SAT0478 AFTER 24 MONTHS OBSERVATION PERIOD THE PATIENTS RELATED OUTCOMES IMPROVE SIGNIFICANTLY IN THE JUVENILE SCLERODERMA INCEPTIONS COHORT. WWW.JUVENILE-SCLERODERMA.COM
Authors: Ivan Foeldvari, Jens Klotsche, Ozgur Kasapcopur, Amra Adrovic, Maria T. Tererri, Jordi Anton, Tadej Avcin, Rolando Cimaz, Mikhail Kostik, Maria Katsikas, Dana Nemcova, Maria Jose Santos, Cristina Battagliotti, Lillemor Berntson, Juergen Brunner, Liora Harel, Tilmann Kallinich, Kirsten Minden, Monika Moll, Anjali Patwardhan, Kathryn Torok, Nicola Helmus
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background</h3> Juvenile systemic sclerosis (jSSc) is an orphan disease with a prevalence of 3 in a 1 000 000 children(1). 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. Objectives: to evaluate the organ involvement and patient reported outcomes (PRO) during the first 24 months in the JSIC <h3>Methods</h3> The JSSIC is a prospective multicenter registry of patients with jSSc, who fulfill the adult classification criteria(2), 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. <h3>Results</h3> 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 &lt;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). <h3>Conclusion</h3> 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. <h3>References</h3> [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] van den Hoogen F, Khanna D, Fransen J, Johnson SR, Baron M, Tyndall A, et al. 2013classification criteria for systemic sclerosis: an American College of Rheumatology/European League against Rheumatism collaborative initiative. Arthritis Rheum. 2013;65(11):2737-47. <h3>Disclosure of Interests</h3> Ivan Foeldvari Consultant for: Chugai, Novartis, Jens Klotsche: None declared, Ozgur Kasapcopur: None declared, Amra Adrovic: None declared, Maria T. Tererri: 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, Tadej Avcin: None declared, Rolando Cimaz: None declared, Mikhail Kostik: None declared, Maria Katsikas: None declared, Dana Nemcova: None declared, Maria Jose Santos: None declared, Cristina Battagliotti: None declared, Lillemor Berntson: None declared, Juergen Brunner: None declared, Liora Harel: None declared, Tilmann Kallinich Grant/research support from: Novartis, Speakers bureau: Sobi, Roche, Novartis, CLB, Kirsten Minden Consultant for: AbbVie, Monika Moll: None declared, Anjali Patwardhan: None declared, Kathryn Torok: None declared, Nicola Helmus: None declared