See the Appendix on page 434 for all participants and their roles in the study.
Article first published online: 17 JUN 2011
Copyright © 2011 American Neurological Association
Annals of Neurology
Volume 70, Issue 3, pages 427–436, September 2011
How to Cite
The Muscle Study Group (2011), A randomized, pilot trial of etanercept in dermatomyositis. Ann Neurol., 70: 427–436. doi: 10.1002/ana.22477
Clinicaltrials.gov registry # NCT00282880.
- Issue published online: 8 SEP 2011
- Article first published online: 17 JUN 2011
- Accepted manuscript online: 11 MAY 2011 01:37PM EST
- Manuscript Accepted: 6 MAY 2011
- Manuscript Revised: 25 APR 2011
- Manuscript Received: 2 FEB 2011
The aims of this pilot study were to assess (1) the safety and tolerability of etanercept in dermatomyositis (DM); (2) the feasibility and safety of a forced prednisone taper; and (3) outcome measures, including those recommended by the International Myositis Assessment Clinical Study (IMACS) group.
We conducted a randomized, double-blind, placebo-controlled trial of etanercept (50mg subcutaneously weekly) for 52 weeks in DM subjects. Subjects were tapered off prednisone in a standardized schedule as tolerated over the initial 24 weeks of the study. Principal outcomes included adverse events, time from randomization to treatment failure (inability to wean off prednisone on schedule), and average prednisone dosage after week 24.
Sixteen subjects were randomized, 11 to etanercept and 5 to placebo. There were no significant differences in adverse event rates between the treatment groups, although 5 etanercept-treated and 1 placebo-treated subjects developed worsening rash. All 5 subjects receiving placebo were treatment failures (median time to treatment failure 148 days). In contrast, 5 of 11 subjects in the etanercept arm were successfully weaned off prednisone; the median time to treatment failure in this group was 358 days (p = 0.0002). The median of the average prednisone dosage after week 24 was 29.2mg/day in the placebo group and 1.2mg/day in the etanercept group (p = 0.02). IMACS and other outcome measures demonstrated excellent test–retest reliability (intraclass correlation coefficients 0.79–0.99). There was no significant treatment effect on functional outcome.
The findings of no major safety concerns and a steroid-sparing effect in our study suggest that further investigation of etanercept as a treatment for DM is warranted. Ann Neurol 2011;