1367: Patient and Physician Reported Outcomes of Juvenile Systemic Sclerosis Patients Significantly Improve over 12 Months Observation Period in the Juvenile Systemic Scleroderma Inception Cohort

Ivan Foeldvari¹, Jens Klotsche², Ozgur Kasapcopur³, Amra Adrovic⁴, Kathryn Torok⁵, Maria Teresa Terreri⁶, Brian Feldman⁷, Jordi Anton⁸, Maria Katsicas⁹, Valda Stanevica¹⁰, FLAVIO SZTAJNBOK¹¹, Simone Appenzeller¹², Tadey Avcin¹³, Mikhail Kostik¹⁴, Edoardo Marrani¹⁵, Walter Alberto Sifuentes-Giraldo¹⁶, Sindu Johnson¹⁷, Raju Khubchandani¹⁸, Dana Nemcova¹⁹, Maria José Santos²⁰, Cristina Battagliotti²¹, Lillemor Berntson²², Blanca Bica²³, Jürgen Brunner²³, Rolando Cimaz²⁴, Despina Eleftheriou²⁵, Liora Harel²⁶, Gerd Horneff²⁷, Mahesh Janarthanan²⁸, Tilmann Kallinich²⁹, Kirsten Minden³⁰, Anjali Patwardhan³¹, Dieneke Schonenberg-Meinema³², Vanessa Smith³³ and Nicola Helmus³⁴, ¹Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany, ²German Rheumatism Research Center, Berlin, Germany, ³Istanbul University-Cerrahpaşa, Cerrahpaşa Medical School, Istanbul, Turkey, ⁴Cerrahpaşa Medical School, Istanbul University, Istanbul, Turkey, ⁵Pediatric Rheumatology, Children's Hospital of UPMC, Pittsburgh, PA, ⁶Universidad Federal São Paulo, São Paulo, Brazil, ⁷Division of Rheumatology, The Hospital for Sick Children; Child Health Evaluative Services, SickKids Research Institute; Department of Paediatrics, University of Toronto, Toronto, ON, Canada, ⁸Pediatric Rheumatology, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain, ⁹Hospital Garrahan, Buenos Aires, Argentina, ¹⁰Children's Clinical University Hospital, Zemgales priekšpilseta, Riga, Latvia, ¹¹UFRJ/UERJ, São Paulo, Brazil, ¹²Unicamp, Campinas, São Paulo, Brazil, ¹³University Children's Hospital University Medical Center Ljubljana, Ljubljana, Slovenia, ¹⁴Saint-Petersburg State Pediatric Medical University, Saint Petersburg, Russia, ¹⁵University of Florence, Firenze, Italy, ¹⁶Hospital Universitario Ramon y Cajal, Madrid, Spain, ¹⁷University of Toronto, Toronto, ON, Canada, ¹⁸SRCC Children's Hospital, Mumbai, India, ¹⁹Charles University, Prague, Czech Republic, ²⁰Hospital Garcia de Orta, Almada, Charneca da Caparica, Portugal, ²¹Hospital de Niños Dr Orlando Alassia, Santa Fe, Argentina, ²²Dept. of Women’s and Children’s Health, Uppsala University, Uppsala, Sweden, ²³UNIVERSIDADE FEDERAL DO RIO DE JANEIRO, Rio de Janeiro, Brazil, ²⁴University of Milano, Milano, Italy, ²⁵Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom, ²⁶Schneider Children's Medical center, Tel Aviv University, Nettnja, Israel, ²⁷Pediatrics, Asklepios Klinik Sankt Augustin GmbH, Sankt Augustin, Germany, ²⁸SRI RAMACHANDRA INSTITUTE OF HIGHER EDUCATION AND RESEARCH, Chennai, India, ²⁹Charité - Universitätsmedizin Berlin, Nuremberg, Germany, ³⁰Charité Universitätsmedizin Berlin, Berlin, Germany, ³¹University of Missouri, Columbia, MO, ³²Emma Children’s Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands, ³³Department of Rheumatology, Ghent University Hospital – Department of Internal Medicine, Ghent University, Belgium – Unit for Molecular Immunology and Inflammation, VIB Inflammation Research Center (IRC), Gent, Belgium, ³⁴Hamburg Centre for Pediatric and Adolescence Rheumatology, Hamburg, Germany

Background/Purpose: 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 longitudinal data prospectively in jSSc, allowing the evaluation of the development of organ involvement and patients and physician reported outcomes in jSSc over time.

Methods: 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 12 months follow up from the time of inclusion until 1^stof December 2021.

Results: 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 significantly, the median modified Rodnan 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 significant change from T0 to T12. All collected patient reported outcomes improved significantly 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 significantly, 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: 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 significantly 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.

This project was supported by an unrestricted grant from "Joachim Herz Stiftung"

Disclosures: I. Foeldvari, None; J. Klotsche, None; O. Kasapcopur, None; A. Adrovic, None; K. Torok, None; M. Terreri, Roche, Pfizer, UCB, Janssen, Bristol-Myers Squibb(BMS), Eli Lilly, AbbVie/Abbott; B. Feldman, Pfizer, AB2-Bio, Janssen; J. Anton, for Sobi, Novimmune, Novartis, Abbvie, Pfizer, GSK, Roche, Amgen, Lilly, BMS, Sanofi, Sobi, Novimmune, Novartis, Pfizer, GSK, Sobi, Novimmune, Novartis, GSK, Pfizer.; M. Katsicas, None; V. Stanevica, None; F. SZTAJNBOK, None; S. Appenzeller, None; T. Avcin, None; M. Kostik, None; E. Marrani, None; W. Sifuentes-Giraldo, None; S. Johnson, None; R. Khubchandani, None; D. Nemcova, None; M. Santos, AbbVie/Abbott, AstraZeneca, pfizer, Novartis, Eli Lilly; C. Battagliotti, None; L. Berntson, Pfizer; B. Bica, None; J. Brunner, None; R. Cimaz, None; D. Eleftheriou, None; L. Harel, None; G. Horneff, Roche, Pfizer, Novartis, Merck/MSD, Eli Lilly, AbbVie/Abbott; M. Janarthanan, None; T. Kallinich, Roche; K. Minden, Novartis, Pfizer, Bristol-Myers Squibb(BMS), Roche, Biogen; A. Patwardhan, None; D. Schonenberg-Meinema, None; V. Smith, Boehringer-Ingelheim, Janssen; N. Helmus, None.