ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 2207

Improvement Across Physician and Patient Reported Outcome Measures over a 24 Months-time Period in the Juvenile Systemic Scleroderma Inception Cohort

Ivan Foeldvari1, Jens Klotsche2, Ozgur Kasapcopur3, Amra Adrovic4, Kathryn Torok5, Maria Teresa Terreri6, Ana Sakamoto7, Jordi Anton8, Brian Feldman9, Raju Khubchandani10, Tadey Avcin11, Sindhu R. Johnson12, Mikhail Kostik13, Edoardo Marrani14, Flavio Sztajnbok15, Maria Katsicas16, Dana Nemcova17, Maria Jose Santos18, Simone Appenzeller19, Cristina Battagliotti20, Lillemor Berntson21, Jürgen Brunner22, Liora Harel23, Gerd Horneff24, Tilmann Kallinich25, Kirsten Minden2, Farzana Nuruzzaman26, Anjali Patwardhan27, Dieneke Schonenberg-Meinema28 and Nicola Helmus29, 1Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany, 2German Rheumatism Research Center, Berlin, Germany, 3Istanbul University-Cerrahpasa, Cerrahpasa Medical School, istanbul, Turkey, 4Cerrahpasa Medical School, Istanbul, Turkey, 5Division of Rheumatology, Scleroderma Center, Department of Pediatrics, UPMC Children’s Hospital of Pittsburgh, Pittsburgh, PA, USA, Pittsburgh, PA, 6UNIFESP, São Paulo, SP, Brazil, 7Federal University of São Paulo (UNIFESP), São Paulo, SP, Brazil, 8Hospital Sant Joan de Déu. Universitat de Barcelona, Esplugues de Llobregat (Barcelona), Spain, 9Division of Rheumatology, The Hospital for Sick Children; Departments of Pediatrics and Medicine, Faculty of Medicine; The Institute for Health Policy, Management and Evaluation, Dalla Lana School of Public Health, University of Toronto, Toronto, ON, Canada, 10SRCC Childrens Hospital, Mumbai, India, 11University Children's Hospital University Medical Center Ljubljana, Ljubljana, Slovenia, 12University of Toronto, Toronto, Canada, 13Saint-Petersburg State Pediatric Medical University, Saint Petersburg, Russia, 14University of Florence, Firenze, Florence, Italy, 15UFRJ/UERJ, SAO PAULO, Brazil, 16Hospital Garrahan, Buenos Aires, Argentina, 17MD, Prague, Czech Republic, 18Hospital Garcia de Orta and Centro Académico de Medicina de Lisboa, Lisboa, Portugal, 19Unicamp, Campinas, SP, Brazil, 20Hospital de Niños Dr Orlando Alassia, Santa Fe, Argentina, 21Dept. of Women’s and Children’s Health, Uppsala University, Uppsala, Sweden, 22Medical University Innsbruck; Department of Pediatrics, Pediatric Rheumatology, Innsbruck, Austria, 23Faculty of Medicine, Tel Aviv University, Tel Aviv; Pediatric Rheumatology Unit, Schneider Children's Medical Center of Israel, Petach Tikva, Israel, Petach Tikva, HaMerkaz, Israel, 24Asklepios Klinik Sankt Augustin GmbH, Sankt Augustin, Germany, 25Charite, Berlin, Germany, 26Stony Brook Children's Hospital, Stony Brook, NY, 27University of Missouri-Columbia, Department of Child Health, 404 N Keene Street, Columbia MO 65210, Columbia, 28Emma Children’s Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands, 29Hamburg Centre for Pediatric and Adolescence Rheumatology, Hamburg, Germany

Meeting: ACR Convergence 2024

Keywords: ,

Session Information

Date: Monday, November 18, 2024

Title: Pediatric Rheumatology – Clinical Poster III

Session Type: Poster Session C

Session Time: 10:30AM-12:30PM

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 enrols 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 (1, 2). We reviewed jSScC patient clinical data and patient and physician reported outcomes of those with 24 months follow up from the time of inclusion until 1st of April 2024.

Results: We could enrol 101 patients with 24 months follow up. 76% of them had diffuse subtype and 82% of them were Caucasian. Median age of onset of Raynaud´s was 9.6 years and median age at the first non-Raynaud was 10.1 years. 31% of the patients were anti-scl70 positive and 3% anti-centromere positive. The Modified Rodnan Skin Score decreased from 12 to 7.5 (p=0.023). The number of patients with active ulceration remained in the same range 20% at time point “0 “and 18% after 24 months . The number of patients with FVC< 80% increased for 36% to 44% and DLCO< 80% from 49% to 50%, both changes were not significant. The cardiac involvement stayed stable at 3% of the patients. The number of patients with pulmonary hypertension increased 5% to 7% (non-significant). The number of patients with 6 Minute Walk Distance (6MWT) less then 10th percentile decreased significantly from 71% to 57% (p=0.047). No patient developed renal crisis and only 1 patient developed hypertension. The gastrointestinal involvement stayed in the same range with 43% at time point “0” and 37% after 24 months. The number of patients with muscle weakness significantly decreased from 16% to 5% (p=0.019) otherwise lo involvement stayed unchanged. Interestingly all patient and physician reported outcomes improved significantly over 24 months (Table 1.), beside the CHAQ score.

Conclusion: The number of patients with decreased 6MWT under the 10 percentile and muscle weakness improved significantly over 24 months. It is reassuring that major internal organ pattern, such as cardiac, pulmonary, renal and gastrointestinal remained stable. No renal crisis occurred over the 24-month time period. All assessed patient and physician-reported outcomes improved significantly over the 24 months period beside the CHAQ score. It seems to be that the applied therapy made measurable improvement for patients with not many significant changes in the organ involvement pattern.

1. Foeldvari I, et al. Differences Sustained Between Diffuse and Limited Forms of Juvenile Systemic Sclerosis in an Expanded International Cohort. Arthritis Care Res (Hoboken). 2022;74(10):1575-84.

2. Foeldvari I, et al. Characteristics of the first 80 patients  at  timepoint of first assessment included in the juvenile systemic sclerosis inception cohort. Journal of Scleroderma and Related Disorders. 2018;4(1-13).

Supporting image 1

Table 1

Disclosures: I. Foeldvari: Boehringer-Ingelheim, 1, Eli Lilly, 6, miirsubishi, 2; J. Klotsche: None; O. Kasapcopur: None; A. Adrovic: None; K. Torok: None; M. Terreri: None; A. Sakamoto: None; J. Anton: None; B. Feldman: None; R. Khubchandani: None; T. Avcin: None; S. Johnson: None; M. Kostik: None; E. Marrani: None; F. Sztajnbok: None; M. Katsicas: None; D. Nemcova: None; M. Santos: None; S. Appenzeller: None; C. Battagliotti: None; L. Berntson: None; J. Brunner: None; L. Harel: None; G. Horneff: None; T. Kallinich: None; K. Minden: None; F. Nuruzzaman: None; A. Patwardhan: None; D. Schonenberg-Meinema: 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, Anton J, Feldman B, Khubchandani R, Avcin T, Johnson S, Kostik M, Marrani E, Sztajnbok F, Katsicas M, Nemcova D, Santos M, Appenzeller S, Battagliotti C, Berntson L, Brunner J, Harel L, Horneff G, Kallinich T, Minden K, Nuruzzaman F, Patwardhan A, Schonenberg-Meinema D, Helmus N. Improvement Across Physician and Patient Reported Outcome Measures over a 24 Months-time Period in the Juvenile Systemic Scleroderma Inception Cohort [abstract]. Arthritis Rheumatol. 2024; 76 (suppl 9). https://acrabstracts.org/abstract/improvement-across-physician-and-patient-reported-outcome-measures-over-a-24-months-time-period-in-the-juvenile-systemic-scleroderma-inception-cohort/. Accessed .

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