Methotrexate in the treatment of pemphigus vulgaris: experience in 23 patients

Summary Background The standard treatment for patients with pemphigus vulgaris has long consisted of high‐dose glucocorticoids. Studies regarding the use of methotrexate in pemphigus vulgaris date back to 1968, but few have quantitatively described a steroid‐sparing effect conferred by methotrexate. Objectives We sought to evaluate the efficacy of methotrexate in 23 patients with pemphigus vulgaris, using the drug's steroid‐sparing effect as the primary indicator of clinical improvement. We investigated whether methotrexate could be used as monotherapy in some patients. Methods Retrospective chart review was used to analyse the records of patients with pemphigus vulgaris treated with methotrexate at the New York University Langone Medical Center for at least three consecutive months between 2000 and 2012. Diagnosis was made by tissue biopsy and either direct or indirect immunofluorescence tests and enzyme‐linked immunosorbent assay. Results Improvement in clinical symptoms was observed in 91% of patients. Sixteen patients (70%) were eventually weaned completely off prednisone, with a mean time to discontinuation of 18 months. In total 23% of patients enjoyed a partial steroid‐sparing effect, requiring a mean maintenance dose of prednisone of 6·75 mg daily. Two patients (9%) developed possible adverse events requiring cessation of the drug, and one patient received no therapeutic benefit from the drug. Conclusions Methotrexate is a useful and well‐tolerated therapy with considerable steroid‐sparing effect in patients with pemphigus vulgaris. It may be considered a first‐line adjuvant therapy in the treatment of this difficult disease. What's already known about this topic? Systemic glucocorticoids have long been the standard first‐line treatment in pemphigus vulgaris, but are associated with considerable morbidity. The optimal adjuvant systemic therapy for pemphigus vulgaris has yet to be determined. What does this study add? In 23 patients with pemphigus vulgaris treated with methotrexate at our centre, 91% were able to reduce their dosage of prednisone, with 70% able to discontinue prednisone completely after a median interval of 18 months. Methotrexate is a useful, well‐tolerated, and possibly underutilized drug in the treatment of pemphigus vulgaris. It successfully controls blistering as a systemic monotherapy in the majority of patients.