No Disease Progression After 36 Months Follow up in the Juvenile Systemic Scleroderma Inception Cohort

Ivan Foeldvari¹, Jens Klotsche², Ozgur Kasapcopur³, Amra Adrovic⁴, Maria Teresa Terreri⁵, Edoardo Marrani⁶, Tadej Avcin⁷, Maria Katsicas⁸, Dana Nemcova⁹, Maria Jose Santos¹⁰, Jürgen Brunner¹¹, Tilmann Kallinich¹², Mikhail Kostik¹³, Kirsten Minden¹⁴, Anjali Patwardhan¹⁵, Kathryn Torok¹⁶ and Nicola Helmus¹⁷, ¹Head of the Hamburg Centre for Pediatric and Adolescence Rheumatology, Budapest, Hungary, ²German Rheumatism Research Center, Berlin, Germany, ³PRINTO, Istituto Giannina Gaslini, Genova, Italy, ⁴Cerrahpaşa Tıp Fakültesi, Istanbul, Turkey, ⁵Federal University of São Paulo, São Paulo, Brazil, ⁶University of Florence, Firenze, Italy, ⁷Ljubljana University Medical Centre, Ljubljana, Slovenia, ⁸Hospital de Pediatria, Buenos Aires, Argentina, ⁹General University Hospital, Prague, Czech Republic, ¹⁰Hospital Garcia de Orta, Almada, Portugal, ¹¹Tirol Kliniken, Innsbruck, Innsbruck, Austria, ¹²Charite, Berlin, Germany, ¹³Saint-Petersburg State Pediatric Medical University, Saint Petersburg, Russia, ¹⁴Deutsches Rheuma-Forschungszentrum Berlin, Berlin, Germany, ¹⁵University of Missouri, Columbia, ¹⁶University of Pittsburgh, Pittsburgh, PA, ¹⁷Hamburg Centre for Pediatric and Adolescence Rheumatology, Hamburg, Germany

Meeting: ACR Convergence 2020

Keywords: Outcome measures, Pediatric rheumatology, Scleroderma, Systemic

Session Information

Date: Sunday, November 8, 2020

Session Title: Pediatric Rheumatology – Clinical Poster II: Systemic JIA, Autoinflammatory, & Scleroderma

Session Type: Poster Session C

Session Time: 9:00AM-11:00AM

Background/Purpose: Juvenile systemic scleroderma (jSSc) is an orphan disease with a prevalence of 3 in 1 000 000 children. Longitudinal prospective follow up data of patients with jSSc is rare. In the international juvenile systemic scleroderma cohort (jSScC) patients are followed with a standardized assessment prospectively.

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 and 80% female. 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. 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 assessed on a VAS scale of 0 to 100. The physician global disease activity decreased from 40.0 to 22.1 (p < 0.001).

Patients global disease activity decreased from 43.3 to 20.4 and patients global disease damage from 45.0 to 21.7 (p< 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: I. Foeldvari, Sanofi, 5, Chugai, 5, Amgen, 5, GSK, 5, Lilly, 5, BMS, 5, Abbvie, 5, Novartis, 5, gilead, 5; J. Klotsche, None; O. Kasapcopur, None; A. Adrovic, None; M. Terreri, None; E. Marrani, None; T. Avcin, AbbVie, 5, 8, Alexion, 5, Octapharma, 5, 8, Takeda, 5, 8; M. Katsicas, None; D. Nemcova, None; M. Santos, None; J. Brunner, None; T. Kallinich, None; M. Kostik, None; K. Minden, Sanofi, 1, gsk, 1, Roche, 1, Abbvie, 1, Biermann, 8, Medac, 8; A. Patwardhan, None; K. Torok, None; N. Helmus, None.

To cite this abstract in AMA style:

Foeldvari I, Klotsche J, Kasapcopur O, Adrovic A, Terreri M, Marrani E, Avcin T, Katsicas M, Nemcova D, Santos M, Brunner J, Kallinich T, Kostik M, Minden K, Patwardhan A, Torok K, Helmus N. No Disease Progression After 36 Months Follow up in the Juvenile Systemic Scleroderma Inception Cohort [abstract]. Arthritis Rheumatol. 2020; 72 (suppl 10). https://acrabstracts.org/abstract/no-disease-progression-after-36-months-follow-up-in-the-juvenile-systemic-scleroderma-inception-cohort/. Accessed July 30, 2021.

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