A critical review of clinical trials in systemic lupus erythematosus

M. A. Mahieu*, V. Strand, L. S. Simon, P. E. Lipsky, R. Ramsey-Goldman

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

46 Scopus citations


One challenge in caring for patients with systemic lupus erythematosus (SLE) is a paucity of approved therapeutics for treatment of the diverse disease manifestations. In the last 60 years, only one drug, belimumab, has been approved for SLE treatment. Critical evaluation of investigator initiated and pharma-sponsored randomized controlled trials (RCTs) highlights barriers to successful drug development in SLE, including disease heterogeneity, inadequate trial size or duration, insufficient dose finding before initiation of large trials, handling of background medications, and choice of primary endpoint. Herein we examine lessons learned from landmark SLE RCTs and subsequent advances in trial design, as well as discuss efforts to address limitations in current SLE outcome measures that will improve detection of true therapeutic responses in future RCTs.

Original languageEnglish (US)
Pages (from-to)1122-1140
Number of pages19
Issue number10
StatePublished - Sep 1 2016


  • Systemic lupus erythematosus
  • clinical trials
  • outcome measures

ASJC Scopus subject areas

  • Rheumatology


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