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期刊来源：Ann Intern Med

原文链接：https://doi.org/10.7326/M23-2515

摘要内容如下：

背景

许多患者参与癌症试验以获得新疗法。新的治疗方法在多大程度上对试验参与者产生临床益处尚不清楚。

目的

在6个实体瘤的随机试验中，评估分配到实验组的无进展生存期（PFS）和总生存期（OS）优势。

数据源

在ClinicalTrials.gov网站上搜索了2017年至2021年期间发布的研究药物试验结果。

研究选择

研究药物被定义为尚未获得美国食品和药物管理局完全批准的研究适应症药物。如果试验是随机的，并测试了药物或生物制剂，则包括在内。

数据提取

数据提取由2名独立评价者完成。使用随机效应模型汇总数据。

数据综合

样本包括128项试验，其中包括141项新药和对照药物的比较。这些比较包括47050名患者。PFS的合并风险比为0.80（95%CI，0.75至0.85），表明实验组患者的获益具有统计学意义。这相当于中位PFS优势为1.25个月（CI，0.80至1.68个月）。OS的合并风险比为0.92（CI，0.88至0.95），相当于生存期增加1.18个月（CI，0.72至1.71个月）。对照组患者发生严重不良事件的绝对风险为29.56%（CI为26.64%至32.65%），实验组患者发生严重不良事件的风险增加7.40%（CI为5.66%至9.14%）。

局限性

该样本中的试验是异质性的。假设对照组的干预措施反映了护理标准。

结论

分配到实验组产生了统计学上显著的生存收益。然而，绝对生存增益很小，而毒性在统计学上显著更大。这篇综述的结果提供了可靠的证据，表明患者不会因为被分配到对照组而处于不利地位。

主要资金来源

加拿大卫生研究院。

英文原文如下：

Abstracts

BACKGROUND  Many patients participate in cancer trials to access new therapies. The extent to which new treatments produce clinical benefit for trial participants is unclear.

PURPOSE  To estimate the progression-free survival (PFS) and overall survival (OS) advantage of assignment to experimental groups in randomized trials for 6 solid tumors.

DATA SOURCES  ClinicalTrials.gov was searched for trials of investigational drugs with results posted between 2017 and 2021.

STUDY SELECTION  Investigational drugs were defined as those not yet having full approval from the U.S. Food and Drug Administration for the study indication. Trials were included if they were randomized and tested drugs or biologics.

DATA EXTRACTION  Data extraction was completed by 2 independent reviewers. Data were pooled using a random-effects model.

DATA SYNTHESIS  The sample included 128 trials comprising 141 comparisons of a new drug and a comparator. These comparisons included 47 050 patients. The pooled hazard ratio for PFS was 0.80 (95% CI, 0.75 to 0.85), indicating statistically significant benefit for patients in experimental groups. This corresponded to a median PFS advantage of 1.25 months (CI, 0.80 to 1.68 months). The pooled hazard ratio for OS was 0.92 (CI, 0.88 to 0.95), corresponding to a survival gain of 1.18 months (CI, 0.72 to 1.71 months). The absolute risk for a serious adverse event for comparator group patients was 29.56% (CI, 26.64% to 32.65%), with an increase in risk of 7.40% (CI, 5.66% to 9.14%) for patients in experimental groups.

LIMITATIONS  Trials in this sample were heterogeneous. Comparator group interventions were assumed to reflect standard of care.

CONCLUSION  Assignment to experimental groups produces statistically significant survival gains. However, the absolute survival gain is small, and toxicity is statistically significantly greater. The findings of this review provide reassuring evidence that patients are not meaningfully disadvantaged by assignment to comparator groups.

PRIMARY FUNDING SOURCE  Canadian Institutes of Health Research.

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