 ClinROs and no PROs, with instrumental measures used as primary endpoints. In telangiectasia, 6 ClinROs and 1 PRO were identified, rarely used in trials. Only three studies included paediatric patients, and no outcome measures were validated in children.
CONCLUSIONS:
Outcome measures for digital vasculopathy in SSc are heterogeneous and derived almost entirely from adult studies, with no paediatric-specific validation. Transferability of these measures to children requires formal evaluation. These findings support the need for standardised, validated outcomes for jSSc and inform core outcome set development.
