Related Experiment Videos
A randomized, controlled trial of methotrexate versus placebo in early diffuse scleroderma
J E Pope1, N Bellamy, J R Seibold
1University of Western Ontario, London, Canada.
Arthritis and Rheumatism
|June 16, 2001
Summary
Methotrexate (MTX) showed a trend toward improving skin scores in early diffuse systemic sclerosis (SSc). However, this study did not find statistically significant evidence of MTX efficacy for treating early diffuse SSc.
Area of Science:
- Rheumatology
- Immunology
- Dermatology
Background:
- Early diffuse systemic sclerosis (SSc) lacks proven treatments.
- Methotrexate (MTX) is a potential therapeutic option for SSc.
Purpose of the Study:
- To evaluate the efficacy of methotrexate (MTX) in improving skin and other disease parameters in patients with early diffuse SSc.
- To assess MTX's impact on skin scores and physician global assessment.
Main Methods:
- A multicenter, randomized, double-blind, placebo-controlled trial involving 71 patients with early diffuse SSc (duration <3 years).
- Patients received either MTX (n=35) or placebo (n=36) for 12 months.
- Primary outcomes included modified Rodnan skin score, UCLA skin score, and physician global assessment.
Main Results:
- At 12 months, MTX showed a favorable trend in improving skin scores (modified Rodnan and UCLA) compared to placebo (P < 0.009 and P < 0.02, respectively).
- Physician global assessment favored MTX, but patient global assessment and carbon monoxide diffusing capacity (DLco) did not show significant differences.
- Statistical significance for skin score improvements was not maintained after adjusting for baseline differences in sex and steroid use.
Conclusions:
- The trial demonstrated a trend favoring MTX over placebo for early diffuse SSc, but between-group differences were small.
- The study had limited power (50%) to rule out false-negative results.
- Current findings do not provide statistically significant evidence for the efficacy of MTX in treating early diffuse SSc.