Klas Katarzyna, Strzebonska Karolina, Zaborowska Lucja, Krawczyk Tomasz, Włodarczyk Alicja, Bąk-Kuchejda Urszula, Polak Maciej, Van Wambeke Simon, Waligora Marcin
Research Ethics in Medicine Study Group (REMEDY), Department of Bioethics, Faculty of Health Sciences, Jagiellonian University Medical College, Michalowskiego 12, 31-126, Kraków, Poland.
Doctoral School of Medical and Health Sciences, Jagiellonian University Medical College, Kraków, Poland.
Target Oncol. 2025 Jan;20(1):89-101. doi: 10.1007/s11523-024-01107-3. Epub 2024 Oct 26.
Oncology research is increasingly adopting new clinical trial models that implement the concept of precision medicine. One of these is the basket clinical trial design. Basket clinical trials allow new treatments to be evaluated across multiple tumor types. Patients recruited to basket clinical trials share certain molecular characteristics of their cancer that are predictive of clinical benefit from the experimental treatment.
Our aim was to describe the risks and benefits of basket clinical trials in oncology.
Our study was prospectively registered in PROSPERO (CRD42023406401). We systematically searched PubMed, Embase, and ClinicalTrials.gov for reports of basket clinical trials in oncology published between 1 January, 2001, and 14 June, 2023. We measured the risk by treatment-related adverse events (grades 3, 4, and 5), and the benefit by objective response rate. We also extracted and analyzed data on progression-free survival and overall survival. When possible, data were meta-analyzed.
We included 126 arms of 75 basket clinical trials accounting for 7659 patients. The pooled objective response rate was 18.0% (95% confidence interval [CI] 14.8-21.1). The rate of treatment-related death was 0.7% (95% CI 0.4-1.0), while 30.4% (95% CI 24.2-36.7) of patients experienced grade 3/4 drug-related toxicity. The median progression-free survival was 3.1 months (95% CI 2.6-3.9), and the median overall survival was 8.9 months (95% CI 6.7-10.2).
Our results provide an empirical basis for communicating about the risks and benefits of basket clinical trials and for refining new models of clinical trials applied in precision medicine.
肿瘤学研究越来越多地采用实施精准医学概念的新临床试验模式。其中之一是篮式临床试验设计。篮式临床试验允许在多种肿瘤类型中评估新疗法。招募到篮式临床试验中的患者具有其癌症的某些分子特征，这些特征可预测实验性治疗的临床获益。
我们的目的是描述肿瘤学中篮式临床试验的风险和获益。
我们的研究已在PROSPERO（CRD42023406401）上进行前瞻性注册。我们系统检索了PubMed、Embase和ClinicalTrials.gov，以获取2001年1月1日至2023年6月14日期间发表的肿瘤学篮式临床试验报告。我们通过治疗相关不良事件（3级、4级和5级）衡量风险，通过客观缓解率衡量获益。我们还提取并分析了无进展生存期和总生存期的数据。如有可能，对数据进行荟萃分析。
我们纳入了75项篮式临床试验的126个臂，涉及7659名患者。汇总的客观缓解率为18.0%（95%置信区间[CI]14.8 - 21.1）。治疗相关死亡率为0.7%（95%CI 0.4 - 1.0），而30.4%（95%CI 24.2 - 36.7）的患者经历了3/4级药物相关毒性。中位无进展生存期为3.1个月（95%CI 2.6 - 3.9），中位总生存期为8.9个月（95%CI 6.7 - 10.2）。
我们的结果为就篮式临床试验的风险和获益进行沟通以及完善精准医学中应用的新临床试验模式提供了实证依据。
