Patient-important outcomes in registered diabetes trials
Patient-important outcomes in registered diabetes trials
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DOI：
10.1001/jama.299.21.2543
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发表时间：
2008-06-04
影响因子：
120.7
通讯作者：
Montori, Victor M.
中科院分区：
文献类型：
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作者：
Gandhi, Gunjan Y.;Murad, M. Hassan;Montori, Victor M.
背景糖尿病干预的安全性和有效性一直受到关注，部分原因是随机临床试验（RCT）尚未测量其对患者重要结局（即死亡和生活质量）的影响（发病率、疼痛、功能）。目的系统地确定正在进行的和未来的糖尿病随机对照试验将在多大程度上确定患者重要的结果。数据来源2007年11月10日，我们检索了主要RCT注册中心ClinicalTrials。gov（http：//www. ClinicalTrials. gov）、国际标准随机对照试验编号注册表（http：//isrctn. org）和澳大利亚新西兰临床试验注册中心（http：//www.澳新中心研究选择我们确定了招募糖尿病患者的2期至4期RCT。在2019项RCT中，1054项合格。我们随机抽取了50%符合条件的随机对照试验（1054人中的527人）并选择了自注册成为强制性（2004年）以来注册的436人。数据提取独立工作的成对评审员收集研究特征并确定测量的结果及其类型（生理结果，被认为反映患者重要结果风险增加的替代结果，结果在纳入的436项已注册RCT中，24项（6%）尚未开始入组，109项（25%）正在积极入组，303项（69%）已完成入组。436项RCT中仅有78项（18%; 95%置信区间[ CI]，14%- 22%）的主要结局为患者重要结局，436项中有69项（16%; 95% CI，13%- 20%）的主要结局为生理和实验室结局，436项中有268项（61%; 95% CI，57%- 66%）的主要结局为替代结局。436例患者中有201例（46%; 95% CI，41%- 51%）将患者重要结局报告为主要或次要结局。在多变量分析中，大型试验（比值比[ OR]，1.10; 95% CI，每增加100例患者1.02- 1.19）和持续时间更长的试验（OR，1.03; 95% CI，每增加30天1.01- 1.06）更有可能，而平行设计RCT（OR，0.15; 95% CI，0.05- 0.44）和2型糖尿病试验（OR，0.23; 95%CI，0.09- 0.61）不太可能将患者重要结局作为主要结局进行评估。只有18%的患者将重要结果作为主要结果。
Context Concerns about the safety and efficacy of diabetes interventions persist, in part because randomized clinical trials ( RCTs) have not measured their effect on patient-important outcomes, ie, death and quality of life ( morbidity, pain, function).Objective To systematically determine the extent to which ongoing and future RCTs in diabetes will ascertain patient- important outcomes.Data Sources On November 10, 2007, we searched primary RCT registries ClinicalTrials. gov ( http:// www. clinicaltrials. gov), International Standard Randomized Controlled Trial Number Register ( http:// isrctn. org), and Australian New Zealand Clinical Trials Registry ( http:// www. anzctr. org. au).Study Selection We identified phase 2 through 4 RCTs enrolling patients with diabetes. Of 2019 RCTs, 1054 proved eligible. We randomly sampled 50% of the eligible RCTs ( 527 of 1054) and selected 436 registered since registration became mandatory ( 2004).Data Extraction Pairs of reviewers working independently collected study characteristics and determined the outcomes measured and their type ( physiological outcomes, surrogate outcomes thought to reflect an increased risk for patient- important outcomes, and patient- important outcomes).Results Of the 436 registered RCTs included in this analysis, 24 ( 6%) had not started enrollment, 109 ( 25%) were actively enrolling, and 303 ( 69%) had completed enrollment. Primary outcomes were patient- important outcomes in only 78 of 436 RCTs ( 18%; 95% confidence interval [ CI], 14%- 22%), physiological and laboratory outcomes in 69 of 436 ( 16%; 95% CI, 13%- 20%), and surrogate outcomes in 268 of 436 ( 61%; 95% CI, 57%- 66%). Patient- important outcomes were reported as primary or secondary outcomes in 201 of 436 ( 46%; 95% CI, 41%- 51%). In multivariate analysis, large trials ( odds ratio [ OR], 1.10; 95% CI, 1.02- 1.19 for every additional 100 patients) and trials of longer duration ( OR, 1.03; 95% CI, 1.01- 1.06 for every additional 30 days) were more likely while parallel design RCTs ( OR, 0.15; 95% CI, 0.05- 0.44) and type 2 diabetes trials ( OR, 0.23; 95% CI, 0.09- 0.61) were less likely to assess patient- important outcomes as a primary outcome.Conclusion In this sample of registered ongoing RCTs in diabetes, only 18% included patient- important outcomes as primary outcomes.