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Made available in DSpace on 2021-06-25T10:38:01Z (GMT). No. of bitstreams: 0 Previous issue date: 2020-11-01 Healthway Objective. To develop a composite disease activity score for systemic JIA (sJIA) and to provide preliminary evidence of its validity. Methods. The systemic Juvenile Arthritis Disease Activity Score (sJADAS) was constructed by adding to the four items of the original JADAS a fifth item that aimed to quantify the activity of systemic features. Validation analyses were conducted on patients with definite or probable/possible sJIA enrolled at first visit or at the time of a flare, who had active systemic manifestations, which should include fever. Patients were reassessed 2 weeks to 3 months after baseline. Three versions were examined, including ESR, CRP or no acute-phase reactant. Results. A total of 163 patients were included at 30 centres in 10 countries. The sJADAS was found to be feasible and to possess face and content validity, good construct validity, satisfactory internal consistency (Cronbach's alpha 0.64-0.65), fair ability to discriminate between patients with different disease activity states and between those whose parents were satisfied or not satisfied with illness outcome (P < 0.0001 for both), and strong responsiveness to change over time (standardized response mean 2.04-2.58). Overall, these properties were found to be better than those of the original JADAS and of DAS for RA and of Puchot score for adult-onset Still's disease. Conclusion. The sJADAS showed good measurement properties and is therefore a valid instrument for the assessment of disease activity in children with sJIA. The performance of the new tool should be further examined in other patient cohorts that are evaluated prospectively. UOC Clinica Pediatrica e Reumatologia IRCCS Istituto Giannina Gaslini Dipartimento di Neuroscienze Riabilitazione Oftalmologia Genetica e Scienze Materno-Infantili (DiNOGMI) Università degli Studi di Genova Dipartimento di Epidemiologia e Biostatistica IRCCS Istituto Giannina Gaslini Faculty of Medicine Ain Shams University Pediatric Rheumatology Division Institute of Child Health Section of Pediatric Rheumatology SRCC Children's Hospital Rheumatology Division Pediatric Department Faculty of Medicine Ramathibodi Hospital Mahidol University Pediatric Rheumatology Clinic R G Kar Medical College Division of Rheumatology IRCCS Ospedale Pediatrico Bambino Gesù Division of Pediatric Rheumatology Institute of Child Health Sir Ganga Ram Hospital Faculty of Medicine Benha University Faculty of Medicine Alexandria University Faculty of Medicine Tanta University Faculty of Medicine Cairo University Pediatric Rheumatology Division Adolescent Health Care Unit Universidade do Estado do Rio de Janeiro Department of Internal Medicine Universidade Federal do Rio de Janeiro Servicio de Inmunología y Reumatología Hospital de Pediatría Garrahan Department of Clinical Sciences and Community Health University of Milan Division of Rheumatology University Hospital Meyer Rheumatology Division National Medical Research Center of Children's Health Sechenov First Moscow State Medical University Department of Immunology Rheumatology and Allergy Queen Rania Children's Hospital Saint-Petersburg State Pediatric Medical University UOC Pediatria a Media Intensità di Cure Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Rheumatology Department Faculty of Medicine Suez Canal University Department of Pediatrics King Faisal Specialist Hospital and Research Center Dipartimento Promise G. D'Alessandro Università degli Studi di Palermo Pediatric Department Hospital das Clínicas Botucatu Medicine University UNESP UOC Pediatria Servizio di Immuno-Reumatologia Pediatrica Azienda Ospedaliera Universitaria Gaetano Martino Messina UO Nefrologia e Reumatologia Pediatrica Azienda Ospedaliera Universitaria Gaetano Martino Department of Pediatrics School of Medicine Institute of Medical Pharmaceutical and Health Sciences Kanazawa University Pediatric Unit Ospedale Vito Fazzi Pediatric Unit AON SS Antonio e Biagio e Cesare Arrigo Children's Hospital Pediatric Department Hospital das Clínicas Botucatu Medicine University UNESP

Systemic juvenile idiopathic arthritis (SJIA) is a disease of unknown etiology characterized by arthritis and systemic symptoms. Evidence shows that SJIA is probably not a single disease but a diverse group of clinically and genetically distinct illnesses. It is classified both as one of the Juvenile idiopathic arthritis (JIA) categories and also among the autoinflammatory diseases.

OBJECTIVE: To develop a composite DAS for JDM and provide preliminary evidence of its validity. METHODS: The Juvenile DermatoMyositis Activity Index (JDMAI) is composed of four items: physician's global assessment of overall disease activity ; parent's/child's global assessment of child's wellbeing ; measurement of muscle strength ; and assessment of skin disease activity. The score of the JDMAI is the arithmetic sum of the scores of each individual component. Six versions of the JDMAI were tested, which differed in the tools used to assess the third and fourth items. Validation procedures were conducted using three large multinational patient samples including a total of 627 patients. RESULTS: The JDMAI was found to possess face and content validity, good construct validity, satisfactory internal consistency (Cronbach's alpha = 0.58- 0.89), fair responsiveness to clinically important change (standardized response mean = 0.82-3.12 among patients improved) and strong capacity to discriminate patients judged as being in the state of inactive disease or low, moderate or high disease activity by the physician (P < 0.001) or whose parents were satisfied or not satisfied with the course of their child's illness (P < 0.001). Overall, the six versions of the JDMAI showed similar metrological performances in validation analyses. CONCLUSION: The JDMAI was found to possess good measurement properties in a large population of patients with a wide range of disease activity, and is, therefore, suitable for use in both clinical and research settings. The final version of the JDMAI will be selected after its prospective validation

Systemic-onset juvenile idiopathic arthritis is a complex, polygenic autoinflammatory disease affecting young individuals. It is an innate immunity-driven condition characterized by chronic, multiorganic inflammatory involvement. Typical clinical features including young age at presentation, fever, neutrophilic skin lesions, synovitis, serositis, organomegaly, as well as increased risk of amyloidosis are reminiscent of (and may mimic) certain monogenic autoinflammatory disorders. Strikingly high acute phase reactants, thrombocytosis, hyperferritinemia, and elevated serum levels of inflammatory-related proteins such as IL-1, IL-18, S100A8/9, and S100A12 characterize active phases. The disease may follow a relapsing or a continuous course into adulthood, frequently leading to joint damage, disability, and poorer quality of life. Major morbidity and mortality arise from the hypercytokinemic, hyperinflammatory stages called macrophage activation syndrome, a form of reactive hemophagocytic lymphohistiocytosis, which may occur in 50% of patients but become clinically overt only in a smaller proportion. Multidisciplinary teams are best fit to treat this complex disease. Decades after nonbiologic drugs and TNF inhibitors had consistently failed, the advent of biologic agents blocking IL-1 and IL-6 has proven dramatically efficacious for medical management.

Made available in DSpace on 2019-10-05T16:54:20Z (GMT). No. of bitstreams: 0 Previous issue date: 2019-04-01 IRCCS Istituto Giannina Gaslini Background To our knowledge, the characteristics and burden of childhood arthritis have never been studied on a worldwide basis. We aimed to investigate, with a cross-sectional study, the prevalence of disease categories, treatment methods, and disease status in patients from across different geographical areas and from countries with diverse wealth status. Methods In this multinational, cross-sectional, observational cohort study, we asked international paediatric rheumatologists from specialised centres to enrol children with a diagnosis of juvenile idiopathic arthritis, according to International League of Associations for Rheumatology criteria, who were seen consecutively for a period of 6 months. Each patient underwent retrospective and cross-sectional assessments, including measures of disease activity and damage and questionnaires on the wellbeing and quality of life of the children. We qualitatively compared the collected data across eight geographical areas, and we explored an association between disease activity and damage and a country's gross domestic product (GDP) with a multiple logistic regression analysis. Findings Between April 4, 2011, and Nov 21, 2016, 9081 patients were enrolled at 130 centres in 49 countries, grouped into eight geographical areas. Systemic arthritis (125 [33.0%] of 379 patients) and enthesitis-related arthritis (113 [29.8%] of 379) were more common in southeast Asia, whereas oligoarthritis was more prevalent in southern Europe (1360 [56.7%] of 2400) and rheumatoid factor-negative polyarthritis was more frequent in North America (165 [31.5%] of 523) than in the other areas. Prevalence of uveitis was highest in northern Europe (161 [19.1%] of 845 patients) and southern Europe (450 [18.8%] of 2400) and lowest in Latin America (54 [6.4%] of 849), Africa and Middle East (71 [5.9%] of 1209), and southeast Asia (19 [5.0%] of 379). Median age at disease onset was lower in southern Europe (3.5 years, IQR 1.9-7.3) than in other regions. Biological, disease-modifying antirheumatic drugs were prescribed more frequently in northern Europe and North America than in other geographical settings. Patients living in countries with lower GDP had greater disease activity and damage than those living in wealthier countries. Damage was associated with referral delay. Interpretation Our study documents a variability in prevalence of disease phenotypes and disparities in therapeutic choices and outcomes across geographical areas and wealth status of countries. The greater disease burden in lowerresource settings highlights the need for public health efforts aimed at improving equity in access to effective treatments and care for juvenile idiopathic arthritis. Copyright (C) 2019 Elsevier Ltd. All rights reserved. Univ Genoa, Ist Giannina Gaslini, IRCCS, Clin Paediat & Rheumatol, Genoa, Italy Univ Genoa, Ist Giannina Gaslini, IRCCS, Epidemiol & Biostat Serv, Genoa, Italy Univ Genoa, Ist Giannina Gaslini, IRCCS, Sci Directory, Genoa, Italy Univ Genoa, PRINTO, IRCCS, Ist Giannina Gaslini, Genoa, Italy Univ Genoa, Dept Neurosci Rehabil Ophthalmol Genet & Maternal, Genoa, Italy Wilhelmina Childrens Hosp, Dept Pediat Immunol & Rheumatol, Utrecht, Netherlands Sanjay Gandhi Postgrad Inst Med Sci, Lucknow, Uttar Pradesh, India Univ Hosp 12 Octubre, Dept Pediat Rheumatol, Madrid, Spain Alfaisal Univ, Dept Pediat Rheumatol, King Faisal Specialist Hosp, Riyadh, Saudi Arabia Alfaisal Univ, Res Ctr, Riyadh, Saudi Arabia Western Univ Childrens Hosp, Hlth Sci Ctr, London, ON, Canada Oslo Univ Hosp, Dept Rheumatol, Oslo, Norway Oslo Univ Hosp, Med Fac, Oslo, Norway Univ Oslo, Oslo, Norway Oslo Univ Hosp, Norwegian Natl Advisory Unit Rheumat Dis Children, Oslo, Norway Univ Hosp Munster, Dept Pediat Rheumatol & Immunol, Munster, Germany Hosp Sor Maria Ludovica, Rheumatol Serv, La Plata, Buenos Aires, Argentina Bucharest Emergency Hosp, Cluj Napoca, Romania Childrens Emergency Hosp, Cluj Napoca, Romania Cincinnati Childrens Hosp Med Ctr, Div Rheumatol, Cincinnati, OH 45229 USA Alberta Childrens Prov Gen Hosp, Div Pediat Rheumatol, Dept Pediat, Calgary, AB, Canada Sofiamed, Pediat Dept, Sofia, Bulgaria Rigshosp, Juliane Marie Ctr, Paediat Rheumatol Unit, Copenhagen, Denmark Natl Inst Rheumatism & Physiotherapy, Clin Immunol Adult & Paediat Rheumatol Dept, Budapest, Hungary Univ Latvia, Pediat Dept, Riga, Latvia Univ Latvia, Univ Childrens Hosp, Riga, Latvia Univ Estadual Paulista, Botucatu Fac Med, Botucatu, SP, Brazil Shariati Hosp, Rheumatol Res Ctr, Dept Pediat & Rheumatol, Tehran, Iran Inst Rheumatol Belgrade, Div Pediat Rheumatol, Belgrade, Serbia Thessaloniki Univ, Dept Pediat 1, Hippokrat Gen Hosp, Sch Med, Thessaloniki, Greece Univ Estadual Paulista, Botucatu Fac Med, Botucatu, SP, Brazil

Objective To develop and test a hybrid measure of muscle strength for juvenile dermatomyositis (JDM), which is based on the combination of the Manual Muscle Testing 8 (MMT8) and the Childhood Myositis Assessment Scale (CMAS), but is more comprehensive than the former and more feasible than the latter. Methods The hybrid MMT/CMAS (hMC) is composed of all 8 items of the MMT8 and 3 items of the CMAS: 1) time of head lift; 2) assessment of abdominal muscles; 3) floor rise. The score ranges from 0 to 100, with 100 indicating normal muscle strength. Validation procedures were conducted using three large multinational patient samples including a total of 810 JDM patients. Results The hMC revealed face and content validity, good construct validity, excellent test-retest reliability (intraclass correlation coefficient = 0.99) and internal consistency (Chronbach's alpha = 0.94), strong responsiveness to clinical change over time (standardized response mean = 0.8 among patients judged as improved by the caring physician), and satisfactory capacity to discriminate patients judged as being in the states of inactive disease or low, moderate, or high disease activity by the physician (p < 0.001) or patients whose parents were satisfied or not satisfied with illness course (p < 0.001). Conclusion The hMC was found to possess good measurement properties in a large population of patients with a wide range of disease activity and severity. The new tool, which is primarily intended for use in routine clinical care, should be further tested in other populations of patients evaluated prospectively. This article is protected by copyright. All rights reserved.

Background Juvenile systemic sclerosis (jSSc) is an orphan autoimmune disease. It was rarely looked at the differences between the clinical presentations of patients at different paediatric age groups. The juvenile scleroderma inception cohort (www.juvenile-scleroderma.com)is a prospective standardized register for patients with jSSc. Objectives Comparison of clinical characteristics of patients with different age range at the time of inclusion in the registry. Methods Patients with jSSc were included worldwide to the juvenile scleroderma inception cohort. We compared the demographics and clinical characteristics of the patients at different age ranges. We created 3 cohorts with different age ranges at onset of disease. Patents aged less than 5 years (Group1), 5–10 years (Group2) and over 10 years (Group3) at the time of diagnosis of the first non-Raynaud involvement of jSSc. Results Up till now 88 patients were enrolled,14 patients (15%) in Group1, 22 (25%) in Group2 and 52 (59%) in Group3. Diffuse subtype occurred in 71% in Group1, in 82% in Group2 and in 65% in Group3. Most patients were Caucasian. Disease duration at time of inclusion into the cohort was 3.9 years in Group1, 4.9 years in Group2 and 2.2 years in Group3. ANA positivity was 57% in Group1, 77% in Group2 and 86% in Group3. Anti-scl 70 was around 30% in all groups. Anti-Centromere positivity was 7 to 10%. Mean modified skin score was 12.4 in Group1, 16.5 in Group2 and 15.9 in Group3. Raynaud Phenomenon occurred in 85 to 95% of the patients. History of active or inactive ulceration occurred in 57% in Group1, 62% in Group2 and 43% in Group3. Decreased FVC under 80% occurred in 43% in Group1, 32% in Group2 and 30% in Group3. Pulmonary hypertension occurred in 7% in Group1 and in 10% in Group3. No renal hypertension was observed. Urinary sedimentary changes occurred in 7% in Group1 and in 10% in Group3. Gastrointestinal involvement occurred in 21% in Group1, 45% in Group2 and 27% in Group3. Musculoskeletal involvement occurred in 58 to 64%. Patient global disease activity (VAS 0–100) was 42.8 to 47.9. Patient global disease damage (VAS 0–100) was 39.6- to 45.0. Physician global disease activity (VAS 0–100) was 35.4–40.0. Physician global disease damage (VAS 0–100) was 37.1 in Group1, 41.3 in Group2 and 27.7 in Group3. Conclusions It seems to be that patients, with onset of the disease in younger age have more severe disease as patients with disease onset after the age of 10 years. We need more patients in our cohort to gain more sufficient data to prove our preliminary observation. Disclosure of Interest None declared

Takayasu's arteritis is a chronic inflammatory disease that primarily affects the large vessels, such as the aorta and its branches. It represents the third most frequent vasculitis during pediatric age. Our objective was to describe clinical and complementary exams features as well as treatment modalities of a case series of pediatric patients. We present 11 patients (10 girls) with median age at onset of 8 years (range: 2-15). The median diagnosis delay was 16 months (range: 2-96). Clinical presentations were lower limb claudication, arterial hypertension, CNS involvement, presence of murmurs, systemic symptoms, lymphadenopathy, chest pain, abdominal pain and arthritis. Laboratory tests showed: elevated ESR, anemia and trombocytosis. Vascular imaging studies exhibited stenosis, dilatation, occlussion and aneurysms. The outcome of the disease was persistent active condition (1 patient), relapse (4 patients), remission (3 patients), motor sequelae (1 patient) and death (2 patients). All patients were treated with steroids and immunosuppressants. Takayasu 's arteritis is a condition that can potentially be life-threatening. The diagnosis should be suspected in a variety of clinical manifestations during childhood.

UNLABELLED CINCA/IOMID is a systemic inflammatory disorder of unknown aetiology that resembles congenital infection and systemic juvenile chronic arthritis (JCA). This disorder is characterized by neonatal onset, persistent rash, ocular inflammatory lesions, and progressive articular and neurological involvement. We report two new patients with this syndrome. Both children presented periodic bouts of cutaneous rash, fever, organomegaly, articular involvement with typical radiological features, and developmental delay. One of the patients presented neonatal jaundice and elevation of liver enzymes; inflammatory infiltrates were observed in the liver biopsy. The other patient showed retinal vasculitis detected at age 18 mo on fundoscopy and fluorescent angiography. Therapy with azathioprine was associated with prolonged remission of this complication. In both cases, the disease was diagnosed after some delay. CONCLUSION Early hepatitis and retinal vasculitis are rare features of CINCA/IOMID that may help differentiate this syndrome from JRA. Azathioprine may have induced the remission of vasculitis in one case.

Aim: To describe two children with hypertrophic osteoarthropathy associated with cholestatic hepatic disease. Both patients suffered from chronic progressive cholestatic liver disease and developed digital clubbing, polyarthritis and periosteal reaction. In one of them, clinical and radiological features normalized after liver transplant. Conclusion: Hepatic hypertrophic osteoarthropathy is a rare disabling condition that responds poorly to conservative management, while liver transplantation appears to be the only effective therapeutic intervention.

Educational strategies and contents developed in the First World are not always adapted to the needs and challenges of developing countries. Cooperation and collaboration between developing countries can be mutually beneficial and create the basis for a longstanding, Third World-centered educational program aimed at increasing the pediatric support for Pediatric Rheumatology (PR).

Background Juvenile Spondyloarthropathy (jSpA) is SpA that starts during childhood and adolescence. Most children with jSpA fall into the categories of enthesitis-related arthritis (ERA), psoriatic arthritis, and undifferentiated arthritis of the ILAR classification of Juvenile idiopathic arthritis (JIA). Several disease activity scores have been developed for adults with SpA and for children with JIA (such as the Juvenile Arthritis Disease Activity Score or JADAS). However, neither of these measures has been validated in jSpA. The Juvenile Spondyloarthritis Disease Activity Index (JSpADA) has been recently developed and retrospectively validated in jSPA (1). Objectives to assess disease activity in a cohort of patients with ERA on anti-TNF therapy using the JADAS-10 and the JSpADA. Methods retrospective review of prospectively collected data. Patients with ERA treated with anti-TNF agents for ≥1 year and complete records were included. Information was collected on visits 0 (immediately prior to anti-TNF therapy start), 1 month after start of therapy, and every 3 months thereafter (up to 36 months) on demographics, therapeutic agents used, and disease activity measures: active joints (AJ), active enthesitis (AE), pain score, wellbeing according to the patient (VASp), disease activity according to the physician (VASphy), JADAS-10, JSpADA, and ESR. Comparisons between visits were performed using paired t-test; correlations between outcome measures was done using Spearman9s correlation test. Results 24 of 28 patients treated with anti-TNF agents fulfilled inclusion criteria. They were all male, 50% HLA-B27 positive; median age at entry 13 y, disease duration 4 y; median observation period was 21 months, 207 visits were recorded. Twenty-two (92%) children showed peripheral arthritis while 17 (71%) exhibited sacroiliitis on MRI/Xrays before starting therapy. Patients received etanercept (17, 2 were later switched to infliximab and adalimumab each), adalimumab (5), or infliximab (2). At baseline patients showed (medians): AJ 4.5, ESR 15 mm/h, VASphy 1, JSpADA 2, and JADAS 9.5. AE was present in 4 (17%) and lumbar limitation (LL) in 9 (37%) children. All patients showed JSpADA >0 and 95% children had JADAS >1. After 3 months JSpADA decreased to 1.25 and JADAS-10 to 6; 5 (21%) patients achieved inactive disease (ID, JADAS-10 ≤1). At visit 12 months: AJ 0 (p=0.0015), ESR 7 mm/h (p=0.06), VASphy 0.5 (p=0.005), JSpADA 1 (p=0.0004), and JADAS 1.75 (p=0.02); AE was present in 0 patients and LL in 6 (25%). JADAS-10 ≤1 was observed in 18 (75%) patients and JSpADA ≤1 in 21 (87%) children during follow-up. At baseline and at visit 12 months JSpADA showed high correlations with JADAS-10 (r=0.97, 0.75) and AJ (r=0.73, 0.77); JADAS-10 also correlated with AJ (r=0.92, 0.83). Conclusions in this retrospective cohort of ERA patients with peripheral and axial active involvement there was a marked and progressive reduction in disease activity after the initiation of anti-TNF therapy. Both JSpADA and JADAS-10 demonstrated to be sensitive, feasible and useful activity measures that could be used in clinical trials or a treat to target strategy for JSpA. References Weiss et al. Development and Retrospective Validation of the Juvenile Spondyloarthritis Disease Activity Index. Arthritis Care Res 2014;66:1775–1782. Disclosure of Interest None declared

Methods Retrospective analysis of clinical data. Early-onset (EO) was defined as the start of SJIA prior to age 18 months. Variables included: demographic and clinical features at onset and outcome variables during disease course (pattern of course, presence of clinical joint damage [using the Juvenile Arthritis Damage Index or JADI], radiographic joint damage [erosions], destructive hip disease, disability [CHAQ > 0.5], growth retardation, development of macrophage activation syndrome [MAS], need for biologic agents, and death). Patients with EO were compared with patients with disease onset at age ≥ 18 months. Chi square and Mann-Whitney tests were used for comparisons.

Background Tocilizumab (TCZ), an IL-6 receptor inhibitor, improves arthritis and systemic symptoms associated with systemic JIA. It may be a valuable option in patients with JIA who show inadequate response to anti-TNF agents. Aim

Made available in DSpace on 2013-08-12T19:10:16Z (GMT). No. of bitstreams: 0 Previous issue date: 2010-01-15 Made available in DSpace on 2013-09-30T19:20:41Z (GMT). No. of bitstreams: 0 Previous issue date: 2010-01-15 Submitted by Vitor Silverio Rodrigues (vitorsrodrigues@reitoria.unesp.br) on 2014-05-20T15:33:16Z No. of bitstreams: 0 Made available in DSpace on 2014-05-20T15:33:16Z (GMT). No. of bitstreams: 0 Previous issue date: 2010-01-15 Myositis Association European Union Objective. To investigate the long-term outcome and prognostic factors of juvenile dermatomyositis (DM) through a multinational, multicenter study.Methods. Patients consisted of inception cohorts seen between 1980 and 2004 in 27 centers in Europe and Latin America. Predictor variables were sex, continent, ethnicity, onset year, onset age, onset type, onset manifestations, course type, disease duration, and active disease duration. Outcomes were muscle strength/endurance, continued disease activity, cumulative damage, muscle damage, cutaneous damage, calcinosis, lipodystrophy, physical function, and health-related quality of life (HRQOL).Results. A total of 490 patients with a mean disease duration of 7.7 years were included. At the cross-sectional visit, 41.2-52.8% of patients, depending on the instrument used, had reduced muscle strength/endurance, but less than 10% had severe impairment. Persistently active disease was recorded in 41.2-60.5% of the patients, depending on the activity measure used. Sixty-nine percent of the patients had cumulative damage. The frequency of calcinosis and lipodystrophy was 23.6% and 9.7%, respectively. A total of 40.7% of the patients had decreased functional ability, but only 6.5% had major impairment. Only a small fraction had decreased HRQOL. A chronic course, either polycyclic or continuous, consistently predicted a poorer outcome. Mortality rate was 3.1%.Conclusion. This study confirms the marked improvement in functional outcome of juvenile DM when compared with earlier literature. However, many patients had continued disease activity and cumulative damage at followup. A chronic course was the strongest predictor of poor prognosis. These findings highlight the need for treatment strategies that enable a better control of disease activity over time and the reduction of nonreversible damage. Ist Ricovero & Cura Carattere Sci G Gaslini, Genoa, Italy Univ Genoa, Genoa, Italy Great Ormond St Hosp Sick Children, London WC1N 3JH, England UCL, Inst Child Hlth, London, England Univ Fed Rio de Janeiro, Rio de Janeiro, Brazil Universidade do Estado do Rio de Janeiro (UERJ), BR-20550011 Rio de Janeiro, Brazil Hosp Gen Ninos Pedro de Elizalde, Buenos Aires, DF, Argentina Fdn IRCCS Policlin, Milan, Italy Hosp Pediat Juan P Garrahan, Buenos Aires, DF, Argentina Univ São Paulo, Fac Med Ribeirao Preto, Hosp Clin, Ribeirao Preto, Brazil Hosp Gen Mexico City, Mexico City, DF, Mexico Fdn Ist Ricovero & Cura Carattere Sci Policlin S, Pavia, Italy Ctr Med Natl La Raza, Mexico City, DF, Mexico Hosp Pequeno Principe, Curitiba, Parana, Brazil Clin Pediat 1, Padua, Italy Ctr Med Nacl Siglo XXI, Mexico City, DF, Mexico Osped Pediat Bambino Gesu, Rome, Italy Osped Villa Monna Tessa, Florence, Italy Univ Naples Federico 2, Naples, Italy Ist Ortoped Gaetano Pini, Milan, Italy Univ estadual Paulista, Botucatu, SP, Brazil II Clin Pediat, Cagliari, Italy Univ São Paulo, São Paulo, Brazil Ist Ricovero & Cura Carattere Sci Burlo Garofalo, Trieste, Italy Univ estadual Paulista, Botucatu, SP, Brazil EU: AML/B7-311/970666/II-0246-FI

Background Juvenile systemic sclerosis (jSSc) is an orphan autoimmune disease. Several publications in adults looked at the differences between limited and diffuse subtypes. 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 features of patients with limited jSSc (ljSSc) and diffuse jSSc (djSSc) subtypes at the time of inclusion in the registry Methods Patients with jSSc were included worldwide into the juvenile scleroderma inception cohort. We compared the demographics and clinical features of the ljSSc and djSSc. Results Up till now 39 patients were enrolled, 29 with djSSc and 10 with ljSSc. 5 in the diffuse (17%) and 3 in the limited subtype (30%) had an overlap feature. The mean follow up of the patients in the cohort was 6.4 years in the djSSc and 5.3 years in ljSSc. 76% in the djSSc and 80% in the ljSSc group were female. The mean age at the onset of Raynaud9s Phenomenon was 8.7 years in the jdSSc and 12.9 years in ljSSc group while the mean age at the onset of the first non-Raynaud presentation was 9.1years in djSSc and 13.8 years ljSSc. At the time of the inclusion the mean modified Rodnan Skin Score was 19.6 in the djSSc and 7.5 in ljSSc. 70% of patients in both groups had already capillary changes, but 67% in djSSc and only 33% in ljSSc had already history of ulcerations and 32.1% presented with active ulceration in the djSSc and none in the ljSSc. 72% of djSSc and 50% of ljSSc had cardiopulmonary involvement. The two patients with pulmonary hypertension had djSSc. 27.5% in djSSc and 30% in ljSSc group showed signs of interstitial lung disease on imaging. All 3 patients with renal involvement had djSSc. In both groups 30% had gastrointestinal involvement. Around 80% had musculoskeletal involvement in both subtypes. Anti-Scl 70 positivity was found in 40% of djSSc and 37.5% in ljSSc. Only 1 patient in the djSSc group had anticentromere antibody. Conclusions We present the data on the first 39 patients with jSSc included in our cohort. Patients with djSSc and ljSSc differ in several characteristics. Patients with djSSc were younger at onset, had more often capillary changes and active ulcerations, pulmonary hypertension and renal involvement. The characteristics of the pediatric subtypes differs from adults with SSc. Disclosure of Interest None declared

Background The Juvenile spondyloarthropaties (JSpA) are a group of related, clinically heterogeneous, seronegative rheumatic diseases. Usually disease onset occurs in late childhood (after 8 years) 1 or adolescence. However, younger patients have been reported, making its diagnosis difficult at an early age. There is sparse information about the clinical features of JSpA in patients with disease onset before 8 years. Objectives To describe and analyze disease features at onset and during the disease course in patients with JSpA starting before 8 years. To compare such characteristics with patients with disease onset after 8 years. Methods Retrospective analysis of clinical data obtained from patients with JSpA (defined as ERA, JPsA or UA according to ILAR) followed in a tertiary center. Patiens were divided into 2 groups: disease onset ≤8 yo and disease onset >8 yo. Recorded variables included: age at onset, sex, disease duration before first visit; clinical features at disease onset and during disease course: number of active joints, number of limited joints, articular pattern (symmetric-asymmetric, oigoarticular/polyarticular, small/large joints and upper/lower limbs), axial involvement (limitation of lumbar spine motion, low back pain), presence of enthesitis, tarsitis, dactylitis, uveitis, radiologic sacroiliitis, diarrhea, urethritis, psoriasis, inflammatory bowel disease, fever, positive HLAB-27 and family history, lowest hemoglobin level, highest erythrocyte sedimentation rate (ESR). Outcome measures (at last visit): activity (BASDAI) and functional capacity (BASFI and CHAQ), use of biologics and presence of bone damage. Chi square and Mann-Whitney tests were used for comparisons Results 110 patiens with JSpA followed for median 7 years were included (M: 96, 87%). Disease onset ≤8 y.o ocurred in 20 (18%) children. Age at onset was 6 (1-8) y in the early onset group and 11 (9-16)y in the later onset group. Diagnosis delay (8 vs 6 months) was similar. Patients with disease onset ≤8 y differed from patients with disease onset >8 y in: articular pattern (symmetric 45% vs 22%, p=0.04) and polyarticular (40% vs 14%, p=0.0097), dactylitis (25% vs 8%, p=0.024), uveitis (25% vs 3%, p=0.0048), positive HLA B-27 (60% vs 20%, p=0.0007), lowest hemoglobin (11 g/dl vs 12.5 g/dl, p=0.0004), ESR (55 mm/h vs 28.5 mm/h, p=0.047) at onset. During disease course patients showed differences in: sacroiilitis (50% vs 22%, p=0.0003), persistent dactylitis (25% vs 7%, p=0.0049), radiographic bone damage (25% vs 7%, p=0.03) and use of biologics (55% vs 21%, p=0.0036). No differences in activity and functional capacity outcomes were found between both groups. Conclusions JSpA patients with disease onset before 8 yo show more inflammatory features at disease onset. These findings seem to identify a distinct population. Recognizing these findings at onset would allow a correct classification of younger patients with JSpA. References Petty RE, Southwood TR, Baum J; Bhettay E, Glass DN, Manners P, Maldonado-Cocco J, Suarez-Almazor M, Orosco-Alcala J, Prier AM. Revision of the proposed classification criteria for juvenile idiopathic arthritis: Durban, 1997. J Rheumatol 1998 Oct;25(10):1991-4. Disclosure of Interest None declared

To evaluate the effectiveness of etanercept in patients with systemic juvenile idiopathic arthritis (SJIA) refractory to methotrexate (MTX) therapy in a pediatric rheumatology practice.Fifteen patients with SJIA with active polyarthritis refractory to higher dose MTX (or = 20 mg/m2/week) for at least 3 months were included. Patients received etanercept 0.4 mg/Kg twice weekly concomitantly with MTX. Observed period of treatment ranged from 5 to 12 months (median 9 months).Improvement of ESR, swollen and limited joint counts, functional capacity, and general wellbeing was achieved by 14/15 patients. The most significant impact on these variables was observed 3 to 5 months after treatment onset. Mean time to improvement was 2 months. In the 4 patients who presented fever and rash, these signs disappeared after the beginning of etanercept treatment and reappeared during flares. Three patients showed sustained clinical and biochemical remission on low dose MTX (or = 5 mg/m2/week). Thirteen relapses were observed in 9 (60%) patients at a mean of 7.6 months after therapy was begun. Etanercept was discontinued due to lack of efficacy in 7 patients, only after higher dose (1 mg/kg/dose) was used. MTX and corticosteroid doses were decreased during the observation period. No serious side effects were observed.Etanercept, in combination with MTX, demonstrated benefit soon after initiation of treatment in patients with refractory SJIA, but flares and progressive loss of effectiveness were observed with continued treatment in most patients. Sharp decreases in the dose of MTX and corticosteroids may have contributed to subsequent occurrence of flares. Changes in MTX and corticosteroids doses should probably need to be made gradually, and it is possible that patients on SJIA should continue on therapeutic doses of MTX while being on etanercept in order to maintain therapeutic benefit.

Patients with autoinflammatory syndromes may present a clinical course characterized by recurrent, episodic manifestations (such as fever, skin rash or visceral involvement) or they may show a continuous, unremitting disease course with persistent clinical manifestations. Patients with certain diseases, such as CAPS or Blau syndrome, usually present this type of course.

Background: Background/Purpose Systemic lupus erythematosus (SLE) is a prototype autoimmune disease. The most widely used classification criteria for SLE were those developed by the American College of Rheumatology (ACR) in 1982. The Systemic Lupus Collaborating Clinics (SLICC) revised the ACR SLE classification criteria and validated new criteria in adults. Objectives To assess sensitivity and specificity of revised and validated new SLICC SLE classification criteria in a cohort of Juvenile SLE (JSLE). Methods The SLICC criteria rule for SLE classification requires: 1) four criteria, with at least one clinical criterion and one immunological criterion or 2) lupus nephritis alone in the presence of ANA or anti-DNA antibodies. Cases were JSLE patients who have attended a single tertiary center in the past 10 years. JSLE had been diagnosed on clinical and immunological grounds by experienced pediatric rheumatologist. Controls were patients with rheumatic diseases other than SLE: Juvenile Idiopathic Arthritis (JIA); Juvenile Dermatomyositis (JDM), Autoimmune Hepatitis (AH), Juvenile Systemic Sclerosis (JSS), ANCA-associated vasculitis (AAV) and Henoch Schonlein Purpura (HSP). Clinical, immunological and pathological were reviewed from prospectively developed databases and medical records in order to establish the number and frequency of new criteria. Statistics included overall sensitivity and specificity. McNemar`s test was used to assess differences between ACR criteria and new current criteria. Results Cases: 107 patients with JSLE were included (F: 89 M: 18), age at onset: 12 (3-16) yo. Controls: 124 patients with JIA (36 patients); JDM (28), AH (28), JSS (10), AAV(12), HSP (10) F:95 M: 29, age at onset: 11 (2-16)yo. SLICC SLE criteria sensitivity was 100 % vs 86% ACR criteria, while specificity was 98% vs 96% (p=0.009). Six patients with a clinical diagnosis of JSLE were correctly classified by SLICC but not by ACR criteria. Conclusions The SLICC new criteria performed better than the ACR 1997 criteria in a cohort of patients with JSLE. Validation of these criteria in a wider, multiethnic cohort of patients with JSLE is necessary. References Disclosure of Interest None Declared

The juvenile spondyloarthropathies (JSpA) are a group of related rheumatic diseases characterized by involvement of peripheral large joints, axial joints, and entheses (enthesitis) that begin in the early years of life (prior to 16(th) birthday).The nomenclature and concept of spondyloarthropathies has changed during the last few decades. Although there is not any specific classification of JSpA, diseases under the spondyloarthropathy nomenclature umbrella in the younger patients include: the seronegative enthesitis and arthropathy (SEA) syndrome, juvenile ankylosing spondylitis, reactive arthritis, and inflammatory bowel disease-associated arthritis. Moreover, the ILAR criteria for Juvenile Idiopathic Arthritis includes two categories closely related to spondyloarthritis: Enthesitis-related arthritis and psoriatic arthritis.We review the pathophysiology and the use of biological agents in JSpA. JSpA are idiopathic inflammatory diseases driven by an altered balance in the proinflammatory cytokines. There is ample evidence on the role of tumor necrosis factor (TNF) and interleukin-17 in the physiopathology of these entities. Several non-biologic and biologic agents have been used with conflicting results in the treatment of these complex diseases. The efficacy and safety of anti-TNF agents, such as etanercept, infliximab and adalimumab, have been analysed in controlled and uncontrolled trials, usually showing satisfactory outcomes. Other biologic agents, such as abatacept, tocilizumab and rituximab, have been insufficiently studied and their role in the therapy of SpA is uncertain. Interleukin-17-blocking agents are promising alternatives for the treatment of JSpA patients in the near future. Recommendations for the treatment of patients with JSpA have recently been proposed and are discussed in the present review.

ss Open Acce Poster presentation Differences in therapeutic approach to juvenile dermatomyositis between Europe and Latin America L Trail*1, C Ferrari1, R Cuttica2, MM Katsicas3, R Russo3, M Bandeira4, V Ferriani5, S Oliveira6, C Saad-Magalhaes7, CA Silva8, V Baca9, R BurgosVargas10, E Solis-Vallejo11, S Maillard12, C Pilkington12, R Barcellona1, M Beltramelli1, L Breda1, C Bruno1, R Cimaz1, E Cortis1, R Gallizzi1, F Garofalo1, A Meini1, R Podda1, A Stabile1, A Martini1 and A Ravelli1

Case reports Neuromyelitis optica (NMO, Devic's disease) is a severe autoimmune disorder predominantly involving optic nerves and spinal cord [1]. Usually isolated, it has been associated with systemic autoimmune diseases in adult patients [2]. We report 2 children with a systemic autoimmune disease who developed NMO. Patients were female; NMO symptoms started at age 8 and 12 years. Patient 1 had a diagnosis of Systemic Lupus Erythematosus (SLE) (fever, cytopenias, mesangeal glomerulonephritis, positive ANA, anti-DNA, anti-Sm). She developed vomiting, tremor, hyperreflexia, paresthesia, neurogenic bladder and progressive vision loss 2 years after SLE onset. Neuroradiological investigations disclosed longitudinally extensive transverse myelitis and bilateral optic nerve involvement. Patient 2 had recurrent parotitis for 1 year before she developed vision loss, papilitis, dystonia and paresthesia. She exhibited positive ANA, anti-Ro and antiLa, and objective eye dryness. MRI evidenced lesions in brainstem and spinal cord, and evoked potentials revealed optic nerve involvement. A diagnosis of Sjogren's Syndrome (SS) associated with NMO was made. NMO-Ig was detected in both patients' sera. Intravenous and p.o. high dose steroids, a 6-month course of monthly I.V. cyclophosphamide (up to 1 g/m2/dose), followed by azathioprine (2–3 mg/Kg/day) as maintenance therapy, were used in both (plasmapheresis in one). Visual, motor and sensitive symptoms dramatically improved. Mild relapses occurred in both children; they were successfully treated with steroids. Patients are currently well, with improved vision and residual lesions in MRI. Conclusion NMO can occur in the setting SLE or SS in children. Intensive immunosuppressive therapy may induce remission and prevent visual loss.