Pediatric Rheumatology Online Journal → June 2003 → Methotrexate→ Abstract #48

METHOTREXATE IN JCA: DEFINING ITS EFFICACY. A SYSTEMATIC REVIEW

V. W. Cartwright, F. M. Wolf

¹Pediatric Rheumatology, Children's Hospital, Seattle, WA; ²Medical Education and Biomedical Informatics, University of Washington, Seattle, WA

Background:Less than one-half of Juvenile Chronic Arthritis (JCA) patients have control of their disease with oral medications such as non-steroidal anti-inflammatory drugs. Other medications are needed to treat JCA and prevent its potential long-term complications.
Objective: To estimate the therapeutic and adverse effects of methotrexate (MTX) in treatment of JCA.
Methods:Systematic review of randomized controlled trials (RCTs) and retrospective studies of children taking MTX for JCA using MEDLINE search [1966-NOV 2002> with subject headings METHOTREXATE, ARTHRITIS, and CHILD, supplemented by a manual search of references. Data on demographics, MTX dose, markers of clinical improvement, and side effects were extracted and reviewed twice to minimize errors.
Results:  Twenty studies fulfilled criteria for inclusion. One systematic review, two randomized placebo-controlled trials, and 17 retrospective cohort studies detailed the efficacy and safety profile of MTX to treat JCA. The two RCTs combined had an odds ratio of 2.66 (95% CI 1.54-4.58) for physician global assessment of disease activity showing a higher response in the MTX groups. Summarizing the retrospective studies provided an average 57% response rate, and a mean 45%complete response[rdquo> rate (95% CI 53-61% and 36-54% respectively) to MTX therapy. Adverse events in the retrospective studies occurred in 33% of the patients (95% CI 29-37%). Reassuringly, the combined effects in the 2 RCTs had an odds ratio of 1.68 (95% CI .63-4.44) portraying no difference between placebo and MTX groups for side effects.
Conclusion:MTX is effective and safe to use in the majority of JCA patients. The hypothesis of MTX efficacy from observational studies was supported by the results of 2 RCTs and a systematic pooling of these two trials data. Continued improvement of clinical research to include patient-centered outcomes, standardized reporting of criteria, more well designed RCTs, and use of similar demographics to categorize patients will help directly apply information in future clinical studies of therapeutics into daily practice.