已发表的随机3期癌症试验遵循常识性肿瘤学提出的原则

IF 7.1 1区医学 Q1 ONCOLOGY

European Journal of Cancer Pub Date : 2025-10-02 DOI:10.1016/j.ejca.2025.116041

Omar Abdihamid , Bishal Gyawali , Christopher M. Booth , Elizabeth A. Eisenhauer , Wilma M. Hopman , Brian Shkabari , Dario Trapani , Haydee C. Verduzco-Aguirre , Brooke E. Wilson , Ian F. Tannock

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引用次数: 0

摘要

背景：随机对照试验（RCTs）应严格设计，适当分析，并准确报告，以便根据患者的优先事项共同制定癌症治疗决策。常识肿瘤学（Common-Sense Oncology， CSO）已经公布了它们的设计、分析和报告原则，并附有检查清单来帮助确保这一点。方法：我们将CSO清单应用于2023年发表的55项评估实体肿瘤全身疗法的随机对照试验，以评估它们遵守CSO原则的程度，并为与未来试验的比较提供基准。在47项晚期疾病试验中，无进展生存期（PFS）和总生存期（OS）分别为53. %和43. %的主要终点。55项试验中有12项（22 %）的对照组在试验开始时被认为是不适当的护理标准，60 %的试验证明他们选择了替代OS的主要终点，42 %的试验将健康相关生活质量（HRQoL）测量作为次要终点。只有两项（3.6% %）试验研究了限制审查和退出的策略：在40% %的试验中，审查患者的数量显示在Kaplan-Meier曲线以下，但只有5项（9% %）试验进行了敏感性分析，以确定审查的潜在影响。只有一项试验报告了持续性毒性，只有3项试验报告提到了成本或成本效益。通过系统地应用CSO检查表，我们发现了评估实体癌全身治疗的随机对照试验的设计、分析和报告中的几个缺陷。我们的调查强调需要改进随机对照试验的设计和报告，使读者能够正确地解释他们的结果。

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Adherence of published randomised Phase 3 cancer trials to principles proposed by common-sense oncology

Background

Randomised Controlled Trials (RCTs) should be designed rigorously, analysed appropriately, and reported accurately to enable shared decision making about cancer treatments that is aligned with patient priorities. Common-Sense Oncology (CSO) has published principles for their design, analysis, and reporting, with checklists to help ensure this.

Methods

We applied CSO checklists to 55 RCTs evaluating systemic therapies for solid tumours published in 2023 to assess the extent to which they adhered to CSO principles, and to provide a benchmark for comparison with future trials.

Results

Progression-free survival (PFS) and overall survival (OS) were primary endpoints in 53 % and 43 % of 47 trials for advanced disease, respectively. Twelve of 55 (22 %) trials had a control arm judged to be an inappropriate standard of care at time of trial initiation, 60 % justified their selection of an alternative primary endpoint to OS, and 42 % included Health-Related Quality-of-Life (HRQoL) measures as a secondary endpoint. Only two (3.6 %) trials addressed strategies to limit censoring and dropout: numbers of censored patients were shown below Kaplan-Meier curves in 40 % of the trials, but sensitivity analysis to determine potential effects of censoring was undertaken in only 5 (9 %) trials. Persistent toxicities were reported in only one trial, and only 3 trial reports mentioned cost or cost-effectiveness.

Conclusions

By applying the CSO checklists systematically, we identified several deficiencies in the design, analysis, and reporting of RCTs evaluating systemic therapies for solid cancers. Our survey highlights the need for improvements in design and reporting of RCTs to enable readers to correctly interpret their results.

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来源期刊

European Journal of Cancer 医学-肿瘤学

CiteScore

11.50

自引率

4.80%

发文量

953

审稿时长

23 days

期刊介绍： The European Journal of Cancer (EJC) serves as a comprehensive platform integrating preclinical, digital, translational, and clinical research across the spectrum of cancer. From epidemiology, carcinogenesis, and biology to groundbreaking innovations in cancer treatment and patient care, the journal covers a wide array of topics. We publish original research, reviews, previews, editorial comments, and correspondence, fostering dialogue and advancement in the fight against cancer. Join us in our mission to drive progress and improve outcomes in cancer research and patient care.