Dall'Era Maria, Wofsy David
Division of Rheumatology, University of California-San Francisco, San Francisco, CA, USA.
Curr Opin Rheumatol. 2006 Sep;18(5):476-80. doi: 10.1097/01.bor.0000240357.22680.63.
The purpose of this review is to discuss the challenges inherent in the design of clinical trials for patients with systemic lupus erythematosus.
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.
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.
本综述旨在探讨系统性红斑狼疮患者临床试验设计中固有的挑战。
近年来，人们对开展系统性红斑狼疮临床试验重新产生了兴趣。在决定这些试验的成败方面，有三个关键的设计决策起着尤为关键的作用：从疾病表现非常异质性的患者中选择研究人群；选择治疗方案和对照方案；以及选择主要终点。最近的四项随机对照试验在包括新生物制剂、激素疗法和抗代谢药物等多种干预措施的背景下处理了这些决策。这些试验为每种方法的意义提供了信息。
由于这种疾病固有的复杂性，系统性红斑狼疮研究人员面临着重大的试验设计挑战。希望从既往和正在进行的试验中吸取的经验教训将为未来的试验设计提供参考，从而能够确定新药物的疗效，并为系统性红斑狼疮患者提供新的治疗方法。
