Literature DB >> 23252525

Randomized trial of tocilizumab in systemic juvenile idiopathic arthritis.

Fabrizio De Benedetti1, Hermine I Brunner, Nicolino Ruperto, Andrew Kenwright, Stephen Wright, Inmaculada Calvo, Ruben Cuttica, Angelo Ravelli, Rayfel Schneider, Patricia Woo, Carine Wouters, Ricardo Xavier, Lawrence Zemel, Eileen Baildam, Ruben Burgos-Vargas, Pavla Dolezalova, Stella M Garay, Rosa Merino, Rik Joos, Alexei Grom, Nico Wulffraat, Zbigniew Zuber, Francesco Zulian, Daniel Lovell, Alberto Martini.   

Abstract

BACKGROUND: Systemic juvenile idiopathic arthritis (JIA) is the most severe subtype of JIA; treatment options are limited. Interleukin-6 plays a pathogenic role in systemic JIA.
METHODS: We randomly assigned 112 children, 2 to 17 years of age, with active systemic JIA (duration of ≥6 months and inadequate responses to nonsteroidal antiinflammatory drugs and glucocorticoids) to the anti-interleukin-6 receptor antibody tocilizumab (at a dose of 8 mg per kilogram of body weight if the weight was ≥30 kg or 12 mg per kilogram if the weight was <30 kg) or placebo given intravenously every 2 weeks during the 12-week, double-blind phase. Patients meeting the predefined criteria for nonresponse were offered open-label tocilizumab. All patients could enter an open-label extension.
RESULTS: At week 12, the primary end point (an absence of fever and an improvement of 30% or more on at least three of the six variables in the American College of Rheumatology [ACR] core set for JIA, with no more than one variable worsening by more than 30%) was met in significantly more patients in the tocilizumab group than in the placebo group (64 of 75 [85%] vs. 9 of 37 [24%], P<0.001). At week 52, 80% of the patients who received tocilizumab had at least 70% improvement with no fever, including 59% who had 90% improvement; in addition, 48% of the patients had no joints with active arthritis, and 52% had discontinued oral glucocorticoids. In the double-blind phase, 159 adverse events, including 60 infections (2 serious), occurred in the tocilizumab group, as compared with 38, including 15 infections, in the placebo group. In the double-blind and extension periods combined, 39 serious adverse events (0.25 per patient-year), including 18 serious infections (0.11 per patient-year), occurred in patients who received tocilizumab. Neutropenia developed in 19 patients (17 patients with grade 3 and 2 patients with grade 4), and 21 had aminotransferase levels that were more than 2.5 times the upper limit of the normal range.
CONCLUSIONS: Tocilizumab was efficacious in severe, persistent systemic JIA. Adverse events were common and included infection, neutropenia, and increased aminotransferase levels. (Funded by Hoffmann-La Roche; ClinicalTrials.gov number, NCT00642460.).

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Year:  2012        PMID: 23252525     DOI: 10.1056/NEJMoa1112802

Source DB:  PubMed          Journal:  N Engl J Med        ISSN: 0028-4793            Impact factor:   91.245


  236 in total

Review 1.  Efficacy and safety of biological agents for systemic juvenile idiopathic arthritis: a systematic review and meta-analysis of randomized trials.

Authors:  Simon Tarp; Gil Amarilyo; Ivan Foeldvari; Robin Christensen; Jennifer M P Woo; Neta Cohen; Tracy D Pope; Daniel E Furst
Journal:  Rheumatology (Oxford)       Date:  2015-11-30       Impact factor: 7.580

2.  Cyclosporine in Resistant Systemic Arthritis - A Cheaper Alternative to Biologics.

Authors:  Priyankar Pal; Prabhas Prasun Giri; Rajiv Sinha
Journal:  Indian J Pediatr       Date:  2019-04-01       Impact factor: 1.967

Review 3.  Understanding Human Autoimmunity and Autoinflammation Through Transcriptomics.

Authors:  Romain Banchereau; Alma-Martina Cepika; Jacques Banchereau; Virginia Pascual
Journal:  Annu Rev Immunol       Date:  2017-01-30       Impact factor: 28.527

4.  A Bullseye for Children With Systemic Juvenile Idiopathic Arthritis.

Authors:  Lauren A Henderson
Journal:  Arthritis Rheumatol       Date:  2019-05-27       Impact factor: 10.995

Review 5.  Biologics in juvenile idiopathic arthritis: a narrative review.

Authors:  Federica Vanoni; Francesca Minoia; Clara Malattia
Journal:  Eur J Pediatr       Date:  2017-07-20       Impact factor: 3.183

Review 6.  Biologic-associated infections in pediatric rheumatology.

Authors:  Gerd Horneff
Journal:  Curr Rheumatol Rep       Date:  2015-11       Impact factor: 4.592

7.  Bayesian comparative effectiveness study of four consensus treatment plans for initial management of systemic juvenile idiopathic arthritis: FiRst-Line Options for Systemic juvenile idiopathic arthritis Treatment (FROST).

Authors:  Peter A Nigrovic; Timothy Beukelman; George Tomlinson; Brian M Feldman; Laura E Schanberg; Yukiko Kimura
Journal:  Clin Trials       Date:  2018-03-15       Impact factor: 2.486

8.  Systemic Juvenile Idiopathic Arthritis-Associated Lung Disease: Characterization and Risk Factors.

Authors:  Grant S Schulert; Shima Yasin; Brenna Carey; Claudia Chalk; Thuy Do; Andrew H Schapiro; Ammar Husami; Allen Watts; Hermine I Brunner; Jennifer Huggins; Elizabeth D Mellins; Esi M Morgan; Tracy Ting; Bruce C Trapnell; Kathryn A Wikenheiser-Brokamp; Christopher Towe; Alexei A Grom
Journal:  Arthritis Rheumatol       Date:  2019-10-01       Impact factor: 10.995

9.  2013 update of the 2011 American College of Rheumatology recommendations for the treatment of juvenile idiopathic arthritis: recommendations for the medical therapy of children with systemic juvenile idiopathic arthritis and tuberculosis screening among children receiving biologic medications.

Authors:  Sarah Ringold; Pamela F Weiss; Timothy Beukelman; Esi Morgan DeWitt; Norman T Ilowite; Yukiko Kimura; Ronald M Laxer; Daniel J Lovell; Peter A Nigrovic; Angela Byun Robinson; Richard K Vehe
Journal:  Arthritis Rheum       Date:  2013-10

Review 10.  Co-occurrence of Kikuchi-Fujimoto's disease and Still's disease: case report and review of previously reported cases.

Authors:  Karen A Toribio; Hideko Kamino; Stephanie Hu; Miriam Pomeranz; Michael H Pillinger
Journal:  Clin Rheumatol       Date:  2014-08-08       Impact factor: 2.980

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