Artikel
Patient and physician reported outcomes of juvenile systemic sclerosis patients significantly improve over 12 months observation period in the juvenile systemic scleroderma inception cohort
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Autoren
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Ivan Foeldvari
- Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg
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Jens Klotsche
- German Rheumatism Research Center, Berlin
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Ozgur Kasapcopur
- jSSc collaborative group, Hamburg
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Amra Adrovic
- jSSc collaborative group, Hamburg
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Kathryn Torok
- jSSc collaborative group, Hamburg
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Maria Teresa Terreri
- jSSc collaborative group, Hamburg
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Brian Feldman
- jSSc collaborative group, Hamburg
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Jordi Anton
- jSSc collaborative group, Hamburg
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Maria Katsicas
- jSSc collaborative group, Hamburg
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Valda Stanevicha
- jSSc collaborative group, Hamburg
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Flavio Sztajnbok
- jSSc collaborative group, Hamburg
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Simone Appenzeller
- jSSc collaborative group, Hamburg
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Tadey Avcin
- jSSc collaborative group, Hamburg
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Mikhail Kostik
- jSSc collaborative group, Hamburg
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Edoardo Marrani
- jSSc collaborative group, Hamburg
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W.-Alberto Sifuentes-Giraldo
- jSSc collaborative group, Hamburg
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Sindhu Johnson
- jSSc collaborative group, Hamburg
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Raju Khubchandani
- jSSc collaborative group, Hamburg
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Dana Nemcova
- jSSc collaborative group, Hamburg
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Maria Jose Santos
- jSSc collaborative group, Hamburg
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Christina Battagliotti
- jSSc collaborative group, Hamburg
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Lillemor Berntson
- jSSc collaborative group, Hamburg
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Blanca Bica
- jSSc collaborative group, Hamburg
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Juergen Brunner
- jSSc collaborative group, Hamburg
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Rolando Cimaz
- jSSc collaborative group, Hamburg
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Despina Eleftheriou
- jSSc collaborative group, Hamburg
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Liora Harel
- jSSc collaborative group, Hamburg
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Gerd Horneff
- jSSc collaborative group, Hamburg
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Mahesh Janarthanan
- jSSc collaborative group, Hamburg
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Tilmann Kallinich
- jSSc collaborative group, Hamburg
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Kirsten Minden
- jSSc collaborative group, Hamburg
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Monika Moll
- jSSc collaborative group, Hamburg
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Susan Nielsen
- jSSc collaborative group, Hamburg
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Anjali Patwardhan
- jSSc collaborative group, Hamburg
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Dieneke Schonenberg
- jSSc collaborative group, Hamburg
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Vanessa Smith
- jSSc collaborative group, Hamburg
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Nicola Helmus
- Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg
| Veröffentlicht: | 31. August 2022 |
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Gliederung
Text
Introduction: Juvenile systemic sclerosis (jSSc) is an orphan disease with a prevalence of 3 in 1,000,000 children [1]. 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 [2], [3]. We reviewed jSScC patient clinical data and patient and physician reported outcomes, who had 12 months follow up from the time of inclusion until 1st of 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.
Disclosures: Supported by the “Joachim Herz Stiftung”.
Webseite: https://www.juvenile-scleroderma.com/
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. J Scleroderma Relat Disord. 2018 Jun;3(2):189-90. DOI: 10.1177/2397198318763701
- 2.
- Foeldvari I, Klotsche J, Kasapcopur O, Adrovic A, Terreri MT, Sakamoto AP, Stanevicha V, Sztajnbok F, Anton J, Feldman B, Alexeeva E, Katsicas M, Smith V, Avcin T, Marrani E, Kostik M, Lehman T, Sifuentes-Giraldo WA, Vasquez-Canizares N, Appenzeller S, Janarthanan M, Moll M, Nemcova D, Patwardhan A, Santos MJ, Sawhney S, Schonenberg-Meinema D, Battagliotti C, Berntson L, Bica B, Brunner J, Costa-Reis P, Eleftheriou D, Harel L, Horneff G, Kaiser D, Kallinich T, Lazarevic D, Minden K, Nielsen S, Nuruzzaman F, Uziel Y, Helmus N, Torok KS. Differences Sustained Between Diffuse and Limited Forms of Juvenile Systemic Sclerosis in an Expanded International Cohort. Arthritis Care Res (Hoboken). 2021 Mar 30. DOI: 10.1002/acr.24609
- 3.
- Foeldvari I, Klotsche J, Torok KS, Kasapcopur O, Adrovic A, Stanevica V, et al. Characteristics of the first 80 patients at timepoint of first assessment included in the juvenile systemic sclerosis inception cohort. J Scleroderma Relat Disord. 2018;4(1-13).
