gms | German Medical Science

Deutscher Rheumatologiekongress 2022, 50. Kongress der Deutschen Gesellschaft für Rheumatologie (DGRh), 36. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie (DGORh), 32. Jahrestagung der Gesellschaft für Kinder- und Jugendrheumatologie (GKJR)

31.08. - 03.09.2022, Berlin

Juvenile systemic sclerosis treatment practices in an international cohort and comparison to recent SHARE consensus guidelines

Meeting Abstract

  • Ivan Foeldvari - Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg
  • Jens Klotsche - German Rheumatism Research Center, Berlin
  • Ozgur Kasapcopur - jSSc collaborative group, Hamburg
  • Amra Adrovic - jSSc collaborative group, Hamburg
  • Kathryn Torok - jSSc collaborative group, Hamburg
  • Maria Teresa Terreri - jSSc collaborative group, Hamburg
  • Ana Paula Sakamoto - jSSc collaborative group, Hamburg
  • Brian Feldman - jSSc collaborative group, Hamburg
  • Jordi Anton - jSSc collaborative group, Hamburg
  • Flavio Sztajnbok - jSSc collaborative group, Hamburg
  • Valda Stanevicha - jSSc collaborative group, Hamburg
  • Simone Appenzeller - jSSc collaborative group, Hamburg
  • Tadey Avcin - jSSc collaborative group, Hamburg
  • Sindhu Johnson - jSSc collaborative group, Hamburg
  • Raju Khubchandani - jSSc collaborative group, Hamburg
  • Mikhail Kostik - jSSc collaborative group, Hamburg
  • Edoardo Marrani - jSSc collaborative group, Hamburg
  • W.-Alberto Sifuentes-Giraldo - jSSc collaborative group, Hamburg
  • Dana Nemcova - jSSc collaborative group, Hamburg
  • Maria Jose Santos - jSSc collaborative group, Hamburg
  • Dieneke Schonenberg - jSSc collaborative group, Hamburg
  • Christina Battagliotti - jSSc collaborative group, Hamburg
  • Lillemor Berntson - jSSc collaborative group, Hamburg
  • Blanca Bica - jSSc collaborative group, Hamburg
  • Juergen Brunner - jSSc collaborative group, Hamburg
  • Rolando Cimaz - jSSc collaborative group, Hamburg
  • Despina Eleftheriou - jSSc collaborative group, Hamburg
  • Liora Harel - jSSc collaborative group, Hamburg
  • Gerd Horneff - jSSc collaborative group, Hamburg
  • Mahesh Janarthanan - jSSc collaborative group, Hamburg
  • Tilmann Kallinich - jSSc collaborative group, Hamburg
  • Thomas Lehman - jSSc collaborative group, Hamburg
  • Monika Moll - jSSc collaborative group, Hamburg
  • Farzana Nuruzzaman - jSSc collaborative group, Hamburg
  • Anjali Patwardhan - jSSc collaborative group, Hamburg
  • Vanessa Smith - jSSc collaborative group, Hamburg
  • Nicola Helmus - Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg

Deutsche Gesellschaft für Rheumatologie. Deutsche Gesellschaft für Orthopädische Rheumatologie. Gesellschaft für Kinder- und Jugendrheumatologie. Deutscher Rheumatologiekongress 2022, 50. Kongress der Deutschen Gesellschaft für Rheumatologie (DGRh), 36. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie (DGORh), 32. Jahrestagung der Gesellschaft für Kinder- und Jugendrheumatologie (GKJR). Berlin, 31.08.-03.09.2022. Düsseldorf: German Medical Science GMS Publishing House; 2022. DocKI.02

doi: 10.3205/22dgrh126, urn:nbn:de:0183-22dgrh1262

Veröffentlicht: 31. August 2022

© 2022 Foeldvari et al.
Dieser Artikel ist ein Open-Access-Artikel und steht unter den Lizenzbedingungen der Creative Commons Attribution 4.0 License (Namensnennung). Lizenz-Angaben siehe http://creativecommons.org/licenses/by/4.0/.


Gliederung

Text

Introduction: Juvenile systemic scleroderma (jSSc) is an orphan disease with a prevalence of 3 in 1,000,000 children [1]. 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[2].

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. 109 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. 77% 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 [Tab. 1].

csDMARDs: Conventional synthetic disease-modifying antirheumatic drugs

bDMARDs: Biological disease-modifying antirheumatic drugs

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). 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, #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.

Disclosures: Supported by the “Joachim Herz Stiftung”.


References

1.
Beukelman T, Xie F, Foeldvari I. Assessing the prevalence of juvenile systemic sclerosis in childhood using administrative claims data from the United States. Journal of Scleroderma and Related Disorders. 2018;3(2):189-90.
2.
Foeldvari I, Culpo R, Sperotto F, Anton J, Avcin T, Baildam E, Boros C, Chaitow J, Constantin T, Kasapcopur O, Knupp Feitosa de Oliveira S, Pilkington C, Toplak N, van Royen A, Saad Magalhaes C, Vastert SJ, Wulffraat N, Zulian F. Consensus-based recommendations for the management of juvenile systemic sclerosis. Rheumatology (Oxford). 2021 Apr 6;60(4):1651-8. DOI: 10.1093/rheumatology/keaa584 Externer Link