ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 1364

Diffuse Juvenile Systemic Sclerosis Patients Show Distinct Organ Involvement and Have More Severe Disease in the Largest jSSc Cohort of the World. Results from the the Juvenile Scleroderma Inception Cohort

Ivan Foeldvari1, Jens Klotsche2, Ozgur Kasapcopur3, Amra Adrovic4, Kathryn Torok5, Maria Teresa Terreri6, Ana Paula Sakamoto7, Brian Feldman8, FLAVIO SZTAJNBOK9, Valda Stanevica10, Jordi Anton11, Sindu Johnson12, Raju Khubchandani13, Ekaterina Alexeeva14, Maria Katsicas15, Sujata Sawhney16, Vanessa Smith17, Simone Appenzeller18, Tadey Avcin19, Mikhail Kostik20, Thomas Lehman21, Hana Malcova22, Edoardo Marrani23, Clare Pain24, Dieneke Schonenberg-Meinema25, Walter Alberto Sifuentes-Giraldo26, Natalia Vasquez-Canizares27, Patricia Costa Reis28, Mahesh Janarthanan29, Dana Nemcova30, Anjali Patwardhan31, Maria José Santos32, Sima Abu Alsaoud33, Cristina Battagliotti34, Lillemor Berntson35, Blanca Bica36, Jürgen Brunner36, Rolando Cimaz37, Despina Eleftheriou38, Liora Harel39, Gerd Horneff40, Daniela Kaiser41, Tilmann Kallinich42, Dragana Lazarevic43, Farzana Nuruzzaman44 and Nicola Helmus45, 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, 9UFRJ/UERJ, São Paulo, Brazil, 10Children's Clinical University Hospital, Zemgales priekšpilseta, Riga, Latvia, 11Pediatric Rheumatology, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain, 12University of Toronto, Toronto, ON, Canada, 13SRCC Children's Hospital, Mumbai, India, 14National Medical Research Center of Children's Health, Sechenov First Moscow State Medical University of the Ministry of Health of the Russian Federation,, Moscow, Russia, 15Hospital Garrahan, Buenos Aires, Argentina, 16Sir Ganga Ram Hospital, Sector 37 noida, Uttar Pradesh, India, 17Department 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, 18Unicamp, Campinas, São Paulo, Brazil, 19University Children's Hospital University Medical Center Ljubljana, Ljubljana, Slovenia, 20Saint-Petersburg State Pediatric Medical University, Saint Petersburg, Russia, 21Hospital for Special Surgery, New York, NY, 22Motol University Hospital, Prague, Czech Republic, 23University of Florence, Firenze, Italy, 24Alder Hey Children’s NHS Foundation Trust, Liverpool, United Kingdom, 25Emma Children’s Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands, 26Hospital Universitario Ramon y Cajal, Madrid, Spain, 27Children’s Hospital at Montefiore, Bronx, NY, 28Hospital de Santa Maria, Lisbon, Portugal, 29SRI RAMACHANDRA INSTITUTE OF HIGHER EDUCATION AND RESEARCH, Chennai, India, 30Charles University, Prague, Czech Republic, 31University of Missouri, Columbia, MO, 32Hospital Garcia de Orta, Almada, Charneca da Caparica, Portugal, 33Makassed, Jerusalem, Israel, 34Hospital de Niños Dr Orlando Alassia, Santa Fe, Argentina, 35Dept. of Women’s and Children’s Health, Uppsala University, Uppsala, Sweden, 36UNIVERSIDADE FEDERAL DO RIO DE JANEIRO, Rio de Janeiro, Brazil, 37University of Milano, Milano, Italy, 38Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom, 39Schneider Children's Medical center, Tel Aviv University, Nettnja, Israel, 40Pediatrics, Asklepios Klinik Sankt Augustin GmbH, Sankt Augustin, Germany, 41Children's Hospital Lucerne, Lucerne, Switzerland, 42Charité - Universitätsmedizin Berlin, Nuremberg, Germany, 43Faculty of Medicine, University of Niš, Nis, Serbia, 44Stony Brook Children's Hospital, East Setauket, NY, 45Hamburg Centre for Pediatric and Adolescence Rheumatology, Hamburg, Germany

Meeting: ACR Convergence 2022

Keywords: , ,

Session Information

Date: Sunday, November 13, 2022

Title: Pediatric Rheumatology – Clinical Poster II: Connective Tissue Disease

Session Type: Poster Session C

Session Time: 1:00PM-3:00PM

Background/Purpose: Juvenile systemic sclerosis (jSSc) is an orphan disease with a prevalence of 3 in 1 000 000 children. In adult patients there are significant differences between the clinical presentation of diffuse and limited subtypes. We reviewed clinical differences in presentation of subtypes in patients in the juvenile systemic scleroderma inception cohort up to 2021.

Methods: We reviewed the clinical baseline characteristics of the patients, who were recruited to the juvenile scleroderma inception cohort (jSScC) till 1st of December 2021. jSScC is a prospective cohort of 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.

Results: 210 patients with jSSc were included in the cohort, 71% (n=162) had diffuse subtype. The median age at onset of Raynaud phenomenon was 10.4 years (7.3 – 12.9) and the median age at the first non-Raynaud symptom was 10.9 years (7.4 – 13.2). Median disease duration was 2.5 years (1 – 4.4) at the time of inclusion. The female/male ratio was significantly lower in the djSSc subtype (3.7:1 versus 5:1, p< 0.001). Antibody profile was quite similar, with the exception of a significantly higher number of anticentromere positive patients in the ljSSc (12% versus 2%, p=0.013). Decreased FVC < 80% was found in approximately 30% and decreased DLCO < 80% was found in around 40% in both subtypes. Pulmonary hypertension assessed by ultrasound was identified in 5% in both groups. Patients with diffuse subtype had significantly higher modified Rodnan Skin Score (mRSS) (16 versus 4.5, p< 0.001), sclerodactyly (84% versus 60%, p< 0.001), history of digital ulceration (62% versus 31%, p< 0.001), decreased Body Mass Index (BMI) ≤ -2 z score (20% versus 4%, p=0.003) and decreased joint range of motion (64% versus 46%, p=0.019). Patients with ljSSc had significantly higher rate of cardiac involvement (13% versus 2%, p=0.001).

Regarding patient related outcomes djSSc patients had more severe disease, looking at patient reported global disease activity (VAS 0 – 100) (40 versus 25, p=0.039), patient reported global disease damage (VAS 0 – 100) (40 versus 25, p=0.021) and patient reported assessment of ulceration activity (10 versus 0, p=0.044). Regarding physician related outcomes the physician reported global disease activity (VAS 0 – 100) (32 versus 20, p< 0.001) and physician reported global disease damage (VAS 0 – 100) (30 versus 15, p=0.014) was significantly higher in djSSc.

Conclusion: In this jSSc cohort, the largest in the world, djSSc patients have a significantly more severe disease than ljSSc patients. Interestingly, we found no differences regarding interstitial lung disease and pulmonary hypertension.

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; F. SZTAJNBOK, None; V. Stanevica, None; J. Anton, for Sobi, Novimmune, Novartis, Abbvie, Pfizer, GSK, Roche, Amgen, Lilly, BMS, Sanofi, Sobi, Novimmune, Novartis, Pfizer, GSK, Sobi, Novimmune, Novartis, GSK, Pfizer.; S. Johnson, None; R. Khubchandani, None; E. Alexeeva, None; M. Katsicas, None; S. Sawhney, None; V. Smith, Boehringer-Ingelheim, Janssen; S. Appenzeller, None; T. Avcin, None; M. Kostik, None; T. Lehman, None; H. Malcova, None; E. Marrani, None; C. Pain, None; D. Schonenberg-Meinema, None; W. Sifuentes-Giraldo, None; N. Vasquez-Canizares, None; P. Costa Reis, None; M. Janarthanan, None; D. Nemcova, None; A. Patwardhan, None; M. Santos, AbbVie/Abbott, AstraZeneca, pfizer, Novartis, Eli Lilly; S. Abu Alsaoud, 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; D. Kaiser, None; T. Kallinich, Roche; D. Lazarevic, None; F. Nuruzzaman, None; N. Helmus, None.

To cite this abstract in AMA style:

Foeldvari I, Klotsche J, Kasapcopur O, Adrovic A, Torok K, Terreri M, Sakamoto A, Feldman B, SZTAJNBOK F, Stanevica V, Anton J, Johnson S, Khubchandani R, Alexeeva E, Katsicas M, Sawhney S, Smith V, Appenzeller S, Avcin T, Kostik M, Lehman T, Malcova H, Marrani E, Pain C, Schonenberg-Meinema D, Sifuentes-Giraldo W, Vasquez-Canizares N, Costa Reis P, Janarthanan M, Nemcova D, Patwardhan A, Santos M, Abu Alsaoud S, Battagliotti C, Berntson L, Bica B, Brunner J, Cimaz R, Eleftheriou D, Harel L, Horneff G, Kaiser D, Kallinich T, Lazarevic D, Nuruzzaman F, Helmus N. Diffuse Juvenile Systemic Sclerosis Patients Show Distinct Organ Involvement and Have More Severe Disease in the Largest jSSc Cohort of the World. Results from the the Juvenile Scleroderma Inception Cohort [abstract]. Arthritis Rheumatol. 2022; 74 (suppl 9). https://acrabstracts.org/abstract/diffuse-juvenile-systemic-sclerosis-patients-show-distinct-organ-involvement-and-have-more-severe-disease-in-the-largest-jssc-cohort-of-the-world-results-from-the-the-juvenile-scleroderma-inception-c/. Accessed .

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