FRI0466 NO DISEASE PROGRESSION AFTER 36 MONTHS FOLLOW UP IN THE JUVENILE SYSTEMIC SCLERODERMA INCEPTION COHORT
Authors: I. Foeldvari, J. Klotsche, O. Kasapcopur, A. Adrovic, M. T. Terreri, R. Cimaz, M. Katsikas, D. Nemcova, M. J. Santos, J. Brunner, M. Kostik, K. Minden, A. Patwardhan, K. Torok, N. Helmus
Journal: Annals of the Rheumatic Diseases (2020)

Background: Juvenile systemic scleroderma (jSSc) is an orphan disease with a prevalence of 3 in 1 000 000 children. There is rare longitudinal prospective follow up data of patients with jSSc. In the international juvenile systemic scleroderma cohort (JSScC) patients are followed with a standardized assessment prospectively. Objectives: To assess the changes regarding organ involvement pattern and patients related outcomes after 36 months follow up in the JSScC. Methods: Patients diagnosed according the ACR 2013 criteria for systemic sclerosis were included, if they developed the first non-Raynaud symptom before the age of 16 and were under the age of 18 at the time of inclusion. Patients were followed prospectively every 6 months with a standardized assessment. Results: 39 patients in the JSScC had 36 months follow up. 80% had a diffuse subtype. 95% of the patients were Caucasian origin. 31 of the patients were female (80%). Mean disease duration at time of inclusion was 3.5 years. Mean age onset of Raynaud’s was 8.8 years and mean age of onset at the first non-Raynaud´s was 9.5 years. Around 30% of the patients were anti-Scl70 positive and none of them anti-centromere positive. The MRSS dropped from the time point of the inclusion into the cohort from 13.9 to 11.8 after 36 months. Pattern of organ involvement did not show any significant change, beside the increase of the nailfold capillary changes from 49% to 73% (p=0.037). No renal crisis occurred. No mortality was observed. They were positive significant changes in the patient related outcomes. The physician global disease activity decreased from 40.0 to 22.1 assessed on a VAS scale of 0 to 100 (p &lt;0.001). Patients global disease activity decreased from 43.3 to 20.4 and patients global disease damage from 45.0 to 21.7 both assessed on a VAS scale of 0 to 100 (p&lt;0.001). Conclusion: After 36 months follow up, we could observe a significant improvement of patient related outcomes and only one significant change in organ pattern involvement. In a mostly diffuse subset patient population this is a very promising result regarding outcome. Supported by the “Joachim Herz Stiftung” Disclosure of Interests: Ivan Foeldvari Consultant of: Novartis, Jens Klotsche: None declared, Ozgur Kasapcopur: None declared, Amra Adrovic: None declared, Maria T. Terreri: None declared, Rolando Cimaz: None declared, Maria Katsikas: None declared, Dana Nemcova: None declared, Maria Jose Santos Speakers bureau: Novartis and Pfizer, Juergen Brunner Grant/research support from: Pfizer, Novartis, Consultant of: Pfizer, Novartis, Abbvie, Roche, BMS, Speakers bureau: Pfizer, Novartis, Abbvie, Roche, BMS, Mikhail Kostik: None declared, Kirsten Minden Consultant of: GlaxoSmithKline, Sanofi, Speakers bureau: Roche, Anjali Patwardhan: None declared, Kathryn Torok: None declared, Nicola Helmus: None declared