Background Juvenile systemic sclerosis (jSSc) is an orphan autoimmune disease. Currently just retrospective data exist regarding evolvement of organ involvement. In the retrospective studies assessment of the organ involvement is not standardized. Our project is the first one, where prospectively and with a standardized assessment data of jSSc patients are collected. Objectives to learn about the characteristics and evolvement of jSSc Methods Patients with jSSc were recruited worldwide and were prospectively assessed, using the proposed standardized patient assessment protocol. Results 26 centers from 17 countries applied to participate on the project. The assent and consent forms were translated into the local native languages. Up till now 74 patients were enrolled. Sixty (81%) of the 74 patients were female. The mean age of the onset of Raynaud symptomatic was 9.2 years (0.2 – 15.9). The mean age at the onset of the non-Raynaud symptomatic were 9,7 years (0.3 -15.9). 56 (76%) of the 74 have diffuse subtype, 10 (14%) of them have an overlap symptomatic. At the time of the inclusion the mean modified Rodnan Skin Score was 16.0. ANA positive were 55/71 (77%), 24/70 (34%) of them were anti-Scl 70 positive and 3/42 (7%) was anticentromere positive. 43/74 (58%) had already capillary changes and 36/72 (50%) inactive ulcerations, 13/72 (18%) had active ulceration at the time of the inclusion. 38/74 (51%) had cardiopulmonary involvement, 19/38 (50%) of had signs of interstitial lung disease on imaging, 18/42 (43%) had FVC <80% and 12/21 (57%) had DLCO <80%. 6/38 (16%) patients had pulmonary hypertension. 5/74 (7%) had renal involvement. 26/74 (35%) had gastrointestinal involvement, and 23/26 (88%) of them esophageal involvement. 46/73 (63%) had musculoskeletal involvement. 2/74 (3%) showed neurologic involvement. The mean CHAQ score was 0.4 (0–2.5). Patient global disease activity on VAS (0–100) was 44.9 and disease damage 41.6. Physician global of disease activity on VAS (0–100) was 39.7 and physician global of disease damage was 34.6. Conclusions The current recruitment data confirms that pediatric patients are different from the adult patients, there is a significantly higher proportion of diffuse subset patients with 81%. 14% of the patients have overlap features. Disclosure of Interest None declared

Background Juvenile systemic sclerosis (jSSc) is an orphan autoimmune disease. Several adult publications looked at the differences between male and female patients with Systemic Sclerosis. There is rarity of data regarding this topic in pediatric jSSc. The juvenile scleroderma inception cohort (www.juvenilescleroderma.com) is a prospective standardized register for patients with jSSc. Objectives comparison of patients characteristic at the time of inclusion in the registry who are male or female. Methods Patients with jSSc were included worldwide to the juvenile scleroderma inception cohort. We compered the demographics and clinical characteristics of the male and female patients. Results Up till now 74 patients were enrolled, 54 with djSSc (76%) and 18 with ljSSc (24%). 14 (19%) of the patients were male (M) and 60 female (F) (81%). The mean disease duration at the time of inclusion was 9.3 in M and 9.2 in F patients. 74.4% of the M and 76.7% of the F had diffuse subset. The mean age of the onset of Raynaud symptomatic was 9.3 in M and 9.2 years in the F patients and the non-Raynaud symptomatic with 9.1 in M and 9.9 in F patients. At the time of the inclusion the mean modified Rodnan Skin Score was 20 in M and 15.1 in F patients. Anti-Scl 70 positivity was found in 42.9% in M and 32.1% in F patients. Anticentromere positivity occurred in 16.7% in M and 3.3% in F patients (p=0.027). Capillary changes were present in 50% of the M and 60% of F patients, but 50% in M and F had already history of ulcerations, but 28.6% in M and 15.5% in F had active ulceration. 57.1% of the M and 50% of the F patients had cardiopulmonary involvement. Six patients had pulmonary hypertension, they were all F. 75% of M and 46.7% of F patients had signs of interstitial lung disease on imaging. Renal involvement was around 7% in both sexes. 21.4% in M and 38.3% in F patients had gastrointestinal involvement. 92.9% of M and 55.9% in M patients had musculoskeletal involvement. Conclusions We present the data on the first 74 patients with jSSc included in our cohort. Patients with male sex have a more severe disease similar to adult male patients. Disclosure of Interest None declared

Background Juvenile systemic sclerosis (jSSc) is an orphan autoimmune disease. Several adult publications looked at the differences between limited (ljSSc) and diffuse subtype (djSSc). There is rarity of data regarding this topic in pediatric jSSc. The juvenile scleroderma inception cohort is a prospective standardized register for patients with jSSc. Objectives comparison of patients characteristic at the time of inclusion with ljSSc and djSSc Methods We compered the demographics and clinical characteristics of the ljSSc and djSSc. Results Up till now 74 patients were enrolled, 54 (76%) with djSSc and 18 with ljSSc (24%). 9% in djSSc and 25% in ljSSc showed overlap features. Disease duration at time of inclusion in the cohort was 3.7 years in the djSSc and 3.3 years in ljSSc. 82% in the djSSc and 78% in the ljSSc group were female. The mean age of the onset of Raynaud symptomatic was 9.0 years in the jdSSc and 9.9 years in ljSSc group and onset of the non-Raynaud symptomatic with 9.4 in djSSc and 10.6 ljSSc. At the time of inclusion the mean modified Rodnan Skin Score was 18.5 in the djSSc and 8,4 in ljSSc (p=0.0001). Anti-Scl 70 positivity was found in 31.5% of djSSc and 30.8% in ljSSc. Only 2 patient in the djSSc group and one in the ljSSc group was anticentromere positive. Capillary changes occurred in 60.7% in the djSSc and 50% in ljSSc, but 58.2% in djSSc and only 23.5% in ljSSc had already history of ulcerations (p=0.013) and 21.8% had active ulceration in the djSSc and 5.9% in the ljSSc. 33.3% of djSSc and 72.7% of ljSSc had cardiac involvement (p=0.027). pulmonary hypertension occurred in 57.1% djSSc and 18.2% in ljSSc. 63% in djSSc and 27.3% in ljSSc group had signs of interstitial lung disease on imaging (p=0.046). Renal involvement occurred in 7.1% djSSc and 5.6% in ljSSc. 39.3% of djSSc and 22.2% of ljSSc had gastrointestinal involvement. 56.4% in djSSc and 83.35 in ljSSc had musculoskeletal involvement (p=0.04). Conclusions Patients with djSSc have younger age at onset, have more often capillary changes and active ulcerations, pulmonary hypertension and less gastorintestinal and joint involvement and more disease damage. The characteristics of the pediatric subtypes differs from adults with SSc, especially the high proportion of patients with diffuse subtype. Disclosure of Interest None declared

Objective. The main objective was to determine the 2-year clinical benefit and safety of etanercept (ETN) in children with the juvenile idiopathic arthritis (JIA) categories of extended oligoarthritis (eoJIA), enthesitis-related arthritis (ERA), or psoriatic arthritis (PsA). Methods. CLIPPER was a 96-week, phase IIIb, open-label, multicenter study. Patients with eoJIA, ERA, or PsA received ETN 0.8 mg/kg once weekly (50 mg max) for up to 96 weeks. The proportions of patients reaching the JIA American College of Rheumatology (ACR) 30/50/70/90/100 and inactive disease responses at Week 96 were calculated. Adverse events (AE) were collected throughout the study (intention-to-treat sample). Results. There were 127 patients (eoJIA n = 60, ERA n = 38, PsA n = 29) who received ≥ 1 dose of ETN. The mean disease duration was 31.6 (eoJIA), 23.0 (ERA), and 21.8 (PsA) months. At Week 96, JIA ACR 30/50/70/90/100/inactive disease responses (95% CI) were achieved by 84.3% (76.7, 90.1), 83.5% (75.8, 89.5), 78.7% (70.6, 85.5), 55.1% (46.0, 63.9), 45.7% (36.8, 54.7), and 27.6% (20.0, 36.2) of patients, respectively. The most common AE (no. events, events per 100 patient-yrs) overall were headache (23, 10.7), pyrexia (12, 5.6), and diarrhea (10, 4.6). The most common infections were upper respiratory tract infection (83, 38.6), pharyngitis (50, 23.2), gastroenteritis (22, 10.2), bronchitis (19, 8.8), and rhinitis (17, 7.9). No cases of malignancy, active tuberculosis, demyelinating disorders, or death were reported. Conclusion. Over 96 weeks of therapy, ETN demonstrated sustained efficacy at treating the clinical symptoms of all 3 JIA categories, with no major safety issues.

Neonatal onset multisystem inflammatory disease is a rare autoinflammatory disorder. Clinical features include fever episodes, urticarial rash, arthralgia and arthritis, eyes and central nervous system involvement. It belongs to the group of cryopyrin-associated periodic syndromes which  result from a gain-of-function mutations of the NLRP3 gene on chromosome 1p44 that encodes the cryopyrin protein. Defects lead to overproduction of inflammatory cytokines involved in the innate immune system, especially interleukin 1. This article reports a clinical case of a 6-year-old boy, who presented with  first clinical signs of the disease soon after birth. We present a diagnostic approach in a case of suspected periodic fever syndrome. It is based on exclusion of infections, primary immunodeficiencies, autoimmune and malignant disorders. For confirmation of the disease, genetic analysis is mandatory. The patient was successfuly treated with biological medications which block interleukin 1.