Background/Purpose: Juvenile systemic sclerosis (jSSc) is a rare fibrosing autoimmune disease associated with significant morbidity and mortality. Data on treatment strategies is limited, primarily based on adult data. As jSSc has many differences from adult SSc, including higher overlap subtype frequency, jSSc studies are needed for optimal care. An international collaboration based on CARRA/PReS was formed to create consensus jSSc organ-specific outcome measures for use in future studies. We report the findings of the skin/musculoskeletal subgroup. This study identified and evaluated cutaneous and musculoskeletal outcome measures for adult and juvenile SSc utilizing a scoping literature review and survey of current practice.

Methods: Four databases identified 24,849 articles. Utilizing inclusion criteria of English language, original peer-reviewed research, and study of a measure from 1 of 6 organs of interest in SSc, 3,284 articles were identified for organ-specific review. The skin/musculoskeletal subgroup then identified 135 articles that reported longitudinal data of measures likely to change in a 1-year clinical trial. These articles underwent full text extraction. Extraction data were used to generate an online survey to identify current practice patterns in assessing cutaneous and musculoskeletal involvement in jSSc. This survey was sent to physicians studying jSSc, including the CARRA/PReS scleroderma working groups (n = 90).

Results: Most studies were single country (80%), single center (61%), and interventional (83%). Prospective cohort (36%) was the most common study type. Only 6 studies included patients with jSSc (4%) and only 5 studies (4%) noted inclusion of overlap subtype. Skin was the most common domain assessed (84%). The musculoskeletal system was investigated less, with few studies assessing joint (19%) and muscle (10%). In addition, 30% of studies evaluated the sclerodermatous hand. The modified Rodnan skin score (mRSS) (77%), joint counts such as DAS28 (7%), and measures of grip/pinch strength (10%) were the most studied measures of the skin, musculoskeletal, and hand domains respectively. Most survey respondents (n = 31) were pediatric rheumatologists (97%) from North America or Europe (77%). While most respondents had received training on the mRSS (90%), only 55% received training during fellowship (Table 1). Reported to be often or always used to assess their jSSc patients, mRSS (71%), joint counts (83%), and muscle enzymes (94%) were the most utilized skin/musculoskeletal measures (Figure 1). Most respondents were unfamiliar with hand measures used to assess adult SSc (17-58%). However, finger to palm measure was always used by 43% of respondents (Figure 2).

Conclusion: This scoping review identified measures used to assess cutaneous and musculoskeletal manifestations of SSc. Our results highlight underrepresentation of jSSc and overlap subtype in the literature. A survey of current clinical practice highlights significant variation and unfamiliarity of measures of the musculoskeletal and hand domains respectively. Despite the prevalence of these domains in SSc, they are understudied, highlighting the need for additional studies to generate evidence-based recommendations.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/outcome-measures-for-the-assessment-of-cutaneous-and-musculoskeletal-manifestations-of-juvenile-systemic-sclerosis-an-international-collaborative-scoping-review-and-survey-of-current-practice/