A Cochrane review included 7 studies with a total of 205 subjects. Ciclosporin either as monotherapy (1 trial, n=20) or with corticosteroids (1 trial, n=39) resulted in improvement compared with placebo (relative rate of improvement 2.44, 95% confidence interval [CI] 1.13 to 5.27). The weighted mean difference in QMG (Quantitative MG Score for Disease Severity) score between the ciclosporin and placebo groups was –0.34 (95% CI –0.52 to –0.17). Azathioprine (plus prednisolone for the first month) had no significant benefit over prednisolone alone (n=41) in terms of treatment failure. Results with azathioprine plus prednisolone versus prednisolone plus placebo (n=34) were similar. The duration of remission was significantly longer in the azathioprine plus prednisolone group compared with the prednisolone plus placebo group (RR 0.28, 95% CI 0.08 to 0.94). Cyclophosphamide was reported to be statistically more efficacious than placebo at 12 months in corticosteroid-dependent participants (n=23), but data for individual participants were unavailable. Trials of mycophenolate mofetil and tacrolimus did not provide relevant endpoint data for the review.
Comment: The quality of evidence is downgraded by imprecise results (limited study size for each comparison) and by inconsistency (heterogeneity in interventions and outcomes).