Pediatria II

Genova, Italy

Pediatria II

Genova, Italy
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Maggio A.,Cervello | Vitrano A.,University of Palermo | Lucania G.,Cervello | Capra M.,Uoc Centro Of Prevenzione Diagnosi E Cura Della Talassemia | And 16 more authors.
American Journal of Hematology | Year: 2012

A multicenter randomized open-label long-term sequential deferiprone-deferoxamine (DFP-DFO) versus DFP alone trial (sequential DFP-DFO) performed in patients with thalassemia major (TM) was retrospectively reanalyzed to assess the variation in the left ventricular ejection fraction (LVEF) [1].

Malattia C.,Pediatria II | Damasio M.B.,Instituto G Gaslini | Pistorio A.,Instituto G Gaslini | Ioseliani M.,Pediatria II | And 11 more authors.
Annals of the Rheumatic Diseases | Year: 2011

Objectives: To develop and validate a paediatric-targeted MRI scoring system for the assessment of disease activity and damage in juvenile idiopathic arthritis (JIA). To compare the paediatric MRI score with the adult-designed. Outcome Measures in Rheumatology Clinical Trials - Rheumatoid Arthritis MRI Score (RAMRIS), whose suitability for assessing growing joints was tested. Methods: In 66 patients with JIA the clinically more affected wrist was studied. Thirty-nine patients had a 1-year MRI follow-up. Two readers independently assigned the paediatric score and the RAMRIS to all studies. Validation procedures included analysis of reliability, construct validity and responsiveness to change. A reduced version of the bone erosion score was also developed and tested. Results: The paediatric score showed an excellent reproducibility (interclass correlation coefficient >0.9). The interobserver agreement of RAMRIS was moderate for bone erosions and excellent for bone marrow oedema (BMO). The paediatric score and RAMRIS provided similar results for construct validity. The responsiveness to change of the paediatric score was moderate for synovitis and bone erosion, and poor for BMO and did not improve when RAMRIS was applied. The reduced version of the bone erosion was valuable for the assessment of joint damage, and provided time-saving advantages. Conclusion: The results demonstrate that the paediatric MRI score is a reliable and valid method for assessing disease activity and damage in JIA. Unexpectedly, the RAMRIS provides acceptable suitability for use in the paediatric age group. Further work, especially in a longitudinal setting, is required before defining the most suitable MRI scale for assessing growing joints.

Ruperto N.,Pediatria II | Martini A.,University of Genoa
Archives of Disease in Childhood | Year: 2011

Networking is key to overcoming the logistical, methodological and ethical problems related to the implementation of paediatric studies. The adoption of legislation to encourage paediatric clinical trials by the American and European regulatory agencies has opened a new era in the assessment of drug safety and efficacy in children. Two very large international trial networks - the Pediatric Rheumatology Collaborative Study Group (PRCSG) and the Paediatric Rheumatology International Trials Organisation (PRINTO) - have played a critical role in the implementation of this legislation and have facilitated several successful controlled studies on the safety and the efficacy of new and old drugs in paediatric rheumatic diseases. The PRINTO and PRCSG networks can be seen as a model for international co-operation in other paediatric subspecialties.

Van Dijkhuizen E.H.P.,Pediatria II | Van Dijkhuizen E.H.P.,University Utrecht | Wulffraat N.M.,University Utrecht
Annals of the Rheumatic Diseases | Year: 2015

Objectives: Juvenile idiopathic arthritis (JIA) is subdivided into seven categories. Even within these categories, the prognosis varies markedly. To start appropriate treatment in patients with JIA and to inform patients and their parents correctly, it is essential to know the individual prognosis, preferably at the time of diagnosis. The aim of this study was to identify variables that predict disease activity, joint damage, functional ability and quality of life (QoL) early in the disease course. Methods: A systematic literature review was performed, and 3679 articles were identified. The results were screened and critically appraised using predefined criteria. Articles that described validated outcomes, such as the Wallace criteria, the childhood health assessment questionnaire (CHAQ) and the juvenile arthritis damage index (JADI), and that determined predictors in the first 6months of disease were selected.Results: Forty mostly retrospective articles were selected.Polyarticular onset predicted a worse prognosis for all outcomes, except QoL. A diagnostic delay and the systemic category predicted continuation of active disease. Notably, antinuclear antibodies (ANA) did not predict disease activity. Symmetric involvement and rheumatoid factor positivity predicted less damage. More disease activity was mainly associated with worse functional outcome. However, most predictors were not validated. Conclusions: Few predictors for the selected outcomes were found. Prospective, longitudinal studies using standardised outcome measurements, and evaluating a broader range of predictors, such as genetics, immunological and imaging data, should be performed. For the outcomes joint assessment and quality of life, standardised and validated outcomes should be developed.

OBJECTIVE: To determine the capability and reliability of dynamic contrast-enhanced MRI (DCE-MRI) in the assessment of disease activity in juvenile idiopathic arthritis (JIA). METHODS: DCE-MRI of the clinically more affected wrist or hip joints was undertaken in 21 patients, coupled with standard clinical assessment and biochemical analysis. Synovial inflammation was assessed by computing the maximum level of synovial enhancement (ME), the maximum rate of enhancement (MV) and the rate of early enhancement (REE) from the enhancement curves generated from region of interest independently delineated by two readers in the area of the ME. Correlations between dynamic parameters and clinical measures of disease activity, and static MRI synovitis score were investigated. RESULTS: In patients with wrist arthritis, REE correlated with the wrist swelling score (r(s) = 0.72), ESR (r(s) = 0.69), pain assessment scale (r(s) = 0.63) and childhood HAQ (r(s) = 0.60). In patients with hip arthritis, ME correlated with the hip limitation of motion (r(s) = 0.69). Static MRI synovitis score based on post-gadolinium enhancement correlated with MV (r(s) = 0.63) in patients with wrist arthritis and with ME (r = 0.68) in those with hip arthritis. The inter-reader agreement assessed by intra-class correlation coefficient (ICC) for ME, MV and REE (ICC = 0.98, 0.97 and 0.84, respectively) was excellent. CONCLUSIONS: DCE-MRI represents a promising method for the assessment of disease activity in JIA, especially in patients with wrist arthritis. As far as we know, this study is the first to demonstrate the feasibility, reliability and construct validity of DCE-MRI in JIA. These results should be confirmed in large-scale longitudinal studies in view of its further application in therapeutic decision making and in clinical trials.

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