Background/Purpose: Juvenile Systemic Sclerosis (jSSc) is an autoimmune and fibrosing disease associated with significant morbidity and mortality risk. Data on treatment is limited and based mainly on adult data despite the many differences between adult and jSSc. There is a need for international jSSc consensus tools and definitions in order to conduct jSSc treatment studies

Methods: An international effort primarily between pediatric rheumatologists (PRs) in CARRA and the Pediatric Rheumatology European Society (PRES) is being led by a core group of 3 CARRA and 2 PRES members (NV, SL, MT, CP, FZ). These steps are planned: literature review (LR) on SSc measures, development of surveys to understand current management strategies, and Delphi consensus meetings. The aim of the LR is to identify available measures used in SSc including adults, and their properties for each of 6 areas (cardiac, pulmonary, vascular, gastrointestinal, skin/musculoskeletal, or health-related quality of life). Results will be used to inform the development of surveys evaluating the use and feasibility of these measures, and scoring levels used by PRs. Covidence is being used to facilitate the LR process

Results: We conducted an initial survey of CARRA and a subset of PRES members to understand the prevalence of jSSc, most commonly used assessments and treatments for cardiopulmonary (CP) disease, and identify PRs interested in participating in our study. Response rate was 25% from 348 CARRA, and 35% from 156 PRES members. Responders from 26 countries follow 649 jSSc patients, with a median of 3 patients followed per center (IQR 2-7). Frequency use of CP assessment tools and proportion of most frequently used immunosuppressive treatments by PRs are shown in Table 1 and Figure 1. The pulmonary function test measurement considered most important for lung evaluation was the DLCO (75%), whereas echocardiogram (77%) was considered the most important cardiac tool. The 3 most commonly used treatments were oral steroids, methotrexate, and mycophenolate mofetil, each used in about 20% of patients. For our LR, 24,849 unique articles identified from 4 databases were screened by 18 PRs. Inclusion criteria included English language, original peer-reviewed research related to SSc, and including a measure from 1 of the 6 areas of interest. A total of 21,565 articles were eliminated, leaving 3284 articles. These articles have been screened by 34 collaborators for relevance by each of the 6 organ-focused groups. From this screen, 300-600 references remain in each organ group for full text screening to identify articles relevant for data extraction

Conclusion: We have established collaboration with 34 members from 12 countries who will be participating in this effort. Our initial survey of 141 PRs identified variation in preferences for CP assessments and treatments. A high proportion of PRs indicated that they have never used or do not have access to some cardiac assessment tools and biologics to manage their jSSc patients. We completed the first phase of our LR, identifying 300-600 articles in each of the 6 organ-focused groups for further screening followed by data extraction. This effort will generate consensus on outcome measures and definitions to use in future jSSc treatment trials

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/identifying-current-assessment-and-treatment-strategies-in-juvenile-systemic-sclerosis-an-international-effort-to-improve-patient-outcomes/