Clinical trial design in systemic lupus erythematosus.
OBJECTIVE
The purpose of this review is to discuss the challenges inherent in the design of clinical trials for patients with systemic lupus erythematosus.


RESULTS
Recent years have witnessed a renewed interest in the conduct of systemic lupus erythematosus clinical trials. Three key design decisions play particularly critical roles in determining the fate of these trials: selection of the study population from among patients with very heterogeneous disease manifestations; selection of the treatment and control regimens; and selection of the primary endpoint. Four recent randomized, controlled trials dealt with these decisions in the context of a variety of interventions, including new biologic agents, hormonal therapies, and anti-metabolites. These trials are informative about the implications of each approach.


CONCLUSIONS
Systemic lupus erythematosus investigators are faced with substantial trial design challenges because of the inherently complex nature of this disease. The hope is that lessons learned from prior and ongoing trials will inform future trial design such that the efficacy of new agents can be determined and new therapies will become available for patients with SLE.
Dall'Era
Maria
M
Division of Rheumatology, University of California-San Francisco, San Francisco, CA, USA.
Wofsy
David
D
D006801:Humans; D008180:Lupus Erythematosus, Systemic / Q000628:therapy*; D016032:Randomized Controlled Trials as Topic / Q000379:methods / Q000592:standards / Q000639:trends; D012107:Research Design / Q000639:trends; D016896:Treatment Outcome