POS0140 PATIENT AND PHYSICIAN REPORTED OUTCOMES OF JUVENILE SYSTEMIC SCLEROSIS PATIENTS SIGNIFICANTLY IMPROVE OVER 24 MONTHS OBSERVATION PERIOD IN THE JUVENILE SYSTEMIC SCLERODERMA INCEPTION COHORT

Abstract

<h3>Background</h3> Juvenile systemic sclerosis (jSSc) is an orphan disease with a prevalence of 3 in 1 000 000 children. The Juvenile Systemic Scleroderma Inception cohort (jSScC) is the largest cohort of jSSc patients in the world. The jSScC collects data prospectively in jSSc, allowing the evaluation of the development of organ involvement and patient and physician reported outcomes in jSSc over time. <h3>Objectives</h3> To review the changes in the clinical characteristics, and patient and physician reported outcomes, over a 24 month observation period from the time of inclusion into the cohort. <h3>Methods</h3> The jSScC cohort enrolls jSSc patients who developed the first non-Raynaud´s symptom before the age of 16 years and are under the age of 18 years at the time of inclusion. We reviewed jSScC patient clinical data and patient and physician reported outcomes, who had 24 months follow up from the time of inclusion until 1^st of December 2022. <h3>Results</h3> We extracted data from 90 patients, 77% of them had the diffuse subtype. The female/male ratio was 3.5:1. Median age of onset of Raynaud´s was 9.4 years and the median age of onset of non-Raynaud´s was 10.0 years. Eighty-nine percent of the patients were treated with disease modifying anti-rheumatic drugs (DMARDs) at time of inclusion in the cohort (T0) and 96% after 24 months (T24). Median disease duration was 2.4 years at T0. No patient died during the follow up. Antibody profile stayed unchanged. Only 3 clinical parameters changed and all improved significantly, the median modified Rodnan skin score improved from 11 to 8 (p=0.021), number of patients with joints with pain on motion decreased from 21% to 10% (p=0.04) and the number of patients with muscle weakness decreased from 13% to 4% (p=0.03). All other organ involvement did not show any statistically significant change from T0 to T24. All collected patient reported outcomes improved significantly from T0 to T24: the patient reported disease activity by VAS 0-100 from 40 to 20 (p=0.001), the patient reported disease damage by VAS 0-100 from 35 to 20 (p=0.027), patient reported ulceration activity by VAS 0-100) from 8 to 0 (p=0.001) and the patient reported Raynaud activity by VAS 0-100 from 20 to 10 (p=0.002). Two of the three physician reported outcomes improved significantly, the physician global disease activity by VAS 0-100 from 30 to 20 (p=0.001) and physician reported ulceration activity by VAS 0-100 from 5 to 0 (p=0.017). (Table 1.) <h3>Conclusion</h3> Skin and musculoskeletal clinical features improved over 24 months, with almost all patients on DMARDs, supporting the response of these features to therapy. It was promising that internal organ involvement, like cardiac, lung and gastrointestinal, did not significantly worsen or increase. The most striking observation is the positive direction and improvement all patients and two of the three physician reported outcome measures over 24 months in this large international cohort. <h3>REFERENCES:</h3> NIL. <h3>Acknowledgements:</h3> NIL. <h3>Disclosure of Interests</h3> None Declared.