<?xml version="1.0" encoding="UTF-8"?><xml><records><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Caorsi, Roberta</style></author><author><style face="normal" font="default" size="100%">Penco, Federica</style></author><author><style face="normal" font="default" size="100%">Grossi, Alice</style></author><author><style face="normal" font="default" size="100%">Insalaco, Antonella</style></author><author><style face="normal" font="default" size="100%">Omenetti, Alessia</style></author><author><style face="normal" font="default" size="100%">Alessio, Maria</style></author><author><style face="normal" font="default" size="100%">Conti, Giovanni</style></author><author><style face="normal" font="default" size="100%">Marchetti, Federico</style></author><author><style face="normal" font="default" size="100%">Picco, Paolo</style></author><author><style face="normal" font="default" size="100%">Tommasini, Alberto</style></author><author><style face="normal" font="default" size="100%">Martino, Silvana</style></author><author><style face="normal" font="default" size="100%">Malattia, Clara</style></author><author><style face="normal" font="default" size="100%">Gallizi, Romina</style></author><author><style face="normal" font="default" size="100%">Podda, Rosa Anna</style></author><author><style face="normal" font="default" size="100%">Salis, Annalisa</style></author><author><style face="normal" font="default" size="100%">Falcini, Fernanda</style></author><author><style face="normal" font="default" size="100%">Schena, Francesca</style></author><author><style face="normal" font="default" size="100%">Garbarino, Francesca</style></author><author><style face="normal" font="default" size="100%">Morreale, Alessia</style></author><author><style face="normal" font="default" size="100%">Pardeo, Manuela</style></author><author><style face="normal" font="default" size="100%">Ventrici, Claudia</style></author><author><style face="normal" font="default" size="100%">Passarelli, Chiara</style></author><author><style face="normal" font="default" size="100%">Zhou, Qing</style></author><author><style face="normal" font="default" size="100%">Severino, Mariasavina</style></author><author><style face="normal" font="default" size="100%">Gandolfo, Carlo</style></author><author><style face="normal" font="default" size="100%">Damonte, Gianluca</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author><author><style face="normal" font="default" size="100%">Ravelli, Angelo</style></author><author><style face="normal" font="default" size="100%">Aksentijevich, Ivona</style></author><author><style face="normal" font="default" size="100%">Ceccherini, Isabella</style></author><author><style face="normal" font="default" size="100%">Gattorno, Marco</style></author></authors></contributors><titles><title><style face="normal" font="default" size="100%">ADA2 deficiency (DADA2) as an unrecognised cause of early onset polyarteritis nodosa and stroke: a multicentre national study.</style></title><secondary-title><style face="normal" font="default" size="100%">Ann Rheum Dis</style></secondary-title><alt-title><style face="normal" font="default" size="100%">Ann. Rheum. Dis.</style></alt-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Adenosine Deaminase</style></keyword><keyword><style  face="normal" font="default" size="100%">Adolescent</style></keyword><keyword><style  face="normal" font="default" size="100%">Age of Onset</style></keyword><keyword><style  face="normal" font="default" size="100%">Case-Control Studies</style></keyword><keyword><style  face="normal" font="default" size="100%">Child</style></keyword><keyword><style  face="normal" font="default" size="100%">Child, Preschool</style></keyword><keyword><style  face="normal" font="default" size="100%">DNA Mutational Analysis</style></keyword><keyword><style  face="normal" font="default" size="100%">Female</style></keyword><keyword><style  face="normal" font="default" size="100%">Heterozygote</style></keyword><keyword><style  face="normal" font="default" size="100%">Homozygote</style></keyword><keyword><style  face="normal" font="default" size="100%">Humans</style></keyword><keyword><style  face="normal" font="default" size="100%">Immunoglobulins</style></keyword><keyword><style  face="normal" font="default" size="100%">Immunosuppressive Agents</style></keyword><keyword><style  face="normal" font="default" size="100%">Infant</style></keyword><keyword><style  face="normal" font="default" size="100%">Intercellular Signaling Peptides and Proteins</style></keyword><keyword><style  face="normal" font="default" size="100%">Italy</style></keyword><keyword><style  face="normal" font="default" size="100%">Livedo Reticularis</style></keyword><keyword><style  face="normal" font="default" size="100%">Male</style></keyword><keyword><style  face="normal" font="default" size="100%">Pedigree</style></keyword><keyword><style  face="normal" font="default" size="100%">Polyarteritis Nodosa</style></keyword><keyword><style  face="normal" font="default" size="100%">Stroke</style></keyword><keyword><style  face="normal" font="default" size="100%">Thalidomide</style></keyword><keyword><style  face="normal" font="default" size="100%">Tumor Necrosis Factor-alpha</style></keyword><keyword><style  face="normal" font="default" size="100%">Young Adult</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2017</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2017 Oct</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">76</style></volume><pages><style face="normal" font="default" size="100%">1648-1656</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;OBJECTIVES: &lt;/b&gt;To analyse the prevalence of  mutations in patients diagnosed with early onset livedo reticularis and/or haemorrhagic/ischaemic strokes in the context of inflammation or polyarteritis nodosa (PAN). Forty-eight patients from 43 families were included in the study.&lt;/p&gt;&lt;p&gt;&lt;b&gt;METHODS: &lt;/b&gt;Direct sequencing of  was performed by Sanger analysis. Adenosine deaminase 2 (ADA2) enzymatic activity was analysed in monocyte isolated from patients and healthy controls incubated with adenosine and with or without an ADA1 inhibitor.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;Biallelic homozygous or compound heterozygous  mutations were detected in 15/48 patients. A heterozygous disease-associated mutation (p.G47V) was observed in two affected brothers. The mean age of onset of the genetically positive patients was 24 months (6 months to 7 years). Ten patients displayed one or more cerebral strokes during their disease course. Low immunoglobulin levels were detected in six patients. Thalidomide and anti-TNF (tumour necrosis factor) blockers were the most effective drugs. Patients without  mutations had a later age at disease onset, a lower prevalence of neurological and skin manifestations; one of these patients displayed all the clinical features of adenosine deaminase 2deficiency (DADA2) and a defective enzymatic activity suggesting the presence of a missed mutation or a synthesis defect.&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSIONS: &lt;/b&gt;DADA2 accounts for paediatric patients diagnosed with PAN-like disease and strokes and might explain an unrecognised condition in patients followed by adult rheumatologist. Timely diagnosis and treatment with anti-TNF agents are crucial for the prevention of severe complications of the disease. Functional assay to measure ADA2 activity should complement genetic testing in patients with non-confirming genotypes.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">10</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/28522451?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Ravelli, Angelo</style></author><author><style face="normal" font="default" size="100%">Davì, Sergio</style></author><author><style face="normal" font="default" size="100%">Bracciolini, Giulia</style></author><author><style face="normal" font="default" size="100%">Pistorio, Angela</style></author><author><style face="normal" font="default" size="100%">Consolaro, Alessandro</style></author><author><style face="normal" font="default" size="100%">van Dijkhuizen, Evert Hendrik Pieter</style></author><author><style face="normal" font="default" size="100%">Lattanzi, Bianca</style></author><author><style face="normal" font="default" size="100%">Filocamo, Giovanni</style></author><author><style face="normal" font="default" size="100%">Verazza, Sara</style></author><author><style face="normal" font="default" size="100%">Gerloni, Valeria</style></author><author><style face="normal" font="default" size="100%">Gattinara, Maurizio</style></author><author><style face="normal" font="default" size="100%">Pontikaki, Irene</style></author><author><style face="normal" font="default" size="100%">Insalaco, Antonella</style></author><author><style face="normal" font="default" size="100%">De Benedetti, Fabrizio</style></author><author><style face="normal" font="default" size="100%">Civino, Adele</style></author><author><style face="normal" font="default" size="100%">Presta, Giuseppe</style></author><author><style face="normal" font="default" size="100%">Breda, Luciana</style></author><author><style face="normal" font="default" size="100%">Marzetti, Valentina</style></author><author><style face="normal" font="default" size="100%">Pastore, Serena</style></author><author><style face="normal" font="default" size="100%">Magni-Manzoni, Silvia</style></author><author><style face="normal" font="default" size="100%">Maggio, Maria Cristina</style></author><author><style face="normal" font="default" size="100%">Garofalo, Franco</style></author><author><style face="normal" font="default" size="100%">Rigante, Donato</style></author><author><style face="normal" font="default" size="100%">Gattorno, Marco</style></author><author><style face="normal" font="default" size="100%">Malattia, Clara</style></author><author><style face="normal" font="default" size="100%">Picco, Paolo</style></author><author><style face="normal" font="default" size="100%">Viola, Stefania</style></author><author><style face="normal" font="default" size="100%">Lanni, Stefano</style></author><author><style face="normal" font="default" size="100%">Ruperto, Nicolino</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author></authors><translated-authors><author><style face="normal" font="default" size="100%">Italian Pediatric Rheumatology Study Group</style></author></translated-authors></contributors><titles><title><style face="normal" font="default" size="100%">Intra-articular corticosteroids versus intra-articular corticosteroids plus methotrexate in oligoarticular juvenile idiopathic arthritis: a multicentre, prospective, randomised, open-label trial.</style></title><secondary-title><style face="normal" font="default" size="100%">Lancet</style></secondary-title><alt-title><style face="normal" font="default" size="100%">Lancet</style></alt-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Adrenal Cortex Hormones</style></keyword><keyword><style  face="normal" font="default" size="100%">Arthritis, Juvenile</style></keyword><keyword><style  face="normal" font="default" size="100%">Humans</style></keyword><keyword><style  face="normal" font="default" size="100%">Injections, Intra-Articular</style></keyword><keyword><style  face="normal" font="default" size="100%">Italy</style></keyword><keyword><style  face="normal" font="default" size="100%">Methotrexate</style></keyword><keyword><style  face="normal" font="default" size="100%">Prospective Studies</style></keyword><keyword><style  face="normal" font="default" size="100%">Treatment Outcome</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2017</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2017 03 04</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">389</style></volume><pages><style face="normal" font="default" size="100%">909-916</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;BACKGROUND: &lt;/b&gt;Little evidence-based information is available to guide the treatment of oligoarticular juvenile idiopathic arthritis. We aimed to investigate whether oral methotrexate increases the efficacy of intra-articular corticosteroid therapy.&lt;/p&gt;&lt;p&gt;&lt;b&gt;METHODS: &lt;/b&gt;We did this prospective, open-label, randomised trial at ten hospitals in Italy. Using a concealed computer-generated list, children younger than 18 years with oligoarticular-onset disease were randomly assigned (1:1) to intra-articular corticosteroids alone or in combination with oral methotrexate (15 mg/m; maximum 20 mg). Corticosteroids used were triamcinolone hexacetonide (shoulder, elbow, wrist, knee, and tibiotalar joints) or methylprednisolone acetate (ie, subtalar and tarsal joints). We did not mask patients or investigators to treatment assignments. Our primary outcome was the proportion of patients in the intention-to-treat population who had remission of arthritis in all injected joints at 12 months. This trial is registered with European Union Clinical Trials Register, EudraCT number 2008-006741-70.&lt;/p&gt;&lt;p&gt;&lt;b&gt;FINDINGS: &lt;/b&gt;Between July 7, 2009, and March 31, 2013, we screened 226 participants and randomly assigned 102 to intra-articular corticosteroids alone and 105 to intra-articular corticosteroids plus methotrexate. 33 (32%) patients assigned to intra-articular corticosteroids alone and 39 (37%) assigned to intra-articular corticosteroids and methotrexate therapy had remission of arthritis in all injected joints (p=0·48). Adverse events were recorded for 20 (17%) patients who received methotrexate, which led to permanent treatment discontinuation in two patients (one due to increased liver transaminases and one due to gastrointestinal discomfort). No patient had a serious adverse event.&lt;/p&gt;&lt;p&gt;&lt;b&gt;INTERPRETATION: &lt;/b&gt;Concomitant administration of methotrexate did not augment the effectiveness of intra-articular corticosteroid therapy. Future studies are needed to define the optimal therapeutic strategies for oligoarticular juvenile idiopathic arthritis.&lt;/p&gt;&lt;p&gt;&lt;b&gt;FUNDING: &lt;/b&gt;Italian Agency of Drug Evaluation.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">10072</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/28162781?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Naselli, Aldo</style></author><author><style face="normal" font="default" size="100%">Penco, Federica</style></author><author><style face="normal" font="default" size="100%">Cantarini, Luca</style></author><author><style face="normal" font="default" size="100%">Insalaco, Antonella</style></author><author><style face="normal" font="default" size="100%">Alessio, Mariolina</style></author><author><style face="normal" font="default" size="100%">Tommasini, Alberto</style></author><author><style face="normal" font="default" size="100%">Maggio, Cristina</style></author><author><style face="normal" font="default" size="100%">Obici, Laura</style></author><author><style face="normal" font="default" size="100%">Gallizi, Romina</style></author><author><style face="normal" font="default" size="100%">Cimmino, Marco</style></author><author><style face="normal" font="default" size="100%">Signa, Sara</style></author><author><style face="normal" font="default" size="100%">Lucherini, Orso Maria</style></author><author><style face="normal" font="default" size="100%">Carta, Sonia</style></author><author><style face="normal" font="default" size="100%">Caroli, Francesco</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author><author><style face="normal" font="default" size="100%">Rubartelli, Anna</style></author><author><style face="normal" font="default" size="100%">Ceccherini, Isabella</style></author><author><style face="normal" font="default" size="100%">Gattorno, Marco</style></author></authors></contributors><titles><title><style face="normal" font="default" size="100%">Clinical Characteristics of Patients Carrying the Q703K Variant of the NLRP3 Gene: A 10-year Multicentric National Study.</style></title><secondary-title><style face="normal" font="default" size="100%">J Rheumatol</style></secondary-title><alt-title><style face="normal" font="default" size="100%">J. Rheumatol.</style></alt-title></titles><dates><year><style  face="normal" font="default" size="100%">2016</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2016 Jun</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">43</style></volume><pages><style face="normal" font="default" size="100%">1093-100</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;OBJECTIVE: &lt;/b&gt;The aim of our study was to analyze the clinical and functional effect of the p.Q703K (p. Q705K, c. 2107C&gt;A) variant of the NLRP3 gene in a population of patients screened for suspected cryopyrin-associated periodic syndrome (CAPS).&lt;/p&gt;&lt;p&gt;&lt;b&gt;METHODS: &lt;/b&gt;Since 2002, 580 patients underwent molecular analysis for NLRP3. Data on clinical presentation, response to treatment, and longterm followup were collected using a uniform questionnaire. The pattern of cytokine secretion after lipopolysaccharide stimulation from isolated monocytes was analyzed in 3 patients carrying the p.Q703K variant and 1 patient with a chronic infantile neurologic, cutaneous, articular syndrome phenotype carrying both the p.M406I and p.Q703K, and compared with 7 patients with CAPS with sure pathogenic variants and 6 healthy controls.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;The p.Q703K variant was found in 57 screened patients with an overall allelic frequency of 5%. The frequency in normal controls was 5.5%. Clinical data at the moment of molecular analysis and at followup were available in 36 patients. Two patients displayed additional mutations of NLRP3. The mean followup was 2.5 years. Thirteen patients (39%) had a final diagnosis different from the original suspicion of CAPS. The remaining 21 patients displayed a mild phenotype mainly characterized by recurrent episodes of urticarial rash and arthralgia. Only 8 patients were treated with anti-interleukin (IL)-1 treatment, with a complete response in 5 patients. The pattern of secretion of IL-1β and other cytokines (IL-6 and IL-1 receptor antagonist) in patients did not display the aberrancies observed in patients with CAPS and was similar to that observed in healthy controls.&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSION: &lt;/b&gt;The present study confirms the weak clinical and functional effect of the p.Q703K variant.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">6</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/27036377?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Rusmini, Marta</style></author><author><style face="normal" font="default" size="100%">Federici, Silvia</style></author><author><style face="normal" font="default" size="100%">Caroli, Francesco</style></author><author><style face="normal" font="default" size="100%">Grossi, Alice</style></author><author><style face="normal" font="default" size="100%">Baldi, Maurizia</style></author><author><style face="normal" font="default" size="100%">Obici, Laura</style></author><author><style face="normal" font="default" size="100%">Insalaco, Antonella</style></author><author><style face="normal" font="default" size="100%">Tommasini, Alberto</style></author><author><style face="normal" font="default" size="100%">Caorsi, Roberta</style></author><author><style face="normal" font="default" size="100%">Gallo, Eleonora</style></author><author><style face="normal" font="default" size="100%">Olivieri, Alma Nunzia</style></author><author><style face="normal" font="default" size="100%">Marzano, AngeloValerio</style></author><author><style face="normal" font="default" size="100%">Coviello, Domenico</style></author><author><style face="normal" font="default" size="100%">Ravazzolo, Roberto</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author><author><style face="normal" font="default" size="100%">Gattorno, Marco</style></author><author><style face="normal" font="default" size="100%">Ceccherini, Isabella</style></author></authors></contributors><titles><title><style face="normal" font="default" size="100%">Next-generation sequencing and its initial applications for molecular diagnosis of systemic auto-inflammatory diseases.</style></title><secondary-title><style face="normal" font="default" size="100%">Ann Rheum Dis</style></secondary-title><alt-title><style face="normal" font="default" size="100%">Ann. Rheum. Dis.</style></alt-title></titles><dates><year><style  face="normal" font="default" size="100%">2015</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2015 Sep 17</style></date></pub-dates></dates><language><style face="normal" font="default" size="100%">ENG</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;OBJECTIVES: &lt;/b&gt;Systemic auto-inflammatory disorders (SAIDs) are a heterogeneous group of monogenic diseases sharing a primary dysfunction of the innate immune system. More than 50% of patients with SAID does not show any mutation at gene(s) tested because of lack of precise clinical classification criteria and/or incomplete gene screening. To improve the molecular diagnosis and genotype interpretation of SAIDs, we undertook the development of a next-generation sequencing (NGS)-based protocol designed to simultaneous screening of 10 genes.&lt;/p&gt;&lt;p&gt;&lt;b&gt;METHODS: &lt;/b&gt;Fifty patients with SAID, already genotyped for the respective causative gene(s), were massively sequenced for the coding portions of MEFV, MVK, TNFRSF1A, NLRP3, NLRP12, NOD2, PSTPIP1, IL1RN, LPIN2 and PSMB8. Three different bioinformatic pipelines (Ion Reporter, CLC Bio Genomics Workbench, GATK-based in-house workflow) were compared.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;Once resulting variants were compared with the expected mutation list, no workflow turned out to be able to detect all the 79 variants known in the 50 DNAs. Additional variants were also detected, validated by Sanger sequencing and compared to assess true and false positive detection rates of the three workflows. Finally, the overall clinical picture of 34 patients was re-evaluated in the light of the new mutations found.&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSIONS: &lt;/b&gt;The present gene panel has resulted suitable for molecular diagnosis of SAIDs. Moreover, genotype-phenotype correlation has confirmed that the interpretation of NGS data in patients with an undefined inflammatory phenotype is remarkably difficult, thus supporting the need of evidence-based and validated clinical criteria to be used concurrently with the genetic analysis for the final diagnosis and classification of patients with SAIDs.&lt;/p&gt;</style></abstract><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/26386126?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Hasija, Rachana</style></author><author><style face="normal" font="default" size="100%">Pistorio, Angela</style></author><author><style face="normal" font="default" size="100%">Ravelli, Angelo</style></author><author><style face="normal" font="default" size="100%">Demirkaya, Erkan</style></author><author><style face="normal" font="default" size="100%">Khubchandani, Raju</style></author><author><style face="normal" font="default" size="100%">Guseinova, Dinara</style></author><author><style face="normal" font="default" size="100%">Malattia, Clara</style></author><author><style face="normal" font="default" size="100%">Canhao, Helena</style></author><author><style face="normal" font="default" size="100%">Harel, Liora</style></author><author><style face="normal" font="default" size="100%">Foell, Dirk</style></author><author><style face="normal" font="default" size="100%">Wouters, Carine</style></author><author><style face="normal" font="default" size="100%">De Cunto, Carmen</style></author><author><style face="normal" font="default" size="100%">Huemer, Christian</style></author><author><style face="normal" font="default" size="100%">Kimura, Yukiko</style></author><author><style face="normal" font="default" size="100%">Mangge, Harald</style></author><author><style face="normal" font="default" size="100%">Minetti, Carlo</style></author><author><style face="normal" font="default" size="100%">Nordal, Ellen Berit</style></author><author><style face="normal" font="default" size="100%">Philippet, Pierre</style></author><author><style face="normal" font="default" size="100%">Garozzo, Rosaria</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author><author><style face="normal" font="default" size="100%">Ruperto, Nicolino</style></author></authors><translated-authors><author><style face="normal" font="default" size="100%">Pediatric Rheumatology International Trials Organization</style></author></translated-authors></contributors><titles><title><style face="normal" font="default" size="100%">Therapeutic approaches in the treatment of juvenile dermatomyositis in patients with recent-onset disease and in those experiencing disease flare: an international multicenter PRINTO study.</style></title><secondary-title><style face="normal" font="default" size="100%">Arthritis Rheum</style></secondary-title><alt-title><style face="normal" font="default" size="100%">Arthritis Rheum.</style></alt-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Adolescent</style></keyword><keyword><style  face="normal" font="default" size="100%">Adrenal Cortex Hormones</style></keyword><keyword><style  face="normal" font="default" size="100%">Child</style></keyword><keyword><style  face="normal" font="default" size="100%">Dermatologic Agents</style></keyword><keyword><style  face="normal" font="default" size="100%">Dermatomyositis</style></keyword><keyword><style  face="normal" font="default" size="100%">Female</style></keyword><keyword><style  face="normal" font="default" size="100%">Humans</style></keyword><keyword><style  face="normal" font="default" size="100%">Longitudinal Studies</style></keyword><keyword><style  face="normal" font="default" size="100%">Male</style></keyword><keyword><style  face="normal" font="default" size="100%">Methotrexate</style></keyword><keyword><style  face="normal" font="default" size="100%">Prospective Studies</style></keyword><keyword><style  face="normal" font="default" size="100%">Treatment Outcome</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2011</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2011 Oct</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">63</style></volume><pages><style face="normal" font="default" size="100%">3142-52</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;OBJECTIVE: &lt;/b&gt;To evaluate response to therapy over a 24-month period in a large prospective international cohort of patients with juvenile dermatomyositis (DM).&lt;/p&gt;&lt;p&gt;&lt;b&gt;METHODS: &lt;/b&gt;The study included 145 patients with recent-onset juvenile DM and 130 juvenile DM patients experiencing disease flare, all of whom were &lt;18 years old. Disease activity parameters and therapeutic approaches in 4 geographic areas were analyzed at baseline and at 6, 12, and 24 months. Response was assessed according to the Pediatric Rheumatology International Trials Organization (PRINTO) juvenile DM response criteria, and data were reported &quot;as observed&quot; and in the intent-to-treat (ITT) population.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;Patients with recent-onset juvenile DM at baseline had higher baseline disease activity and greater improvement over 24 months when compared to juvenile DM patients experiencing disease flare at baseline. Methotrexate (MTX) or high-dose corticosteroids were administered more frequently to patients with recent-onset juvenile DM, compared to juvenile DM patients experiencing disease flare, who were more likely to receive cyclosporine. Compared to patients from Western and Eastern Europe, a higher proportion of patients from South and Central America and North America received pulse steroids, and the average steroid dosage was higher in the North American and South and Central American patients. The use of MTX was similar in all 4 regions, while cyclosporin A was more frequently used in Western Europe. In the &quot;as observed&quot; analysis, 57.9% of the patients with recent-onset juvenile DM and 36.4% of the patients experiencing disease flare (P&lt;0.001) reached at least a 70% response by PRINTO criteria at 6 months; these proportions had increased at month 24 to 78.4% and 51.2%, respectively (P&lt;0.001). Corresponding results of the ITT analysis were much lower, with only one-third of the patients able to maintain the initial assigned therapy over 24 months.&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSION: &lt;/b&gt;Patients with recent-onset juvenile DM are more likely to achieve significant clinical improvement over 24 months, when compared to patients experiencing flares of juvenile DM. Internationally, various therapeutic approaches are used to treat this disease.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">10</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/21647864?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Ruperto, Nicolino</style></author><author><style face="normal" font="default" size="100%">Lovell, Daniel J</style></author><author><style face="normal" font="default" size="100%">Li, Tracy</style></author><author><style face="normal" font="default" size="100%">Sztajnbok, Flavio</style></author><author><style face="normal" font="default" size="100%">Goldenstein-Schainberg, Claudia</style></author><author><style face="normal" font="default" size="100%">Scheinberg, Morton</style></author><author><style face="normal" font="default" size="100%">Penades, Inmaculada Calvo</style></author><author><style face="normal" font="default" size="100%">Fischbach, Michael</style></author><author><style face="normal" font="default" size="100%">Alcala, Javier Orozco</style></author><author><style face="normal" font="default" size="100%">Hashkes, Philip J</style></author><author><style face="normal" font="default" size="100%">Hom, Christine</style></author><author><style face="normal" font="default" size="100%">Jung, Lawrence</style></author><author><style face="normal" font="default" size="100%">Lepore, Loredana</style></author><author><style face="normal" font="default" size="100%">Oliveira, Sheila</style></author><author><style face="normal" font="default" size="100%">Wallace, Carol</style></author><author><style face="normal" font="default" size="100%">Alessio, Maria</style></author><author><style face="normal" font="default" size="100%">Quartier, Pierre</style></author><author><style face="normal" font="default" size="100%">Cortis, Elisabetta</style></author><author><style face="normal" font="default" size="100%">Eberhard, Anne</style></author><author><style face="normal" font="default" size="100%">Simonini, Gabriele</style></author><author><style face="normal" font="default" size="100%">Lemelle, Irene</style></author><author><style face="normal" font="default" size="100%">Chalom, Elizabeth Candell</style></author><author><style face="normal" font="default" size="100%">Sigal, Leonard H</style></author><author><style face="normal" font="default" size="100%">Block, Alan</style></author><author><style face="normal" font="default" size="100%">Covucci, Allison</style></author><author><style face="normal" font="default" size="100%">Nys, Marleen</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author><author><style face="normal" font="default" size="100%">Giannini, Edward H</style></author></authors><translated-authors><author><style face="normal" font="default" size="100%">Paediatric Rheumatology International Trials Organisation (PRINTO)</style></author><author><style face="normal" font="default" size="100%">Pediatric Rheumatology Collaborative Study Group (PRCSG)</style></author></translated-authors></contributors><titles><title><style face="normal" font="default" size="100%">Abatacept improves health-related quality of life, pain, sleep quality, and daily participation in subjects with juvenile idiopathic arthritis.</style></title><secondary-title><style face="normal" font="default" size="100%">Arthritis Care Res (Hoboken)</style></secondary-title><alt-title><style face="normal" font="default" size="100%">Arthritis Care Res (Hoboken)</style></alt-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Adolescent</style></keyword><keyword><style  face="normal" font="default" size="100%">Arthritis, Juvenile</style></keyword><keyword><style  face="normal" font="default" size="100%">Child</style></keyword><keyword><style  face="normal" font="default" size="100%">Double-Blind Method</style></keyword><keyword><style  face="normal" font="default" size="100%">Female</style></keyword><keyword><style  face="normal" font="default" size="100%">Health Status</style></keyword><keyword><style  face="normal" font="default" size="100%">Humans</style></keyword><keyword><style  face="normal" font="default" size="100%">Immunoconjugates</style></keyword><keyword><style  face="normal" font="default" size="100%">Male</style></keyword><keyword><style  face="normal" font="default" size="100%">Pain</style></keyword><keyword><style  face="normal" font="default" size="100%">Quality of Life</style></keyword><keyword><style  face="normal" font="default" size="100%">Questionnaires</style></keyword><keyword><style  face="normal" font="default" size="100%">Sleep Stages</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2010</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2010 Nov</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">62</style></volume><pages><style face="normal" font="default" size="100%">1542-51</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;OBJECTIVE: &lt;/b&gt;To assess health-related quality of life (HRQOL) in abatacept-treated children/adolescents with juvenile idiopathic arthritis (JIA).&lt;/p&gt;&lt;p&gt;&lt;b&gt;METHODS: &lt;/b&gt;In this phase III, double-blind, placebo-controlled trial, subjects with active polyarticular course JIA and an inadequate response/intolerance to ≥1 disease-modifying antirheumatic drug (including biologics) received abatacept 10 mg/kg plus methotrexate (MTX) during the 4-month open-label period (period A). Subjects achieving the American College of Rheumatology Pediatric 30 criteria for improvement (defined &quot;responders&quot;) were randomized to abatacept or placebo (plus MTX) in the 6-month double-blind withdrawal period (period B). HRQOL assessments included 15 Child Health Questionnaire (CHQ) health concepts plus the physical (PhS) and psychosocial summary scores (PsS), pain (100-mm visual analog scale), the Children's Sleep Habits Questionnaire, and a daily activity participation questionnaire.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;A total of 190 subjects from period A and 122 from period B were eligible for analysis. In period A, there were substantial improvements across all of the CHQ domains (greatest improvement was in pain/discomfort) and the PhS (8.3 units) and PsS (4.3 units) with abatacept. At the end of period B, abatacept-treated subjects had greater improvements versus placebo in all domains (except behavior) and both summary scores. Similar improvement patterns were seen with pain and sleep. For participation in daily activities, an additional 2.6 school days/month and 2.3 parents' usual activity days/month were gained in period A responders with abatacept, and further gains were made in period B (1.9 versus 0.9 [P = 0.033] and 0.2 versus -1.3 [P = 0.109] school days/month and parents' usual activity days/month, respectively, in abatacept- versus placebo-treated subjects).&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSION: &lt;/b&gt;Improvements in HRQOL were observed with abatacept, providing real-life tangible benefits to children with JIA and their parents/caregivers.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">11</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/20597110?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Ruperto, Nicolino</style></author><author><style face="normal" font="default" size="100%">Ozen, Seza</style></author><author><style face="normal" font="default" size="100%">Pistorio, Angela</style></author><author><style face="normal" font="default" size="100%">Dolezalova, Pavla</style></author><author><style face="normal" font="default" size="100%">Brogan, Paul</style></author><author><style face="normal" font="default" size="100%">Cabral, David A</style></author><author><style face="normal" font="default" size="100%">Cuttica, Ruben</style></author><author><style face="normal" font="default" size="100%">Khubchandani, Raju</style></author><author><style face="normal" font="default" size="100%">Lovell, Daniel J</style></author><author><style face="normal" font="default" size="100%">O'Neil, Kathleen M</style></author><author><style face="normal" font="default" size="100%">Quartier, Pierre</style></author><author><style face="normal" font="default" size="100%">Ravelli, Angelo</style></author><author><style face="normal" font="default" size="100%">Iusan, Silvia M</style></author><author><style face="normal" font="default" size="100%">Filocamo, Giovanni</style></author><author><style face="normal" font="default" size="100%">Magalhães, Claudia Saad</style></author><author><style face="normal" font="default" size="100%">Unsal, Erbil</style></author><author><style face="normal" font="default" size="100%">Oliveira, Sheila</style></author><author><style face="normal" font="default" size="100%">Bracaglia, Claudia</style></author><author><style face="normal" font="default" size="100%">Bagga, Arvind</style></author><author><style face="normal" font="default" size="100%">Stanevicha, Valda</style></author><author><style face="normal" font="default" size="100%">Manzoni, Silvia Magni</style></author><author><style face="normal" font="default" size="100%">Pratsidou, Polyxeni</style></author><author><style face="normal" font="default" size="100%">Lepore, Loredana</style></author><author><style face="normal" font="default" size="100%">Espada, Graciela</style></author><author><style face="normal" font="default" size="100%">Kone-Paut, Isabella</style></author><author><style face="normal" font="default" size="100%">Paut, Isabelle Kone</style></author><author><style face="normal" font="default" size="100%">Zulian, Francesco</style></author><author><style face="normal" font="default" size="100%">Barone, Patrizia</style></author><author><style face="normal" font="default" size="100%">Bircan, Zelal</style></author><author><style face="normal" font="default" size="100%">Maldonado, Maria del Rocio</style></author><author><style face="normal" font="default" size="100%">Russo, Ricardo</style></author><author><style face="normal" font="default" size="100%">Vilca, Iris</style></author><author><style face="normal" font="default" size="100%">Tullus, Kjell</style></author><author><style face="normal" font="default" size="100%">Cimaz, Rolando</style></author><author><style face="normal" font="default" size="100%">Horneff, Gerd</style></author><author><style face="normal" font="default" size="100%">Anton, Jordi</style></author><author><style face="normal" font="default" size="100%">Garay, Stella</style></author><author><style face="normal" font="default" size="100%">Nielsen, Susan</style></author><author><style face="normal" font="default" size="100%">Barbano, Giancarlo</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author></authors><translated-authors><author><style face="normal" font="default" size="100%">Paediatric Rheumatology International Trials Organisation (PRINTO)</style></author></translated-authors></contributors><titles><title><style face="normal" font="default" size="100%">EULAR/PRINTO/PRES criteria for Henoch-Schönlein purpura, childhood polyarteritis nodosa, childhood Wegener granulomatosis and childhood Takayasu arteritis: Ankara 2008. Part I: Overall methodology and clinical characterisation.</style></title><secondary-title><style face="normal" font="default" size="100%">Ann Rheum Dis</style></secondary-title><alt-title><style face="normal" font="default" size="100%">Ann. Rheum. Dis.</style></alt-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Adolescent</style></keyword><keyword><style  face="normal" font="default" size="100%">Biopsy</style></keyword><keyword><style  face="normal" font="default" size="100%">Child</style></keyword><keyword><style  face="normal" font="default" size="100%">Delphi Technique</style></keyword><keyword><style  face="normal" font="default" size="100%">Granulomatosis with Polyangiitis</style></keyword><keyword><style  face="normal" font="default" size="100%">Humans</style></keyword><keyword><style  face="normal" font="default" size="100%">International Cooperation</style></keyword><keyword><style  face="normal" font="default" size="100%">Internet</style></keyword><keyword><style  face="normal" font="default" size="100%">Polyarteritis Nodosa</style></keyword><keyword><style  face="normal" font="default" size="100%">Purpura, Schoenlein-Henoch</style></keyword><keyword><style  face="normal" font="default" size="100%">Reproducibility of Results</style></keyword><keyword><style  face="normal" font="default" size="100%">Takayasu Arteritis</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2010</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2010 May</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">69</style></volume><pages><style face="normal" font="default" size="100%">790-7</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;OBJECTIVES: &lt;/b&gt;To report methodology and overall clinical, laboratory and radiographic characteristics for Henoch-Schönlein purpura (HSP), childhood polyarteritis nodosa (c-PAN), c-Wegener granulomatosis (c-WG) and c-Takayasu arteritis (c-TA) classification criteria.&lt;/p&gt;&lt;p&gt;&lt;b&gt;METHODS: &lt;/b&gt;The preliminary Vienna 2005 consensus conference, which proposed preliminary criteria for paediatric vasculitides, was followed by a EULAR/PRINTO/PRES - supported validation project divided into three main steps. Step 1: retrospective/prospective web-data collection for HSP, c-PAN, c-WG and c-TA, with age at diagnosis &lt;or=18 years. Step 2: blinded classification by consensus panel of a subgroup of 280 cases (128 difficult cases, 152 randomly selected) enabling expert diagnostic verification. Step 3: Ankara 2008 Consensus Conference and statistical evaluation (sensitivity, specificity, area under the curve, kappa-agreement) using as 'gold standard' the final consensus classification or original treating physician diagnosis.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;A total of 1183/1398 (85%) samples collected were available for analysis: 827 HSP, 150 c-PAN, 60 c-WG, 87 c-TA and 59 c-other. Prevalence, signs/symptoms, laboratory, biopsy and imaging reports were consistent with the clinical picture of the four c-vasculitides. A representative subgroup of 280 patients was blinded to the treating physician diagnosis and classified by a consensus panel, with a kappa-agreement of 0.96 for HSP (95% CI 0.84 to 1), 0.88 for c-WG (95% CI 0.76 to 0.99), 0.84 for c-TA (95% CI 0.73 to 0.96) and 0.73 for c-PAN (95% CI 0.62 to 0.84), with an overall kappa of 0.79 (95% CI 0.73 to 0.84).&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSION: &lt;/b&gt;EULAR/PRINTO/PRES propose validated classification criteria for HSP, c-PAN, c-WG and c-TA, with substantial/almost perfect agreement with the final consensus classification or original treating physician diagnosis.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">5</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/20388738?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Lepore, Loredana</style></author><author><style face="normal" font="default" size="100%">Paloni, Giulia</style></author><author><style face="normal" font="default" size="100%">Caorsi, Roberta</style></author><author><style face="normal" font="default" size="100%">Alessio, Maria</style></author><author><style face="normal" font="default" size="100%">Rigante, Donato</style></author><author><style face="normal" font="default" size="100%">Ruperto, Nicola</style></author><author><style face="normal" font="default" size="100%">Cattalini, Marco</style></author><author><style face="normal" font="default" size="100%">Tommasini, Alberto</style></author><author><style face="normal" font="default" size="100%">Zulian, Francesco</style></author><author><style face="normal" font="default" size="100%">Ventura, Alessando</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author><author><style face="normal" font="default" size="100%">Gattorno, Marco</style></author></authors></contributors><titles><title><style face="normal" font="default" size="100%">Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with Anakinra.</style></title><secondary-title><style face="normal" font="default" size="100%">J Pediatr</style></secondary-title><alt-title><style face="normal" font="default" size="100%">J. Pediatr.</style></alt-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Adolescent</style></keyword><keyword><style  face="normal" font="default" size="100%">Adult</style></keyword><keyword><style  face="normal" font="default" size="100%">Case-Control Studies</style></keyword><keyword><style  face="normal" font="default" size="100%">Child</style></keyword><keyword><style  face="normal" font="default" size="100%">Child, Preschool</style></keyword><keyword><style  face="normal" font="default" size="100%">Cryopyrin-Associated Periodic Syndromes</style></keyword><keyword><style  face="normal" font="default" size="100%">Female</style></keyword><keyword><style  face="normal" font="default" size="100%">Follow-Up Studies</style></keyword><keyword><style  face="normal" font="default" size="100%">Humans</style></keyword><keyword><style  face="normal" font="default" size="100%">Inflammation</style></keyword><keyword><style  face="normal" font="default" size="100%">Interleukin 1 Receptor Antagonist Protein</style></keyword><keyword><style  face="normal" font="default" size="100%">Interleukin-1</style></keyword><keyword><style  face="normal" font="default" size="100%">Male</style></keyword><keyword><style  face="normal" font="default" size="100%">Phenotype</style></keyword><keyword><style  face="normal" font="default" size="100%">Quality of Health Care</style></keyword><keyword><style  face="normal" font="default" size="100%">Quality of Life</style></keyword><keyword><style  face="normal" font="default" size="100%">Questionnaires</style></keyword><keyword><style  face="normal" font="default" size="100%">Syndrome</style></keyword><keyword><style  face="normal" font="default" size="100%">Treatment Outcome</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2010</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2010 Aug</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">157</style></volume><pages><style face="normal" font="default" size="100%">310-315.e1</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;OBJECTIVE: &lt;/b&gt;To evaluate the quality of life and long-term follow-up of patients enrolled in the Italian registry of cryopyrin-associated periodic syndromes (CAPS).&lt;/p&gt;&lt;p&gt;&lt;b&gt;STUDY DESIGN: &lt;/b&gt;Since 2004, 20 patients with CAPS were enrolled in a common registry from different Italian Centers of Pediatric Rheumatology; 14 patients were treated with Anakinra in an open fashion. Both treated and untreated patients were routinely followed according to standard of care. The Child Health Questionnaire (CHQ-PF 50) was used to assess the health-related quality of life.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;The mean duration of follow-up was 37.5 months. In all treated patients, a complete and persistent control of the inflammatory manifestations was observed with no further progression of the disease. At enrollment in the registry, patients showed a poorer health-related quality of life than healthy children in both physical and the psychosocial summary scores. Treatment was associated with a dramatic and sustained amelioration of a variety of measures of poor quality of life, particularly in those concerning the global health perception, bodily pain-discomfort, and other physical domains.&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSIONS: &lt;/b&gt;Long-term IL-1 blockade produces a significant and persistent improvement in the clinical manifestations associated with the disease and on the overall quality of life.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">2</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/20472245?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Foell, Dirk</style></author><author><style face="normal" font="default" size="100%">Wulffraat, Nico</style></author><author><style face="normal" font="default" size="100%">Wedderburn, Lucy R</style></author><author><style face="normal" font="default" size="100%">Wittkowski, Helmut</style></author><author><style face="normal" font="default" size="100%">Frosch, Michael</style></author><author><style face="normal" font="default" size="100%">Gerss, Joachim</style></author><author><style face="normal" font="default" size="100%">Stanevicha, Valda</style></author><author><style face="normal" font="default" size="100%">Mihaylova, Dimitrina</style></author><author><style face="normal" font="default" size="100%">Ferriani, Virginia</style></author><author><style face="normal" font="default" size="100%">Tsakalidou, Florence Kanakoudi</style></author><author><style face="normal" font="default" size="100%">Foeldvari, Ivan</style></author><author><style face="normal" font="default" size="100%">Cuttica, Ruben</style></author><author><style face="normal" font="default" size="100%">Gonzalez, Benito</style></author><author><style face="normal" font="default" size="100%">Ravelli, Angelo</style></author><author><style face="normal" font="default" size="100%">Khubchandani, Raju</style></author><author><style face="normal" font="default" size="100%">Oliveira, Sheila</style></author><author><style face="normal" font="default" size="100%">Armbrust, Wineke</style></author><author><style face="normal" font="default" size="100%">Garay, Stella</style></author><author><style face="normal" font="default" size="100%">Vojinovic, Jelena</style></author><author><style face="normal" font="default" size="100%">Norambuena, Ximena</style></author><author><style face="normal" font="default" size="100%">Gamir, María Luz</style></author><author><style face="normal" font="default" size="100%">García-Consuegra, Julia</style></author><author><style face="normal" font="default" size="100%">Lepore, Loredana</style></author><author><style face="normal" font="default" size="100%">Susic, Gordana</style></author><author><style face="normal" font="default" size="100%">Corona, Fabrizia</style></author><author><style face="normal" font="default" size="100%">Dolezalova, Pavla</style></author><author><style face="normal" font="default" size="100%">Pistorio, Angela</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author><author><style face="normal" font="default" size="100%">Ruperto, Nicolino</style></author><author><style face="normal" font="default" size="100%">Roth, Johannes</style></author></authors><translated-authors><author><style face="normal" font="default" size="100%">Paediatric Rheumatology International Trials Organization (PRINTO)</style></author></translated-authors></contributors><titles><title><style face="normal" font="default" size="100%">Methotrexate withdrawal at 6 vs 12 months in juvenile idiopathic arthritis in remission: a randomized clinical trial.</style></title><secondary-title><style face="normal" font="default" size="100%">JAMA</style></secondary-title><alt-title><style face="normal" font="default" size="100%">JAMA</style></alt-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Adolescent</style></keyword><keyword><style  face="normal" font="default" size="100%">Antirheumatic Agents</style></keyword><keyword><style  face="normal" font="default" size="100%">Arthritis, Juvenile</style></keyword><keyword><style  face="normal" font="default" size="100%">ATP-Binding Cassette Transporters</style></keyword><keyword><style  face="normal" font="default" size="100%">Calgranulin B</style></keyword><keyword><style  face="normal" font="default" size="100%">Child</style></keyword><keyword><style  face="normal" font="default" size="100%">Child, Preschool</style></keyword><keyword><style  face="normal" font="default" size="100%">Female</style></keyword><keyword><style  face="normal" font="default" size="100%">Humans</style></keyword><keyword><style  face="normal" font="default" size="100%">Infant</style></keyword><keyword><style  face="normal" font="default" size="100%">Male</style></keyword><keyword><style  face="normal" font="default" size="100%">Methotrexate</style></keyword><keyword><style  face="normal" font="default" size="100%">Predictive Value of Tests</style></keyword><keyword><style  face="normal" font="default" size="100%">Prospective Studies</style></keyword><keyword><style  face="normal" font="default" size="100%">Recurrence</style></keyword><keyword><style  face="normal" font="default" size="100%">Remission Induction</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2010</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2010 Apr 7</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">303</style></volume><pages><style face="normal" font="default" size="100%">1266-73</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;CONTEXT: &lt;/b&gt;Novel therapies have improved the remission rate in chronic inflammatory disorders including juvenile idiopathic arthritis (JIA). Therefore, strategies of tapering therapy and reliable parameters for detecting subclinical inflammation have now become challenging questions.&lt;/p&gt;&lt;p&gt;&lt;b&gt;OBJECTIVES: &lt;/b&gt;To analyze whether longer methotrexate treatment during remission of JIA prevents flares after withdrawal of medication and whether specific biomarkers identify patients at risk for flares.&lt;/p&gt;&lt;p&gt;&lt;b&gt;DESIGN, SETTING, AND PATIENTS: &lt;/b&gt;Prospective, open, multicenter, medication-withdrawal randomized clinical trial including 364 patients (median age, 11.0 years) with JIA recruited in 61 centers from 29 countries between February 2005 and June 2006. Patients were included at first confirmation of clinical remission while continuing medication. At the time of therapy withdrawal, levels of the phagocyte activation marker myeloid-related proteins 8 and 14 heterocomplex (MRP8/14) were determined.&lt;/p&gt;&lt;p&gt;&lt;b&gt;INTERVENTION: &lt;/b&gt;Patients were randomly assigned to continue with methotrexate therapy for either 6 months (group 1 [n = 183]) or 12 months (group 2 [n = 181]) after induction of disease remission.&lt;/p&gt;&lt;p&gt;&lt;b&gt;MAIN OUTCOME MEASURES: &lt;/b&gt;Primary outcome was relapse rate in the 2 treatment groups; secondary outcome was time to relapse. In a prespecified cohort analysis, the prognostic accuracy of MRP8/14 concentrations for the risk of flares was assessed.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;Intention-to-treat analysis of the primary outcome revealed relapse within 24 months after the inclusion into the study in 98 of 183 patients (relapse rate, 56.7%) in group 1 and 94 of 181 (55.6%) in group 2. The odds ratio for group 1 vs group 2 was 1.02 (95% CI, 0.82-1.27; P = .86). The median relapse-free interval after inclusion was 21.0 months in group 1 and 23.0 months in group 2. The hazard ratio for group 1 vs group 2 was 1.07 (95% CI, 0.82-1.41; P = .61). Median follow-up duration after inclusion was 34.2 and 34.3 months in groups 1 and 2, respectively. Levels of MRP8/14 during remission were significantly higher in patients who subsequently developed flares (median, 715 [IQR, 320-1 110] ng/mL) compared with patients maintaining stable remission (400 [IQR, 220-800] ng/mL; P = .003). Low MRP8/14 levels indicated a low risk of flares within the next 3 months following the biomarker test (area under the receiver operating characteristic curve, 0.76; 95% CI, 0.62-0.90).&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSIONS: &lt;/b&gt;In patients with JIA in remission, a 12-month vs 6-month withdrawal of methotrexate did not reduce the relapse rate. Higher MRP8/14 concentrations were associated with risk of relapse after discontinuing methotrexate.&lt;/p&gt;&lt;p&gt;&lt;b&gt;TRIAL REGISTRATION: &lt;/b&gt;isrctn.org Identifier: ISRCTN18186313.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">13</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/20371785?dopt=Abstract</style></custom1></record><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Vilca, Iris</style></author><author><style face="normal" font="default" size="100%">Munitis, Pablo Garcia</style></author><author><style face="normal" font="default" size="100%">Pistorio, Angela</style></author><author><style face="normal" font="default" size="100%">Ravelli, Angelo</style></author><author><style face="normal" font="default" size="100%">Buoncompagni, Antonella</style></author><author><style face="normal" font="default" size="100%">Bica, Blanca</style></author><author><style face="normal" font="default" size="100%">Campos, Lucia</style></author><author><style face="normal" font="default" size="100%">Häfner, Renate</style></author><author><style face="normal" font="default" size="100%">Hofer, Michael</style></author><author><style face="normal" font="default" size="100%">Ozen, Seza</style></author><author><style face="normal" font="default" size="100%">Huemer, Christian</style></author><author><style face="normal" font="default" size="100%">Bae, Sang Cheol</style></author><author><style face="normal" font="default" size="100%">Sztajnbok, Flavio</style></author><author><style face="normal" font="default" size="100%">Arguedas, Olga</style></author><author><style face="normal" font="default" size="100%">Foeldvari, Ivan</style></author><author><style face="normal" font="default" size="100%">Huppertz, Hans Iko</style></author><author><style face="normal" font="default" size="100%">Gamir, María Luz</style></author><author><style face="normal" font="default" size="100%">Magnusson, Bo</style></author><author><style face="normal" font="default" size="100%">Dressler, Frank</style></author><author><style face="normal" font="default" size="100%">Uziel, Yosef</style></author><author><style face="normal" font="default" size="100%">van Rossum, Marion A J</style></author><author><style face="normal" font="default" size="100%">Hollingworth, Peter</style></author><author><style face="normal" font="default" size="100%">Cawkwell, Gail</style></author><author><style face="normal" font="default" size="100%">Martini, Alberto</style></author><author><style face="normal" font="default" size="100%">Ruperto, Nicolino</style></author></authors><translated-authors><author><style face="normal" font="default" size="100%">Pediatric Rheumatology International Trials Organisation (PRINTO)</style></author></translated-authors></contributors><titles><title><style face="normal" font="default" size="100%">Predictors of poor response to methotrexate in polyarticular-course juvenile idiopathic arthritis: analysis of the PRINTO methotrexate trial.</style></title><secondary-title><style face="normal" font="default" size="100%">Ann Rheum Dis</style></secondary-title><alt-title><style face="normal" font="default" size="100%">Ann. Rheum. Dis.</style></alt-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Adolescent</style></keyword><keyword><style  face="normal" font="default" size="100%">Antibodies, Antinuclear</style></keyword><keyword><style  face="normal" font="default" size="100%">Antirheumatic Agents</style></keyword><keyword><style  face="normal" font="default" size="100%">Arthritis, Juvenile</style></keyword><keyword><style  face="normal" font="default" size="100%">Child</style></keyword><keyword><style  face="normal" font="default" size="100%">Child, Preschool</style></keyword><keyword><style  face="normal" font="default" size="100%">Disability Evaluation</style></keyword><keyword><style  face="normal" font="default" size="100%">Female</style></keyword><keyword><style  face="normal" font="default" size="100%">Follow-Up Studies</style></keyword><keyword><style  face="normal" font="default" size="100%">Humans</style></keyword><keyword><style  face="normal" font="default" size="100%">Immunosuppressive Agents</style></keyword><keyword><style  face="normal" font="default" size="100%">Male</style></keyword><keyword><style  face="normal" font="default" size="100%">Methotrexate</style></keyword><keyword><style  face="normal" font="default" size="100%">Prognosis</style></keyword><keyword><style  face="normal" font="default" size="100%">Treatment Outcome</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2010</style></year><pub-dates><date><style  face="normal" font="default" size="100%">2010 Aug</style></date></pub-dates></dates><volume><style face="normal" font="default" size="100%">69</style></volume><pages><style face="normal" font="default" size="100%">1479-83</style></pages><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;&lt;b&gt;OBJECTIVES: &lt;/b&gt;To determine whether baseline demographic, clinical, articular and laboratory variables predict methotrexate (MTX) poor response in polyarticular-course juvenile idiopathic arthritis.&lt;/p&gt;&lt;p&gt;&lt;b&gt;METHODS: &lt;/b&gt;Patients newly treated for 6 months with MTX enrolled in the Paediatric Rheumatology International Trials Organization (PRINTO) MTX trial. Bivariate and logistic regression analyses were used to identify baseline predictors of poor response according to the American College of Rheumatology pediatric (ACR-ped) 30 and 70 criteria.&lt;/p&gt;&lt;p&gt;&lt;b&gt;RESULTS: &lt;/b&gt;In all, 405/563 (71.9%) of patients were women; median age at onset and disease duration were 4.3 and 1.4 years, respectively, with anti-nuclear antibody (ANA) detected in 259/537 (48.2%) patients. With multivariate logistic regression analysis, the most important determinants of ACR-ped 70 non-responders were: disease duration &gt; 1.3 years (OR 1.93), ANA negativity (OR 1.77), Childhood Health Assessment Questionnaire (CHAQ) disability index &gt; 1.125 (OR 1.65) and the presence of right and left wrist activity (OR 1.55). Predictors of ACR-ped 30 non-responders were: ANA negativity (OR 1.92), CHAQ disability index &gt; 1.14 (OR 2.18) and a parent's evaluation of child's overall well-being &lt; or = 4.69 (OR 2.2).&lt;/p&gt;&lt;p&gt;&lt;b&gt;CONCLUSION: &lt;/b&gt;The subgroup of patients with longer disease duration, ANA negativity, higher disability and presence of wrist activity were significantly associated with a poorer response to a 6-month MTX course.&lt;/p&gt;</style></abstract><issue><style face="normal" font="default" size="100%">8</style></issue><custom1><style face="normal" font="default" size="100%">http://www.ncbi.nlm.nih.gov/pubmed/20525842?dopt=Abstract</style></custom1></record></records></xml>