☆ 4.5 Article

Networking in paediatrics: the example of the Paediatric Rheumatology International Trials Organisation (PRINTO)

ARCHIVES OF DISEASE IN CHILDHOOD (2011)

期刊

ARCHIVES OF DISEASE IN CHILDHOOD

卷 96, 期 6, 页码 596-601

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BMJ PUBLISHING GROUP

DOI: 10.1136/adc.2010.188946

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Pediatrics

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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.

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Mycophenolate Mofetil Versus Cyclophosphamide for Remission Induction in Childhood Polyarteritis Nodosa: An Open-Label, Randomized, Bayesian Noninferiority Trial

Paul A. Brogan, Barbara Arch, Helen Hickey, Jordi Anton, Este Iglesias, Eileen Baildam, Kamran Mahmood, Gavin Cleary, Elena Moraitis, Charalampia Papadopoulou, Michael W. Beresford, Phil Riley, Selcan Demir, Seza Ozen, Giovanna Culeddu, Dyfrig A. Hughes, Pavla Dolezalova, Lisa Hampson, John Whitehead, David Jayne, Nicola Ruperto, Despina Eleftheriou

Summary: The study showed that the effectiveness of CYC and MMF treatment for remission induction in childhood PAN is similar, but MMF may be associated with better health-related quality of life. Patients in the MMF group achieved the primary endpoint in a shorter time, with no relapses occurring in either group within 18 months.

ARTHRITIS & RHEUMATOLOGY (2021)

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Editorial Material Allergy

Persistence of disease flares is associated with an inadequate colchicine dose in familial Mediterranean fever: A national multicenter longitudinal study

Marta Bustaffa, Francesca Mazza, Diana Sutera, Maria Domenica Carrabba, Maria Alessio, Luca Cantarini, Laura Obici, Donato Rigante, Maria Cristina Maggio, Antonella Insalaco, Gabriele Simonini, Marco Cattalini, Giovanni Conti, Alma Nunzia Olivieri, Patrizia Barone, Angela Miniaci, Valentina Moressa, Maria Greca Magnolia, Luciana Breda, Davide Montin, Alessandra Spagnolo, Giovanna Fabio, Francesca Orlando, Carla Gaggiano, Roberta Mussinelli, Giovanna Capozio, Camilla Celani, Edoardo Marrani, Francesca Ricci, Francesco Calzatini, Maddalena Lancieri, Nicolino Ruperto, Marco Gattorno, Romina Gallizzi

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE (2021)

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Article Pediatrics

Improving clinical paediatric research and learning from COVID-19: recommendations by the Conect4Children expert advice group

Athimalaipet Ramanan, Neena Modi, Saskia N. de Wildt

Summary: The COVID-19 pandemic has stimulated new approaches in pediatric clinical research, which Conect4children recommends retaining for future use to improve efficiency, reduce costs, and promote research equity.

PEDIATRIC RESEARCH (2022)

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Editorial Material Rheumatology

To Randomize, or Not to Randomize, That is the Question

Nicolino Ruperto, Alberto Martini, Angela Pistorio

ARTHRITIS & RHEUMATOLOGY (2021)

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Article Rheumatology

Efficacy and safety of belimumab in paediatric and adult patients with systemic lupus erythematosus: an across-study comparison

Hermine Brunner, Carlos Abud-Mendoza, Masaaki Mori, Clarissa A. Pilkington, Reema Syed, Syuji Takei, Diego O. Viola, Richard A. Furie, Sandra Navarra, Fengchun Zhang, Damon L. Bass, Gina Eriksson, Anne E. Hammer, Beulah N. Ji, Mohamed Okily, David A. Roth, Holly Quasny, Nicolino Ruperto

Summary: The efficacy and safety of belimumab in treating pediatric and adult patients with systemic lupus erythematosus were found to be consistent in various studies. Patients treated with belimumab showed higher response rates and reduced risk of severe flare compared to those on placebo.

RMD OPEN (2021)

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Article Medicine, General & Internal

Tofacitinib in juvenile idiopathic arthritis: a double-blind, placebo-controlled, withdrawal phase 3 randomised trial

Nicolino Ruperto, Hermine Brunner, Olga Synoverska, Tracy Ting, Carlos Abud Mendoza, Alberto Spindler, Yulia Vyzhga, Katherine Marzan, Lyudmila Grebenkina, Irit Tirosh, Lisa Imundo, Rita Jerath, Daniel J. Kingsbury, Betul Sozeri, Sheetal S. Vora, Sampath Prahalad, Elena Zholobova, Yonatan Butbul Aviel, Vyacheslav Chasnyk, Melissa Lerman, Kabita Nanda, Heinrike Schmeling, Heather Tory, Yosef Uziel, Diego O. Viola, Holly B. Posner, Keith S. Kanik, Ann Wouters, Cheng Chang, Richard Zhang, Irina Lazariciu, Ming-Ann Hsu, Ricardo M. Suehiro, Alberto Martini, Daniel J. Lovell

Summary: The trial showed that tofacitinib is an effective treatment for patients with polyarticular course JIA. New oral therapies are particularly relevant for children and adolescents, who might prefer to avoid injections.

LANCET (2021)

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Article Rheumatology

The impact of the Eurofever criteria and the new InFevers MEFV classification in real life: Results from a large international FMF cohort

Marta Bustaffa, Isabelle Kone-Paut, Seza Ozen, Gayane Amaryan, Efimia Papadopoulou-Alataki, Romina Gallizzi, Maria Carrabba, Yonatan Butbul Aviel, Luca Cantarini, Maria Alessio, Jordi Anton, Laura Obici, Faysal Gok, Ezgi Deniz Batu, Estefania Moreno, Paul Brogan, Maria Trachana, Gabriele Simonini, Donato Rigante, Yosef Uziel, Antonella Insalaco, Maria Cristina Maggio, Nicolino Ruperto, Marco Gattorno, L. Rossi Semerano

Summary: The new EPCC and INSAID classification of MEFV variants can identify two distinct groups of FMF patients, which differ in clinical characteristics, therapeutic approach, and response to treatment.

SEMINARS IN ARTHRITIS AND RHEUMATISM (2022)

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Article Rheumatology

Definition and Validation of the American College of Rheumatology 2021 Juvenile Arthritis Disease Activity Score Cutoffs for Disease Activity States in Juvenile Idiopathic Arthritis

Chiara Trincianti, Evert Hendrik Pieter Van Dijkhuizen, Alessandra Alongi, Marta Mazzoni, Joost F. Swart, Irina Nikishina, Pekka Lahdenne, Lidia Rutkowska-Sak, Tadej Avcin, Pierre Quartier, Violeta Panaviene, Yosef Uziel, Chris Pruunsild, Veronika Vargova, Soamarat Vilaiyuk, Pavla Dolezalova, Sarah Ringold, Marco Garrone, Nicolino Ruperto, Angelo Ravelli, Alessandro Consolaro

Summary: This study developed and validated new Juvenile Arthritis Disease Activity Score 10 (JADAS10) and clinical JADAS10 (cJADAS10) cutoffs to distinguish different disease activity states in children. Results showed that the cutoffs had good metrologic properties and are suitable for use in clinical trials and routine practice.

ARTHRITIS & RHEUMATOLOGY (2021)

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