POS1302 PATIENT AND PHYSICIAN REPORTED OUTCOMES OF JUVENILE SYSTEMIC SCLEROSIS PATIENTS SIGNIFICANTLY IMPROVE OVER 12 MONTHS OBSERVATION PERIOD IN THE JUVENILE SYSTEMIC SCLERODERMA INCEPTION COHORT. WWW.JUVENILE-SCLERODERMA.COM

Abstract

Background: Juvenile systemic sclerosis (jSSc) is an orphan disease with a prevalence of 3 in 1 000 000 children (1). The Juvenile Systemic Scleroderma Inception cohort (jSScC) is the largest cohort of jSSc patients in the world. The jSScC collects longitudinal data prospectively in jSSc, allowing the evaluation of the development of organ involvement and patients and physician reported outcomes in jSSc over time. Objective(s): To review the changes in the clinical characteristics and patient and physician reported outcomes over 12 months observation period from the time of inclusion into the cohort. Method(s): The jSScC cohort enrolls jSSc patients who developed the frst non-Raynaud's symptom before the age of 16 years and are under the age of 18 years at the time of inclusion (2, 3). We reviewed jSScC patient clinical data and patient and physician reported outcomes, who had 12 months follow up from the time of inclusion until 1st of December 2021. Result(s): We could extract data of 113 patients. The female/male ratio was 3.5:1. Median age of onset of Raynaud's was 10.1 years and the median age of onset of non-Raynaud's was 10.8 years. Eighty-eight percent of the patients were treated with disease modifying anti-rheumatic drugs (DMARDs) at time of inclusion in the cohort (T0) and 93% after 12 months (T12). Median disease duration was 2.5 years at T0. Antibody profile stayed unchanged. Only 3 clinical parameters changed and improved signifcantly, the median modifed Rod-nan skin score improved from 13 to 8 (p=0.002), the number of patients with swollen joints decreased from 17% to 8% (p=0.043) and number of patients with joints with pain on motion decreased from 20% to 12% (p=0.048). All other organ involvement did not show any statistically signifcant change from T0 to T12. All collected patient reported outcomes improved signifcantly from T0 to T12: the patient reported disease activity (VAS 0-100) from 40 to 20 (p=0.011), the patient reported disease damage (VAS 0-100) from 40 to 20 (p=0.001), patient reported ulceration activity (VAS 0-100) from 10 to 0 (p=0.02) and the CHAQ score from 0.3 to 0.1 (p=0.002). Two of the three physician reported outcomes improved signifcantly, the physician global disease activity (VAS 0-100) from 30 to 20 (p=0.011) and physician reported global disease damage (VAS 0-100) from 30 to 25 (p=0.028). Conclusion(s): Skin and musculoskeletal clinical features improved over 12 months, with almost all patients on DMARDs, supporting likely response of these features to therapy. It was promising that internal organ involvement, like cardiac and lung, although potentially stable, did not signifcantly worsen or increase. The most striking observation in the positive direction is improvement across several patient and physician reported outcome measures over the 12 month time period in this large international cohort.