Artikel
Diffuse juvenile systemic sclerosis patients show distinct organ involvement, antibody pattern and have significantly more severe disease in the largest jSSc cohort of the world. Results from the juvenile scleroderma inception cohort
Suche in Medline nach
Autoren
-
Ivan Foeldvari
- Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg
-
Jens Klotsche
- German Rheumatism Research Center, Berlin
-
Kathryn Torok
- jSSc collaborative group, Hamburg
-
Ozgur Kasapcopur
- jSSc collaborative group, Hamburg
-
Amra Adrovic
- jSSc collaborative group, Hamburg
-
Maria Teresa Terreri
- jSSc collaborative group, Hamburg
-
Ana Paula Sakamoto
- jSSc collaborative group, Hamburg
-
Brian Feldman
- jSSc collaborative group, Hamburg
-
Flavio Sztajnbok
- jSSc collaborative group, Hamburg
-
Jordi Anton
- jSSc collaborative group, Hamburg
-
Valda Stanevicha
- jSSc collaborative group, Hamburg
-
Sindhu Johnson
- jSSc collaborative group, Hamburg
-
Raju Khubchandani
- jSSc collaborative group, Hamburg
-
Dieneke Schonenberg
- jSSc collaborative group, Hamburg
-
Eslam Al-Abadi
- jSSc collaborative group, Hamburg
-
Ekaterina Alexeeva
- jSSc collaborative group, Hamburg
-
Maria Katsicas
- jSSc collaborative group, Hamburg
-
Sujata Sawhney
- jSSc collaborative group, Hamburg
-
Vanessa Smith
- jSSc collaborative group, Hamburg
-
Simone Appenzeller
- jSSc collaborative group, Hamburg
-
Tadey Avcin
- jSSc collaborative group, Hamburg
-
Mikhail Kostik
- jSSc collaborative group, Hamburg
-
Thomas Lehman
- jSSc collaborative group, Hamburg
-
Hana Malcova
- jSSc collaborative group, Hamburg
-
Edoardo Marrani
- jSSc collaborative group, Hamburg
-
Clare Pain
- jSSc collaborative group, Hamburg
-
Anjali Patwardhan
- jSSc collaborative group, Hamburg
-
W.-Alberto Sifuentes-Giraldo
- jSSc collaborative group, Hamburg
-
Natalia Vasquez-Canizares
- jSSc collaborative group, Hamburg
-
Patricia Costa Reis
- jSSc collaborative group, Hamburg
-
Mahesh Janarthanan
- jSSc collaborative group, Hamburg
-
Dana Nemcova
- jSSc collaborative group, Hamburg
-
Maria Jose Santos
- jSSc collaborative group, Hamburg
-
Sima Abu Al-Saoud
- 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
-
Despina Eleftheriou
- jSSc collaborative group, Hamburg
-
Liora Harel
- jSSc collaborative group, Hamburg
-
Gerd Horneff
- jSSc collaborative group, Hamburg
-
Daniela Kaiser
- jSSc collaborative group, Hamburg
-
Tilmann Kallinich
- jSSc collaborative group, Hamburg
-
Dragana Lazarevic
- jSSc collaborative group, Hamburg
-
Kirsten Minden
- jSSc collaborative group, Hamburg
-
Susan Nielsen
- jSSc collaborative group, Hamburg
-
Farzana Nuruzzaman
- jSSc collaborative group, Hamburg
-
Siri Opsahl Hetlevik
- jSSc collaborative group, Hamburg
-
Yosef Uziel
- jSSc collaborative group, Hamburg
-
Nicola Helmus
- Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg
| Veröffentlicht: | 30. August 2023 |
|---|
Gliederung
Text
Introduction: 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 (jSScC).
Methods: We reviewed the baseline clinical characteristics of the patients, who were recruited to the jSScC till December 2022. 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: The JSScC included 232 patients, 68% (n=159) had diffuse subtype. The median age at onset of Raynaud phenomenon was 10.4 years (7.3–12.9), at the first non-Raynaud symptom 10.9 years (7.3–13.0) and median disease duration 2.5 years (1.0–4.6). The female/male ratio was significantly lower in the djSSc subtype (3:1 versus 5:1, p<0.001). Antibody profile was similar, with the exception of a significantly higher number of anticentromere positive patients in the ljSSc. Decreased FVC<80% was found in approximately 30% and decreased DLCO<80% was found in around 40% in both subtypes. Abnormal HRCT findings were found in 44% of patients. Pulmonary hypertension assessed by ultrasound occurred in approximately 5% in both groups and gastrointestinal involvement in 43% of djSSc and 36% in ljSSc (p=0.303). Patients with djSSc had significantly higher modified Rodnan Skin Score, more frequently sclerodactyly, a history of digital ulceration active ulceration, telangiectasia, a decreased Body Mass Index z score<-2 and decreased joint range of motion. Patients with ljSSc had significantly higher rate of cardiac involvement. Regarding patient related outcomes assessed by VAS 0-100 djSSc patients had more severe disease also physician related outcome assessed by VAS 0-100 were significantly higher in djSSc (see Table 1 [Tab. 1]).
Conclusion: In the largest jSSc cohort in the world, djSSc patients have a significantly more severe disease. Patients and physician related outcomes were significantly more severe in djSSc group. Interestingly, we found no differences regarding interstitial lung disease, pulmonary hypertension or gastrointestinal involvement, although the number of patients with decreased BMI<-2 z score was significantly higher in the djSSc patients.
Disclosure: Supported by the “Joachim Herz Stiftung”.
