Background/Purpose: Juvenile systemic sclerosis (jSSc) is an orphan autoimmune disease that primary affect the vascular system determining multiple organ damage and reducing quality of life [1]. Currently, no standardized and validated outcome measures are available for jSSc [2]. The aim of this study was to comprehensively identify possible outcome measure to assess response to treatment for digital vasculopathy in jSSc.

Methods: A systematic literature review was conducted in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. The electronic bibliographic databases MEDLINE/PubMed, Embase, Web of Science and the Central Cochrane database were searched to identify studies on vasculopathy outcomes in adult (a) and jSSc. Only prospective studies that reported the outcomes of interest (i.e. Raynaud phenomenon, nailfold capillaroscopy, digital ulcers, acute digital ischemia/gangrene, acrosteolysis, telangiectasia, large vessel disease) measured at least two different time points were selected. The outcomes of interest were categorized in four domains: digital ulcers, Raynaud phenomenon, microvascular involvement, and telangiectasia.

Results: Of the 3285 retrieved publications, 83 papers were identified. These included 54 (65.1%) observational longitudinal studies, 26 (31.3%) randomized controlled trials, and 3 (3.6%) open label studies or non-randomized interventional studies. Eighty (96.4%) studies included both diffuse cutaneous (dc) SSc and limited cutaneous (lc) SSc and 3 (3.6%) papers included only lcSSc. Eighty (96.3%) studies included only adult SSc and 3 (3.6%) papers included children and adult jSSc. Overall, the pediatric patients were 3, all aged more than 10 years. Fifty-seven (68.7%) were single center studies and 26 (31.3%) were multicenter studies. Digital ulcers were reported in 55 publications (66.2%), microvascular involvement in 37 papers (44.6%), Raynaud phenomenon in 33 studies (39.8%), and telangiectasia in 2 (2.4%).

Conclusion: Our study provides a comprehensive identification of main domains to be assessed for identifying outcome measures in jSSc. No standard outcome measures have been applied and studies in jSSc are lacking. Our results highlight the urgent need to identify a core outcome set for jSSc and for assessing their feasibility in the pediatric population.

ACR Meeting Abstracts - https://acrabstracts.org/abstract/domains-and-outcome-measures-for-the-assessment-of-digital-vasculopathy-and-raynaud-phenomenon-in-juvenile-systemic-sclerosis-a-systematic-review/