Li Yimei, Yuan Ying
Department of Biostatistics, Epidemiology and Informatics, University of Pennsylvania & The Children's Hospital of Philadelphia, Philadelphia, USA.
Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, USA.
J Biopharm Stat. 2025 Jun 4:1-20. doi: 10.1080/10543406.2025.2512203.
The basket trial is a novel type of trial that evaluates one treatment in multiple indications (such as cancer types) simultaneously. One challenge of applying the basket trial design to pediatric studies is limited accrual, resulting in low statistical power. To address this issue, we propose a Bayesian asket design for ediatric trials with xternal ata (BPED) that performs dual-information borrowing to improve the design efficiency: borrow information from the external data to the pediatric trial, and borrow information between the cancer types within the pediatric trial. BPED also accommodates potential heterogeneous treatment effects across cancer types by allowing each cancer type belonging to the sensitive or insensitive latent subgroups. The design adaptively updates the members of the subgroups based on the accumulated pediatric and external data to make go/no-go decisions for each cancer type. The simulation study shows that, compared to some existing designs, BPED yields higher power to detect the treatment effect for sensitive cancer types and maintains a desirable type I error rate for insensitive cancer types.
篮子试验是一种新型试验，可同时评估一种治疗方法在多种适应症（如癌症类型）中的效果。将篮子试验设计应用于儿科研究的一个挑战是入组受限，导致统计效力较低。为解决这一问题，我们提出了一种用于儿科试验的带有外部数据的贝叶斯篮子设计（BPED），该设计通过进行双重信息借用以提高设计效率：从外部数据向儿科试验借用信息，并在儿科试验中的癌症类型之间借用信息。BPED还通过允许每种癌症类型属于敏感或不敏感潜在亚组来适应不同癌症类型之间潜在的异质性治疗效果。该设计基于累积的儿科和外部数据自适应地更新亚组成员，以便对每种癌症类型做出继续/停止决策。模拟研究表明，与一些现有设计相比，BPED在检测敏感癌症类型的治疗效果方面具有更高的效力，并对不敏感癌症类型维持理想的I型错误率。
