Treatment of refractory myositis. A randomized crossover study of two new cytotoxic regimens

Lourdes Villalba, Jeanne E. Hicks, Elizabeth M. Adams, Jeffrey B. Sherman, Mark F. Gourley, Richard L. Leff, Bonnie C. Thornton, Suzanne H. Burgess, Paul H. Plotz, Frederick W. Miller

Research output: Contribution to journalArticlepeer-review

152 Scopus citations


Objective. To assess the clinical usefulness of 2 novel therapies for treatment-resistant myositis. Methods. Thirty patients with refractory myositis, of whom 25 had an inadequate or no response to previous cytotoxic therapy, were randomized to begin either a combination of weekly oral methotrexate and daily azathioprine (MTX/AZA) or intravenous methotrexate with leucovorin rescue (IV MTX) every 2 weeks for 6 months. Crossover to the alternate therapy occurred according to defined rules; evaluations of muscle strength and functional abilities were performed at the beginning, and after 3 and 6 months, of each treatment. Results. Of the 15 patients initially randomized to oral MTX/AZA, 8 improved with oral therapy and 1 improved with IV MTX during the crossover period. Of the 15 patients initially randomized to IV MTX therapy, 3 improved with the IV therapy and 4 with the oral combination during the crossover period. Although the study lacked the power to directly compare both treatments, intention-to-treat analysis showed a trend in favor of those patients who first received oral combination therapy (P = 0.025). There were 0.09 adverse events per patient-month with oral combination therapy and 0.16 per patient-month with IV therapy (P = 0.09). Conclusion. Combination oral MTX/AZA may benefit patients with treatment- resistant myositis, including those who previously had inadequate responses to either MTX or AZA alone. IV MTX with leucovorin rescue may also benefit some patients with refractory myositis.

Original languageEnglish (US)
Pages (from-to)392-399
Number of pages8
JournalArthritis and Rheumatism
Issue number3
StatePublished - Mar 1998
Externally publishedYes

ASJC Scopus subject areas

  • Immunology
  • Rheumatology


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