Methotrexate (MTX) is primarily used in the treatment of
malignancies. It has also been used as an
immunosuppressive agent in the treatment of
pemphigus and
pemphigoid. The objective of this study was to determine the role of MTX in the treatment of
pemphigus and
pemphigoid based on an analysis of the available literature. A retrospective analysis of the English language literature was conducted. The studies included in this analysis were required to fulfil the following inclusion criteria: English language; diagnosis based on histology and immunopathology; minimum of five patients in each series; and data for efficacy, spectrum of responses and follow-up. A total of 136 patients with
pemphigus were reported in seven studies. One hundred and eleven of the 136 patients (82%) showed clinical improvement with MTX. A total of 79 patients with
pemphigoid were reported in six studies. Overall, 74 of the 79 patients (94%) showed clinical improvement.
Nausea and
infection were the most common side-effects. Death due to MTX resulted in seven of 215 patients (3%). There is a lack of randomized controlled trials. In many studies in this review there was insufficient information on clinical follow-up post-
therapy and on serological correlations. Analysis of the data suggests that MTX may be useful and effective in patients with
pemphigus vulgaris, who are
corticosteroid dependent or who develop significant complications in relation to
corticosteroids. MTX is likely to be more beneficial in patients with
pemphigoid, particularly in
bullous pemphigoid, than in patients with
pemphigus. Given the limitations of the available data, it appears that when there is a need for adjuvant
therapy, MTX may be considered early in the management of moderate to moderately severe disease.