Artikel
After 24 months observation period patient reported outcomes improved significantly in the juvenile Scleroderma Inceptions 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
- Deutsches Rheuma-Forschungszentrum (DRFZ), Programmbereich Epidemiologie, Berlin
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Ozgur Kasapcopur
- Department of Pediatric Nephrology, Cerrahpasa Faculty of Medicine, Istanbul University, Istanbul, Turkey
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Amra Adrovic
- Department of Pediatric Nephrology, Cerrahpasa Faculty of Medicine, Istanbul University, Istanbul, Turkey
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M. T. Terreri
- Universidade Federal de São Paulo, Pediatric Rheumatology, Sao Paulo, Brazil
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Jordi Anton
- University Children’s Hospital, Pediatric Rheumatology, Barcelona, Spain
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Tadey Avcin
- University Childrens Hospital, Pediatric Rheumatology, Ljubljana, Slovenia
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Rolando Cimaz
- University of Florence, Florence, Italy
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Mikhail Kostik
- Saint-Petersburg State Pediatric Medical University, St. Petersburg, Russia
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Maria Katsicas
- Hospital de Pediatria, Buenos Aires, Argentina
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Dana Nemcova
- University Childrens Hospital, Pediatric Rheumatology, Prague, Czech Republic
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Maria Jose Santos
- Serviço de Reumatologia, Hospital Garcia de Orta, Almada, Portugal
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Cristina Battagliotti
- Hospital den Ninos Dr. Orlando Alassia, Santa Fe, Argentine
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Lillemor Berntson
- Dept. of Pediatrics, Uppsala University Hospital, Uppsala, Sweden
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Jürgen Brunner
- Medizinische Universität Innsbruck, Department für Kinder- und Jugendheilkunde, Innsbruck, Österreich
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Liora Harel
- Pediatric Rheumatology, Nettnja, Israel
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Tilmann Kallinich
- Charité - Universitätsmedizin Berlin, Klinik für Pädiatrie mit Schwerpunkt Pneumologie und Immunologie, Sektion Rheumatologie, Berlin
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Kirsten Minden
- Deutsches Rheuma-Forschungszentrum (DRFZ) und Charité Universitätsmedizin Berlin, Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie, Berlin
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Monika Moll
- Universitätsklinikum Tübingen, Klinik für Kinder- und Jugendmedizin, Tübingen
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Anjali Patwardhan
- University of Missouri, Columbia, USA
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Kathryn Torok
- University Childrens Hospital, Pittsburgh, United States of America
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Nicola Helmus
- Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg
| Veröffentlicht: | 8. Oktober 2019 |
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Gliederung
Text
Background: Juvenile systemic sclerosis (jSSc) is an orphan disease with a prevalence of 3 in a 1 000 000 children. Currently there is nearly no data published about the course of jSSc patients with standardized assessment. We report our date from Juvenile Scleroderma Inception cohort (JSSIC) with a follow up of 24 months.
Methods: The JSSIC is a prospective multicenter registry of patients with jSSc, who fulfill the adult classification criteria, and presented the first non-Raynaud symptom before 16 years of age and were younger then 18 years at the time of inclusion in the JSSIC. Patients who were followed at least 24 months in the JSSIC, were evaluated.
Results: 52 patients were followed at least 24 months in the registry. 77 % were female and 77% had diffuse subtype. 19% had overlap features. Mean disease duration at time of inclusion was 3.2 years. Mean age of at Raynaud’s onset was 8.8 years and the first non-Raynaud’s symptom 9.4 years. 85% received DMARDs at the time of inclusion and 96 % after 24 months. 88% of the patients were ANA positive, 35% anti-Scl70 positive and 3% anticentromere positive. The mean modified skin score decreased from 14.3 to 12.6. The frequency of Raynaud’s stayed around 87%. The frequency of the nailfold capillary changes increased from 56% to 63%, but the frequency of active ulcerations stayed stable around 21%. The number of patients with FVC <80 % decreased from 39 % to 34% (p=0.734). The number of patients with pulmonary hypertension assessed by ultrasound increased from 4% to 8% (p=0.652). No patient developed systemic hypertension or renal crisis. Urinary sedimentary changes decreased from 8% to 4%. Gastrointestinal involvement decreased from 33% to 29% (p=0.829). Number of joints with decreased range increased from 46% to 63% (p=0.076). Total muscle weakness decreased from 8 % to 3% (p=0.237) and elevated CK from 22% to 9% (p=0.033) too.
Several PROs improved significantly. Patient global disease activity (VAS 0-100) changed from 46 to 29 (p=0.002), patient global disease damage (VAS 0-100) from 46 to 28 (p=0.02) and patient Raynaud activity VAS 0-100) from 27 to 14 (p=0.009) as physician global disease activity (VAS 0-100) from 43 to 29 (p=0.021) and physician global disease damage from 46 to 28 (P=0.01).
Conclusion: Over the 24 months observation period patient and physician related outcomes improved significantly. Regarding organ involvement there was an increase in patients of pulmonary hypertension and joint contractures.
Supported by the "Joachim Herz Stiftung"
