Denise Pires Marafon

Temporomandibular Joint Involvement is Associated with Quality of Life, Disability and High Disease Activity in Juvenile Idiopathic Arthritis

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.

Anakinra in Systemic Juvenile Idiopathic Arthritis: A Single-center Experience.

Objective. To assess anakinra as a therapy for systemic juvenile idiopathic arthritis (sJIA) in a single-center series. Methods. We reviewed 25 patients with sJIA treated with anakinra for at least 6 months. The primary outcome was the number of patients who achieved clinically inactive disease at 6 months, according to preliminary criteria for inactive disease and clinical remission of JIA. Results. Among 25 patients evaluated, 14 (56%) met the criteria for inactive disease at 6 months and were classified as responders. For each individual patient, we compared the dose administered with the ideal dose of anakinra and we found that there was no relation with response. We also compared demographic characteristics and clinical and laboratory features at baseline in responders and non-responders: no differences were observed in relation with the number of active joints before starting anakinra or concomitant glucocorticoids treatment. The only variable significantly associated with response was the time from disease onset to receiving anakinra, with earlier treatment being associated with a better outcome. Conclusion. Anakinra is associated with rapid attainment of inactive disease in a significant portion of patients. We found that only the earlier treatment is associated with better outcome. However, formal studies on early treatment and on the pathophysiology and response to treatments, including anakinra, of early- and late-onset sJIA are needed to optimize the management of this challenging disease.

Predictors of Relapse after Discontinuing Systemic Treatment in Childhood Autoimmune Chronic Uveitis

Objective. To identify clinical predictors of relapse in childhood autoimmune chronic uveitis after stopping systemic treatment. Methods. A retrospective, multicenter, cohort study. Results. Ninety-four children in remission, receiving no treatments and with at least a 6-month followup, were enrolled. A higher probability of maintaining remission after discontinuing treatment was shown in idiopathic compared with juvenile idiopathic arthritis uveitis (Mantel-Cox chi-square = 23.21) if inactivity had been obtained within 6 months from starting systemic treatment (Mantel-Cox chi-square = 24.17) and by antitumor necrosis factor-α treatment (Mantel-Cox chi-square = 6.43). Conclusion. Type of disease, time, and type of systemic therapy to achieve inactivity predict different duration of uveitis remission after treatment withdrawal.

Lipid profiles in a large cohort of Italian children with Down syndrome

OBJECTIVES Results of epidemiological studies of lipid profiles in individuals with Down Syndrome (DS) in different settings showed discordant results but laboratory norms for this population has been lacking. The aim of our study is to evaluate lipid profiles in a large population of Italian children with DS. METHODS Lipid profiles of 357 patients with diagnosis of DS were recorded. RESULTS Multiple linear regression was employed to estimate models for each lipid fraction as a function of sex and age in patients with DS. CONCLUSIONS The main contribution of this paper is to provide data about lipid profile on a large cohort of people with Down syndrome. Long-term surveillance will be crucial to establish if this specific lipid profile may translate into increased morbidity and mortality from cardiovascular diseases (CVD).

Development and testing of a hybrid measure of muscle strength in juvenile dermatomyositis for use in routine care

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 in 8 muscles (MMT‐8) and the Childhood Myositis Assessment Scale (CMAS) but is more comprehensive than the former and more feasible than the latter.

Switched Memory B Cells Are Increased in Oligoarticular and Polyarticular Juvenile Idiopathic Arthritis and Their Change Over Time Is Related to Response to Tumor Necrosis Factor Inhibitors

To investigate whether abnormalities in B cell subsets in patients with juvenile idiopathic arthritis (JIA) correlate with clinical features and response to treatment.

A Meta‐Analysis to Estimate the Placebo Effect in Juvenile Idiopathic Arthritis in Randomized Controlled Trials

To estimate the placebo effect in juvenile idiopathic arthritis (JIA) through a meta‐analysis of phase III clinical trials with placebo comparator.

A Meta-Analysis to Estimate the Placebo Effect in Randomized Controlled Trials in Juvenile Idiopathic Arthritis.

To estimate the placebo effect in juvenile idiopathic arthritis (JIA) through a meta‐analysis of phase III clinical trials with placebo comparator.

Anakinra in a Cohort of Children with Chronic Nonbacterial Osteomyelitis

Objective. To report efficacy and safety in patients with chronic nonbacterial osteomyelitis (CNO) unresponsive to nonsteroidal antiinflammatory drugs (NSAID) and bisphosphonates and/or glucocorticoids treated with anakinra. Methods. Nine patients (6 females) with refractory CNO were treated with anakinra for at least 6 months. We recorded, at baseline and after 6 months of treatment, clinical and laboratory features, and number and distribution of bone lesions detected by 99mTc-MDP bone scintigraphy. Disease activity was evaluated using a physician’s global assessment (PGA). Results. At baseline, 9/9 patients had mild to severe PGA. After 6 months of treatment, in 5 patients the PGA score was graded from none to minimal. At baseline, erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP) were elevated in 8 out of 9 patients. After 6 months, 5/9 patients had normalized CRP and ESR and in all except 1, CRP and ESR decreased. Before starting anakinra, a total of 77 bone lesions were detected by bone scintigraphy. After 6 months of treatment of the 77 lesions, 42 had resolved and 35 were stable. In 7/9 patients, 20 new lesions appeared during treatment; 2 of these 7 patients were symptomatic. At the last followup visit (median 1.7 yrs, range 0.8–2.8), 6/9 patients maintained a PGA graded as none to minimal. Conclusion. Anakinra is a possible therapeutic alternative in patients with refractory CNO. The practical significance of clinically silent bone lesions detected by bone scintigraphy remains to be established.

PReS-FINAL-2011: Preliminary validation of a new hybrid measure of muscle strength for juvenile dermatomyositis

Juvenile dermatomyositis (JDM) is a multisystem vasculopathic disease characterized by muscle inflammation that causes symmetrical muscle weakness. Assessment of muscle strength is, therefore, a fundamental component of the clinical evaluation of children with JDM. This assessment is traditionally made using the 8-muscle Manual Muscle Testing (MMT) and the Childhood Myositis Assessment Scale (CMAS). However, the MMT does not cover all muscles or muscle groups affected in JDM, namely abdominal muscles. Although the CMAS is more comprehensive than the MMT, it is lengthy and, therefore, may not be feasible in a busy clinical setting or when a physical therapist is not available.