0515: Juvenile Systemic Sclerosis Treatment Practices in an International Cohort and Comparison to Recent SHARE Consensus Guidelines

Ivan Foeldvari¹, Jens Klotsche², Ozgur Kasapcopur³, Amra Adrovic⁴, Kathryn Torok⁵, Maria Teresa Terreri⁶, Ana Paula Sakamoto⁷, Brian Feldman⁸, Jordi Anton⁹, FLAVIO SZTAJNBOK¹⁰, Valda Stanevica¹¹, Simone Appenzeller¹², Tadey Avcin¹³, Sindu Johnson¹⁴, Raju Khubchandani¹⁵, Mikhail Kostik¹⁶, Edoardo Marrani¹⁷, Walter Alberto Sifuentes-Giraldo¹⁸, Dana Nemcova¹⁹, Maria José Santos²⁰, Dieneke Schonenberg-Meinema²¹, Cristina Battagliotti²², Lillemor Berntson²³, Blanca Bica²⁴, Jürgen Brunner²⁴, Rolando Cimaz²⁵, Despina Eleftheriou²⁶, Liora Harel²⁷, Gerd Horneff²⁸, Mahesh Janarthanan²⁹, Tilmann Kallinich³⁰, Thomas Lehman³¹, Farzana Nuruzzaman³², Anjali Patwardhan³³, Vanessa Smith³⁴ and Nicola Helmus³⁵, 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, ⁷Federal University of Sao Paulo (UNIFESP), São Paulo, Brazil, ⁸Division of Rheumatology, The Hospital for Sick Children; Child Health Evaluative Services, SickKids Research Institute; Department of Paediatrics, University of Toronto, Toronto, ON, Canada, ⁹Pediatric Rheumatology, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain, ¹⁰UFRJ/UERJ, São Paulo, Brazil, ¹¹Children's Clinical University Hospital, Zemgales priekšpilseta, Riga, Latvia, ¹²Unicamp, Campinas, São Paulo, Brazil, ¹³University Children's Hospital University Medical Center Ljubljana, Ljubljana, Slovenia, ¹⁴University of Toronto, Toronto, ON, Canada, ¹⁵SRCC Children's Hospital, Mumbai, India, ¹⁶Saint-Petersburg State Pediatric Medical University, Saint Petersburg, Russia, ¹⁷University of Florence, Firenze, Italy, ¹⁸Hospital Universitario Ramon y Cajal, Madrid, Spain, ¹⁹Charles University, Prague, Czech Republic, ²⁰Hospital Garcia de Orta, Almada, Charneca da Caparica, Portugal, ²¹Emma Children’s Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands, ²²Hospital de Niños Dr Orlando Alassia, Santa Fe, Argentina, ²³Dept. of Women’s and Children’s Health, Uppsala University, Uppsala, Sweden, ²⁴UNIVERSIDADE FEDERAL DO RIO DE JANEIRO, Rio de Janeiro, Brazil, ²⁵University of Milano, Milano, Italy, ²⁶Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom, ²⁷Schneider Children's Medical center, Tel Aviv University, Nettnja, Israel, ²⁸Pediatrics, Asklepios Klinik Sankt Augustin GmbH, Sankt Augustin, Germany, ²⁹SRI RAMACHANDRA INSTITUTE OF HIGHER EDUCATION AND RESEARCH, Chennai, India, ³⁰Charité - Universitätsmedizin Berlin, Nuremberg, Germany, ³¹Hospital for Special Surgery, New York, NY, ³²Stony Brook Children's Hospital, East Setauket, NY, ³³University of Missouri, Columbia, MO, ³⁴Department 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, ³⁵Hamburg Centre for Pediatric and Adolescence Rheumatology, Hamburg, Germany

Background/Purpose: Juvenile systemic scleroderma (jSSc) is an orphan disease with a prevalence of 3 in 1,000,000 children. Currently no medications are licensed for the treatment of jSSc. Due to its rarity, only recently have the first management and treatment guidelines been published, the jSSc SHARE (Single Hub and Access point for paediatric Rheumatology in Europe) recommendations, reflecting consensus opinion upon pediatric rheumatologists (1).

Methods: The juvenile systemic sclerosis inceptions cohort (jSScC) is a multinational cohort that prospectively collects clinical data, including medications at baseline and subsequent visits. The jSScC enrollment criteria include age of onset of the first non-Raynaud symptom younger than 16 years and age younger than 18 years at cohort entrance. The frequency of medications (general category and specific medication) was calculated across the cohort at timepoint 0 (enrollment), 12 months and 24 months.

Results: We extracted data from the jSScC of patients who were followed for 12 or 24 months. One-hundred and nine patients were followed at time point 0 (T0) and 12 months (T12), and data was available for 77 of them up at 24 months (T24). The mean age of the patients was 13.2 years at the timepoint 0. Three-quarters were female and 75 % had diffuse subtype. Disease duration at baseline visit was 3.1 years. The medications the patients were on recorded by the physician were captured at T0, T12 and T24 listed in Table 1.

Conclusion: At baseline half of the patients were on corticosteroids. This is more frequent than typical adult SSc practice but coincides with jSSc SHARE treatment recommendations (#1)(1). After 12 months observation in the cohort over 90% of patients received a DMARD therapy. Methotrexate and mycophenolate mofetil were the most commonly prescribed DMARDs, which also reflects the SHARE treatment recommendations (#2 and #3). At 12 months the use of glucocorticoid decreased and the use of bDMARDs increased. In general, biological DMARDs are typically considered in severe or refractory (SHARE recommendation #7), reflecting the lower percentage compared to csDMARDs. Autologous stem cell transplantation was observed in one patient at 12 months, reflecting an option in jSSc with progressive and refractory disease (SHARE recommendation #8). Endothelial receptor antagonists, such as bosentan, were used over time in approximately 20% of the patients, reflecting SHARE recommendation #6 for pulmonary hypertension and/or digital tip ulcers. This is the first evaluation looking at clinical medication practice pattern in jSSc, and its comparison to recently published consensus guidelines.

References:

1. Foeldvari I, Culpo R, Sperotto F, Anton J, Avcin T, Baildam E, et al. Consensus-based recommendations for the management of juvenile systemic sclerosis. Rheumatology (Oxford). 2021;60(4):1651-8.


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; A. Sakamoto, None; 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.; F. SZTAJNBOK, None; V. Stanevica, None; S. Appenzeller, None; T. Avcin, None; S. Johnson, None; R. Khubchandani, None; M. Kostik, None; E. Marrani, None; W. Sifuentes-Giraldo, None; D. Nemcova, None; M. Santos, AbbVie/Abbott, AstraZeneca, pfizer, Novartis, Eli Lilly; D. Schonenberg-Meinema, None; 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; T. Lehman, None; F. Nuruzzaman, None; A. Patwardhan, None; V. Smith, Boehringer-Ingelheim, Janssen; N. Helmus, None.