4.7 Review

Domains and outcome measures for the assessment of limited cutaneous systemic sclerosis: an international collaborative scoping review

Journal

RHEUMATOLOGY
Volume 61, Issue 8, Pages 3132-3148

Publisher

OXFORD UNIV PRESS
DOI: 10.1093/rheumatology/keac049

Keywords

limited cutaneous systemic sclerosis; scleroderma; outcome measure; domain; combined response index

Categories

Funding

  1. Scleroderma Raynaud UK/World Scleroderma Foundation (UHUHR1)
  2. Scleroderma Clinical Trials Consortium (SCTC)
  3. French network of the University Hospitals HUGO (Hopitaux Universitaires du Grand Ouest) (AAP-GIRCI JCM2020)
  4. Rennes University Hospital (CORECT Visiting Grant 2020)
  5. University of Michigan's Advanced Rehabilitation Research Training Program in Community Living and Participation from the National Institute of Disability, Independent Living, and Rehabilitation Research, Administration for Community Living [90ARCP0003]
  6. National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases T32 grant [AR007080]
  7. National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases [K24-AR-063120]

Ask authors/readers for more resources

The aim of this study was to identify instruments used to assess lcSSc, and the results show that lcSSc is underrepresented in the literature.
Objectives The aim of this study was to comprehensively identify instruments within relevant domains employed to assess lcSSc since the endorsement of its consensus definition in 1988. The overall objective is to inform the creation of a Combined Response Index for Scleroderma Trials Assessing lcSSc (CRISTAL). Methods MEDLINE and Embase were searched using terms selected to comprehensively retrieve titles and abstracts mentioning both lcSSc and dcSSc, along with those only mentioning lcSSc, SSc sine scleroderma, limited SSc and/or CREST/CRST. Because our initial assessment of the literature revealed that very few studies included only lcSSc subjects, we also assessed literature that included both cutaneous subsets. A total of 3964 titles and abstracts were screened by two reviewers, and 270 articles were selected for data extraction. Results We identified 27 domains encompassing 459 instruments. Instruments from 'Skin involvement', 'Pulmonary involvement' and 'Health-related quality of life and general functioning' were the most frequently retrieved. Among the 15 most represented instruments announced as primary end points in efficacy or effectiveness studies, 7 were clinician-reported outcomes (ROs), 7 were patient ROs, and one was a performance outcome (6 min-walk test). The mean proportion of lcSSc patients in studies of lcSSc, including studies that mention both lcSSc and dcSSc, was 56.4%, demonstrating that this subset is underrepresented in the literature, given that the prevalence of lcSSc ranges from 60% to 80% in national registries and international cohorts. Conclusion This scoping literature review provides a comprehensive identification of domains and outcomes used to assess lcSSc. Our results also highlight that lcSSc is underrepresented in the literature.

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