Karassa Fotini B, Tatsioni Athina, Ioannidis John P A
Clinical Trials and Evidence-Based Medicine Unit, the University of Ioannina School of Medicine, Ioannina, Greece.
J Rheumatol. 2003 May;30(5):979-84.
To appraise systematically the study design and quality of reporting of randomized controlled trials (RCT) on systemic lupus erythematosus (SLE) and to identify potential defects and biases.
RCT with at least 5 patients with SLE were retrieved from MEDLINE, EMBASE, and the Cochrane Library. We analyzed study design, quality of reporting, and trial results.
Ninety-four trial reports (37 on lupus nephritis) were eligible with 2,257 SLE patients (n = 795 in lupus nephritis trials). Median sample size was 28 patients. Fifty-one trials (54.3%) were double blind, but only 31 (33.0%) mentioned the randomization mode, only 19 (20.2%) described allocation concealment, and only 7 (7.5%) were adequately powered. Sixty-three trials (67%) described adequately reasons for withdrawals. Nephritis trials had on average longer followup (p = 0.001) and were less likely to be double blind (p < 0.001), to describe reasons for withdrawals [both overall (p = 0.008) and per arm (p = 0.009)] and to involve a comparison against placebo or no treatment (p < 0.001). Larger trials scored higher on several quality characteristics. Significant efficacy or trend for efficacy was claimed in 72 reports (76.6%) and this was even more common in trials published in 1999-2002 (89.5%). Significant efficacy was found more frequently in trials that clearly specified withdrawals per arm (p = 0.001) and outcomes (p = 0.001) and used intention-to-treat analyses (p = 0.03). Besides outcome specification, no other quality variables seemed to improve significantly over time.
Several aspects of the design and reporting of RCT on SLE can be improved. Larger, adequately powered, and accurately reported trials are needed.
系统评价系统性红斑狼疮（SLE）随机对照试验（RCT）的研究设计及报告质量，并识别潜在缺陷与偏倚。
从MEDLINE、EMBASE和Cochrane图书馆检索至少纳入5例SLE患者的RCT。我们分析了研究设计、报告质量及试验结果。
94篇试验报告（37篇关于狼疮性肾炎）符合要求，共纳入2257例SLE患者（狼疮性肾炎试验中n = 795例）。样本量中位数为28例患者。51项试验（54.3%）为双盲试验，但仅31项（33.0%）提及随机化方式，仅19项（20.2%）描述了分配隐藏，仅7项（7.5%）有足够的检验效能。63项试验（67%）充分描述了退出原因。肾炎试验平均随访时间更长（p = 0.001），双盲试验的可能性更小（p < 0.001），描述退出原因的可能性更小[总体（p = 0.008）及各治疗组（p = 0.009）]，且与安慰剂或不治疗进行比较的可能性更小（p < 0.001）。规模较大的试验在多个质量特征方面得分更高。72篇报告（76.6%）宣称有显著疗效或疗效趋势，在1999 - 2002年发表的试验中更为常见（89.5%）。在明确规定各治疗组退出情况（p = 0.001）和结局（p = 0.001）并采用意向性分析的试验中，更频繁地发现显著疗效（p = 0.03）。除结局明确外，似乎没有其他质量变量随时间有显著改善。
SLE的RCT在设计和报告的几个方面有待改进。需要开展规模更大、检验效能充足且报告准确的试验。
