Insights into the clinical presentation of juvenile systemic lupus erythematosus: the PRAGMA monocentric cohort of 177 patients.

Objectives: The aim of this work is to describe the clinical manifestations at onset and during follow-up in a monocentric cohort of patients with juvenile systemic lupus erythematosus (jSLE) from the Paediatric Rheumatology group of the Milan area (PRAGMA).

Methods: Patients were retrospectively included in case of i) SLE diagnosis according to the 1997 American College of Rheumatology or the 2012 SLICC classification criteria and ii) disease onset before 18 years.

Results: Among the 177 recruited patients (155 females), haematologic involvement was the most common disease manifestation (75%), followed by joint and cutaneous involvements (70% and 57%, respectively).

Canakinumab in patients with systemic juvenile idiopathic arthritis and active systemic features: results from the 5-year long-term extension of the phase III pivotal trials.

Objectives: To evaluate the long-term efficacy and safety of canakinumab in patients with active systemic juvenile idiopathic arthritis (JIA).

Methods: Patients (2-19 years) entered two phase III studies and continued in the long-term extension (LTE) study. Efficacy assessments were performed every 3 months, including adapted JIA American College of Rheumatology (aJIA-ACR) criteria, Juvenile Arthritis Disease Activity Score (JADAS) and ACR clinical remission on medication criteria (CR).

Correction to: The Italian version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR).

The family name of author Francesco La Torre was incorrect in the published article. The correct family name should read as La Torre F.

The Italian version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR).

The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) is a new parent/patient reported outcome measure that enables a thorough assessment of the disease status in children with juvenile idiopathic arthritis (JIA). We report the results of the cross-cultural adaptation and validation of the parent and patient versions of the JAMAR in the Italian language.The reading comprehension of the questionnaire was tested in 10 JIA parents and patients.

Disease status, reasons for discontinuation and adverse events in 1038 Italian children with juvenile idiopathic arthritis treated with etanercept.

Background: Data from routine clinical practice are needed to further define the efficacy and safety of biologic medications in children with juvenile idiopathic arthritis (JIA). The aim of this analysis was to investigate the disease status, reasons for discontinuation and adverse events in Italian JIA patients treated with etanercept (ETN).

Methods: In 2013, all centers of the Italian Pediatric Rheumatology Study Group were asked to make a census of patients given ETN after January 2000.

Temporomandibular Joint Involvement in Association With Quality of Life, Disability, and High Disease Activity in Juvenile Idiopathic Arthritis.

Objective: To evaluate the demographic, disease activity, disability, and health-related quality of life (HRQOL) differences between children with juvenile idiopathic arthritis (JIA) and their healthy peers, and between children with JIA with and without clinical temporomandibular joint (TMJ) involvement and its determinants.

Methods: This study is based on a cross-sectional cohort of 3,343 children with JIA and 3,409 healthy peers, enrolled in the Pediatric Rheumatology International Trials Organisation HRQOL study or in the methotrexate trial. Potential determinants of TMJ involvement included demographic, disease activity, disability, and HRQOL measures selected through univariate and multivariable logistic regression.

Recurrent pericarditis in children and adolescents: a multicentre cohort study.

Objective: Limited data are available about recurrent pericarditis in children. We sought to explore contemporary causes, characteristics, therapies and outcomes of recurrent pericarditis in paediatric patients.

Methods: A multicentre (eight sites) cohort study of 110 consecutive cases of paediatric patients with at least two recurrences of pericarditis over an 11-year period (2000-2010) [median 13 years, interquartile range (IQR) 5, 69 boys].

Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial.

Background: Most data for treatment of dermatomyositis and juvenile dermatomyositis are from anecdotal, non-randomised case series. We aimed to compare, in a randomised trial, the efficacy and safety of prednisone alone with that of prednisone plus either methotrexate or ciclosporin in children with new-onset juvenile dermatomyositis.

Methods: We did a randomised trial at 54 centres in 22 countries.

Nervous system involvement in Farber disease.

A 30 months-old boy with Farber disease developed nystagmus 12 months after hematopoietic stem cell transplantation (HSCT). At 40 months, gait ataxia was evident, and brain MRI showed increased size of pericerebellar sulci and 4th ventricle. EMG showed denervation in the tongue and upper limb muscles, consistent with motor neuron disease.

May biscuits contribute to iron balance? An observation in children with juvenile idiopatic arthritis.

Within an observational open study on the effects of a scheduled dosage of biscuits with iron, children with juvenile idiopathic arthritis were either supplemented with biscuits supplying iron fumarate (median 3.6 mg per day) or left to their customary dietary habits. After 4 months, supplemented children showed a more favourable percentage change of blood haemoglobin, while ferritin levels (markers of inflammation) remained stable.

Anti-nuclear antibodies as predictor of outcome in a multi-center cohort of Italian children and adolescents with Raynaud's phenomenon.

A retrospective multi-center data collection of clinical, laboratory, and treatment characteristics of 94 Caucasian children and adolescents with Raynaud's phenomenon (RP) started at a mean age of 12.8 ± 5 years, with variable involvement of hands, feet, and face, was performed for a period of 3 years. Collected data included nailfold videocapillaroscopy (NVC), lung function tests, and different laboratory tests finalized to characterize an eventual connective tissue disease (CTD), disclosed by RP itself.

Immunogenicity, safety and tolerability of a bivalent human papillomavirus vaccine in adolescents with juvenile idiopathic arthritis.

Aims: To evaluate the immunogenicity, safety and tolerability of the bivalent HPV vaccine in female patients with juvenile idiopathic arthritis (JIA).

Methods: Twenty-one patients with JIA aged 12-25 years and 21 healthy controls were enrolled and received three doses of the bivalent HPV vaccine.

Results: All of the subjects were seronegative at baseline and seroconverted after the scheduled doses.

Performance of current guidelines for diagnosis of macrophage activation syndrome complicating systemic juvenile idiopathic arthritis.

Objective: To compare the capacity of the 2004 diagnostic guidelines for hemophagocytic lymphohistiocytosis (HLH-2004) with the capacity of the preliminary diagnostic guidelines for systemic juvenile idiopathic arthritis (JIA)-associated macrophage activation syndrome (MAS) to discriminate MAS complicating systemic JIA from 2 potentially confusable conditions, represented by active systemic JIA without MAS and systemic infection.

Methods: International pediatric rheumatologists and hemato-oncologists were asked to retrospectively collect clinical information from patients with systemic JIA-associated MAS and confusable conditions. The ability of the guidelines to differentiate MAS from the control diseases was evaluated by calculating the sensitivity and specificity of each set of guidelines and the kappa statistics for concordance with the physician's diagnosis.

Clinical overview and outcome in a cohort of children with polyarteritis nodosa.

Objectives: Polyarteritis nodosa (PAN) is a rare vasculitis in childhood and poor information is known about its long-term outcome. Our aim was to describe the clinical features, at onset and during the disease course, of childhood-onset PAN and identify a potential correlation with persistent organ damage and worse outcome in a cohort of paediatric patients with a confirmed diagnosis of PAN.

Methods: A retrospective collection of demographic and clinical data of 52 Caucasian children diagnosed with PAN, fulfilling the EULAR/PRES diagnostic criteria, recruited from eight paediatric rheumatologic centres and one transition unit, was performed.

Chorea, a little-known manifestation in systemic lupus erythematosus: short literature review and four case reports.

Chorea is a movement disorder that may be found in children due to several causes. Here we focus especially on Systemic Lupus Erythematosus associated chorea. First we outline its epidemiology, hypothesized pathogenesis, clinical presentation and treatment, then we report four significant clinical cases, which represent well the extreme variability of set of symptoms that may accompany lupus chorea.

Lupus nephritis in children and adolescents: results of the Italian Collaborative Study.

Background: Lupus nephritis (LN) strongly affects the outcome in children with systemic lupus erythematosus (SLE). Many patients, however, have renal disease at onset, but lack a sufficient number of criteria to be diagnosed as SLE and develop delayed symptoms over time (d-SLE). Data on the clinical course, long-term outcome and predictors of disease progression in children with LN are scant.

Safety and efficacy of infliximab and adalimumab for refractory uveitis in juvenile idiopathic arthritis: 1-year followup data from the Italian Registry.

Objective: To evaluate safety and efficacy of adalimumab (ADA) and infliximab (IFX) for the treatment of juvenile idiopathic arthritis-related anterior uveitis (JIA-AU).

Methods: Starting January 2007, patients with JIA-AU treated with IFX and ADA were managed by a standard protocol and data were entered into the National Italian Registry (NIR). At baseline, all patients were refractory to standard immunosuppressive treatment and/or were corticosteroid-dependent.

A preliminary disease severity score for juvenile systemic sclerosis.

Objective: To develop a preliminary disease severity score for juvenile systemic sclerosis (SSc).

Methods: We conducted an evidence- and consensus-based study that included the following 5 phases: 1) prospective collection of data regarding the demographic and clinical characteristics of patients with diffuse juvenile SSc who were followed up for at least 4 years or until death; 2) blinded evaluation of the disease course profiles of these patients by experts in juvenile SSc, so that patient profiles with a defined clinical course could be used as the gold standard for the score validation phase; 3) definition of candidate severity indices to be included in potential scores; 4) selection of the pediatric severity score with the best statistical performance, as determined by its ability to classify individual patients as having improvement or worsening of disease compared with baseline values or the previous evaluation; 5) validation of the efficiency of the selected score in patients with a mild, moderate, or severe disease course and comparison with the Medsger severity score for adults with SSc.

Results: Thirty-five patients classified as having a mild (n = 17), moderate (n = 10), or severe (n = 8) disease course entered the study.

Immunogenicity, safety and tolerability of MF59-adjuvanted seasonal influenza vaccine in children with juvenile idiopathic arthritis.

In order to evaluate the immunogenicity, safety, and tolerability of the MF-59 adjuvanted seasonal influenza vaccine in children and adolescents with juvenile idiopathic arthritis (JIA) treated with different anti-rheumatic drugs, 60 pediatric patients with JIA (30 treated with disease-modifying anti-rheumatic drugs [DMARDs] and 30 with etanercept) were compared with 30 healthy controls of similar gender and age. All of the patients received a single dose of the MF59-adjuvanted seasonal influenza vaccine (Fluad, Siena, Italy). Immunogenicity was assessed at baseline, and 1 and 3 months post-vaccination; safety and tolerability were also evaluated during the study period.

Vaccination of children and adolescents with rheumatic diseases.

Children with rheumatic diseases (RDs) are at greater risk of infection because of their aberrant immunity and frequent use of immunosuppressive drugs. However, the use of vaccinations in such children is debated by many experts who think that the patients' immune response is insufficient to assure protection; some of them are also afraid that vaccines could trigger a persistent autoimmune response and lead to severe clinical problems including a relapse of the RD. This review describes the available data regarding the risks of vaccine administration, and the immunogenicity, efficacy and tolerability of the vaccines usually recommended for children with RDs.

Comparison of clinical features and drug therapies among European and Latin American patients with juvenile dermatomyositis.

Objectives: To compare the demographic features, presenting manifestations, diagnostic investigations, disease course, and drug therapies of children with juvenile dermatomyositis (JDM) followed in Europe and Latin America.

Methods: Patients were inception cohorts seen between 1980 and 2004 in 27 paediatric rheumatology centres. The following information was collected through the review of patient charts: sex; age at disease onset; date of disease onset and diagnosis; onset type; presenting clinical features; diagnostic investigations; course type; and medications received during disease course.