Domains and outcome measures for the assessment of digital vasculopathy and Raynaud's phenomenon in adult and juvenile systemic sclerosis: a scoping literature review.

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.