SAT0479 UPDATE FROM THE JUVENILE SCLERODERMA INCEPTION COHORT. WWW.JUVENILE-SCLERODERMA.COM
Authors: Ivan Foeldvari, Jens Klotsche, Ozgur Kasapcopur, Amra Adrovic, Kathryn Torok, Valda Stanevicha, Flávio R. Sztajnbok, Maria T. Tererri, Ekaterina Alexeeva, Jordi Anton, Maria Katsikas, Vanessa Smith, Tadej Avcin, Rolando Cimaz, Mikhail Kostik, Thomas Lehman, Walter Alberto Sifuentes-Giraldo, Simone Appenzeller, Mahesh Janarthanan, Monika Moll, Dana Nemcova, Maria Jose Santos, Dieneke Schonenberg, Cristina Battagliotti, Lillemor Berntson, Blanca Bica, Juergen Brunner, Patricia Costa Reis, Despina Eleftheriou, Liora Harel, Gerd Horneff, Tilmann Kallinich, Dragana Lazarevic, Kirsten Minden, Susan Nielsen, Farzana Nuruzzaman, Anjali Patwardhan, Yosef Uziel, Nicola Helmus
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background</h3> Juvenile systemic scleroderma (jSSc) is an orphan disease with a prevalence of 3 in 1 000 000 children. There are limited data regarding the clinical presentation of jSSc. The Juvenile Systemic Scleroderma Inception Cohort (JSSIC) is a multinational registry that prospectively collects information regarding patients with this disease in a standardized manner. <h3>Objectives</h3> Evaluation of the jSSc patients at the time of inclusion in the JSSIC. <h3>Methods</h3> Patients were included in the JSSIC if they fulfilled the adult classification criteria, if they presented the first non Raynaud symptom before 16 years old and if they were younger than 18 years of age at the time of inclusion. Patients' characteristics at time of inclusion were evaluated. <h3>Results</h3> Currently, the cohort includes 120 patients, being 89% Caucasian and 80% female. The majority had diffuse subtype (74%) and 18% had overlap features. The mean age of onset of Raynaud phenomenon was 9.7 years in the diffuse subtype (djSSc) and 10.7 years in the limited subtype (ljSSc). The mean age of non-Raynaud's symptoms was 10.0 years in the djSSc and 11.4 years in the ljSSc (p=0.041). Mean disease duration at time of inclusion was 3.4 years in the djSSc and 2.4 years in the ljSSc group. Mean Modified Rodnan skin score was 17.5 in the djSSc and 7.3 in the ljSSc (p=0.002). Gottron papulae were significantly more common in the djSSc compared to ljSSc group (29% vs 6%, respectively) (p=0.011). History of ulceration was significantly more common in the djSSc than in the ljSSc group (57% vs 30%, respectively) (p=0.004). FVC&lt;80% occurred in 31% in the djSSc and 24% in the ljSSc group (p=0.55). Pulmonary hypertension assessed by echocardiogram occurred around 7% in both groups. No systemic hypertension or renal crisis was reported. Gastrointestinal involvement occurred in 39% in the djSSc and in 26% in the ljSSc (p=0.176). Number of joints with decreased range of motion was observed in approximately half of patients in both groups. Muscle weakness with joint contractures was present in 18% in the djSSc and 38% in the ljSSc group (p=0.271). Tendon friction rub was present in 11% in djSSc and 4% in the ljSSc group. djSSc patients had significantly worse scores for physician global disease activity (VAS 0-100) (41vs 30) (p=0.020) and for physician global disease damage (VAS 0-100) (37 vs 18) (p=0.001). Patient judgment of disease activity and damage was similar in both subtypes. ANA positivity was 88% in both groups. Anti-Scl70 was positive in 33% in djSSc and 37% in the ljSSc group. Anticentromere positivity occurred in 3% in the djSSc and 10% in the ljSSc group. ESR was elevated in 30% in djSSc compared to 18% in the ljSSc group. DMARDs were used in 86% of the patients. <h3>Conclusion</h3> In this large cohort of jSSc patients there were surprisingly not many significant differences between djSSc and ljSSc. According to the physician global scores the djSSc patients have a significantly more severe disease. <h3>Disclosure of Interests</h3> Ivan Foeldvari Consultant for: Chugai, Novartis, Jens Klotsche: None declared, Ozgur Kasapcopur: None declared, Amra Adrovic: None declared, Kathryn Torok: None declared, Valda Stanevicha: None declared, Flávio R. Sztajnbok: None declared, Maria T. Tererri: None declared, Ekaterina Alexeeva: None declared, Jordi Anton Grant/research support from: Grant/research support, consultant or speakers bureau from AbbVie, Alexion, Bristol-Myers Squibb, ChemoCentryx, Gebro, GlaxoSmithKline, Novartis, Novimmune, Pfizer, Roche, Sanofi and Sobi, Consultant for: Grant/research support, consultant or speakers bureau from AbbVie, Alexion, Bristol-Myers Squibb, ChemoCentryx, Gebro, GlaxoSmithKline, Novartis, Novimmune, Pfizer, Roche, Sanofi and Sobi, Speakers bureau: Grant/research support, consultant or speakers bureau from AbbVie, Alexion, Bristol-Myers Squibb, ChemoCentryx, Gebro, GlaxoSmithKline, Novartis, Novimmune, Pfizer, Roche, Sanofi and Sobi, Maria Katsikas: None declared, Vanessa Smith: None declared, Tadej Avcin: None declared, Rolando Cimaz: None declared, Mikhail Kostik: None declared, Thomas Lehman: None declared, Walter Alberto Sifuentes-Giraldo: None declared, Simone Appenzeller: None declared, Mahesh Janarthanan: None declared, Monika Moll: None declared, Dana Nemcova: None declared, Maria Jose Santos: None declared, Dieneke Schonenberg: None declared, Cristina Battagliotti: None declared, Lillemor Berntson: None declared, Blanca Bica: None declared, Juergen Brunner: None declared, Patricia Costa Reis: None declared, Despina Eleftheriou: None declared, Liora Harel: None declared, Gerd Horneff: None declared, Tilmann Kallinich Grant/research support from: Novartis, Speakers bureau: Sobi, Roche, Novartis, CLB, Dragana Lazarevic: None declared, Kirsten Minden Consultant for: AbbVie, Susan Nielsen: None declared, Farzana Nuruzzaman: None declared, Anjali Patwardhan: None declared, Yosef Uziel: None declared, Nicola Helmus: None declared