• Athimalaipet V Ramanan, Andrew D Dick, Ashley P Jones, Andrew McKay, Paula R Williamson, Sandrine Compeyrot-Lacassagne, Ben Hardwick, Helen Hickey, Dyfrig Hughes, Patricia Woo, Diana Benton, Clive Edelsten, Michael W Beresford, and SYCAMORE Study Group.
• From University Hospitals Bristol NHS Foundation Trust (A.V.R., A.D.D., D.B.) and the School of Clinical Sciences, University of Bristol (A.V.R., A.D.D.), Bristol, National Institute for Health Research (NIHR) Biomedical Research Centre at Moorfields Eye Hospital and University College London Institute of Ophthalmology (A.D.D., P.W.) and Great Ormond Street Hospital (S.C.-L., C.E.), London, Institute of Translational Medicine, University of Liverpool (A.P.J., A.M., P.R.W., B.H., H.H., M.W.B.), and the Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust (M.W.B.), Liverpool, and Bangor University, Bangor (D.H.) - all in the United Kingdom.
• N. Engl. J. Med. 2017 Apr 27; 376 (17): 1637-1646.

BackgroundAdalimumab, a fully human anti-tumor necrosis factor α monoclonal antibody, is effective in the treatment of juvenile idiopathic arthritis (JIA). We tested the efficacy of adalimumab in the treatment of JIA-associated uveitis.MethodsIn this multicenter, double-blind, randomized, placebo-controlled trial, we assessed the efficacy and safety of adalimumab in children and adolescents 2 years of age or older who had active JIA-associated uveitis. Patients who were taking a stable dose of methotrexate were randomly assigned in a 2:1 ratio to receive either adalimumab (at a dose of 20 mg or 40 mg, according to body weight) or placebo, administered subcutaneously every 2 weeks. Patients continued the trial regimen until treatment failure or until 18 months had elapsed. They were followed for up to 2 years after randomization. The primary end point was the time to treatment failure, defined according to a multicomponent intraocular inflammation score that was based on the Standardization of Uveitis Nomenclature criteria.ResultsThe prespecified stopping criteria were met after the enrollment of 90 of 114 patients. We observed 16 treatment failures in 60 patients (27%) in the adalimumab group versus 18 treatment failures in 30 patients (60%) in the placebo group (hazard ratio, 0.25; 95% confidence interval [CI], 0.12 to 0.49; P<0.0001 [the prespecified stopping boundary]). Adverse events were reported more frequently in patients receiving adalimumab than in those receiving placebo (10.07 events per patient-year [95% CI, 9.26 to 10.89] vs. 6.51 events per patient-year [95% CI, 5.26 to 7.77]), as were serious adverse events (0.29 events per patient-year [95% CI, 0.15 to 0.43] vs. 0.19 events per patient-year [95% CI, 0.00 to 0.40]).ConclusionsAdalimumab therapy controlled inflammation and was associated with a lower rate of treatment failure than placebo among children and adolescents with active JIA-associated uveitis who were taking a stable dose of methotrexate. Patients who received adalimumab had a much higher incidence of adverse events and serious adverse events than those who received placebo. (Funded by the NIHR Health Technology Assessment Programme and Arthritis Research UK; SYCAMORE EudraCT number, 2010-021141-41 .).

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