Current evidence of methotrexate efficacy in childhood chronic uveitis: a systematic review and meta-analysis approach

Rheumatology (Oxford). 2013 May;52(5):825-31. doi: 10.1093/rheumatology/kes186. Epub 2012 Aug 9.


Objective: To summarize evidence regarding the effectiveness of MTX in the treatment of childhood autoimmune chronic uveitis (ACU).

Methods: A systematic search of articles between January 1990 and June 2011 was conducted using EMBASE, Ovid MEDLINE, Evidence-Based Medicine Reviews-ACP Journal Club, the Cochrane Library and EBM Reviews. Studies investigating the efficacy of MTX as a single immunosuppressant medication in the treatment of ACU refractory to therapy with topical treatment and/or systemic treatment in children (≤16 years) were eligible for inclusion. The primary outcome measure was the improvement of intraocular inflammation, expressed as Tyndall, as defined by the Standardization of Uveitis Nomenclature working group criteria. The effect measure for each study was the proportion of people classified as responders. We determined a combined estimate of the proportion of children in the eligible studies responding to MTX.

Results: The initial search identified 246 articles of which 52 were potentially eligible. Nine eligible articles, all retrospective chart reviews, remained in the analysis. The number of children in studies ranged from 3 to 25, and the dose of MTX varied from 7.5 to 30 mg/m2. Altogether, 95 of 135 children responded to MTX. The pooled analysis suggested that MTX has a favourable effect in the improvement of intraocular inflammation: the proportion of responding subjects was 0.73 (95% CI 0.66, 0.81).

Conclusion: Although randomized controlled trials are needed, the available evidence supports the use of MTX in the treatment of childhood ACU: approximately three-quarters of patients on MTX can expect improvement in intraocular inflammation.

Publication types

  • Comparative Study
  • Meta-Analysis
  • Review
  • Systematic Review

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Chronic Disease
  • Cohort Studies
  • Dose-Response Relationship, Drug
  • Drug Administration Schedule
  • Evidence-Based Medicine
  • Female
  • Follow-Up Studies
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Methotrexate / therapeutic use*
  • Retrospective Studies
  • Risk Assessment
  • Severity of Illness Index
  • Treatment Outcome
  • Uveitis / diagnosis*
  • Uveitis / drug therapy*
  • Uveitis / immunology


  • Immunosuppressive Agents
  • Methotrexate