Ginzler Ellen M, Moldovan Ioana
SUNY Downstate Medical Center, Rheumatology Division, Brooklyn, New York 11203, USA.
Curr Opin Rheumatol. 2004 Sep;16(5):499-504. doi: 10.1097/01.bor.0000133659.14482.59.
The purpose of this review is to discuss the most recent published clinical trials for systemic lupus erythematosus and to identify important issues that have arisen in association with the search for new therapies for systemic lupus erythematosus, as well as new regimens or indications for the use of "standard-of-care" agents such as corticosteroids and cyclophosphamide.
Important developments have occurred during the past 2 years as interest in this area has increased, largely because of the participation of pharmaceutical and biotechnical companies in the development and testing of novel agents for systemic lupus erythematosus. Several important large-scale, multicenter, randomized controlled trials have been completed, but none has yet resulted in a new, approved indication for systemic lupus erythematosus. Many issues in the identification of new therapeutic modalities remain. These include the fact that a majority of published reports include either small numbers of patients in controlled trials that lack statistical power to draw conclusions, or are uncontrolled anecdotal series or individual case reports. Among the larger controlled trials, a pervasive issue in the failure to reach statistical significance may be the initial study design. Inclusion of patients with mild and/or stable disease activity does not allow for an effect size sufficient to show differences in treatment arms without recruitment of very large numbers of subjects. Finally, several potentially important trials have been reported only in abstract form to date. Further assessment of the results must await formal publication of these studies.
本综述旨在讨论系统性红斑狼疮最新发表的临床试验，并确定在寻找系统性红斑狼疮新疗法过程中出现的重要问题，以及诸如皮质类固醇和环磷酰胺等“标准治疗”药物使用的新方案或新适应症。
在过去两年中，随着该领域关注度的提高，出现了一些重要进展，这主要归功于制药和生物技术公司参与系统性红斑狼疮新型药物的研发和测试。几项重要的大规模、多中心、随机对照试验已经完成，但尚未有一项试验为系统性红斑狼疮带来新的获批适应症。在确定新治疗方式方面仍存在许多问题。这些问题包括：大多数已发表的报告要么是对照试验中患者数量较少，缺乏得出结论的统计学效力，要么是无对照的轶事系列或个别病例报告。在较大规模的对照试验中，未能达到统计学显著性的一个普遍问题可能是初始研究设计。纳入病情轻度和/或稳定的患者，若不招募大量受试者，就无法获得足以显示治疗组间差异的效应量。最后，到目前为止，几项潜在重要试验仅以摘要形式报道。对结果的进一步评估必须等待这些研究的正式发表。
