查看文章

Objectives

Single-arm basket trials represent significant challenges for assessing comparative effectiveness. We compared progression-free survival (PFS) and overall survival (OS) of patients with eight metastatic cancers who received pembrolizumab versus patients receiving NCCN-guideline-based chemotherapies.

Methods

We applied inclusion/exclusion criteria from microsatellite instability–high (MSI-H) trials (Keynote-164 and Keynote-158) to TriNetx electronic health record database to derive chemotherapy control cohorts. Gaussian copulas were fitted to extract the dependence structure from the real-world controls, and trial samples were simulated by combining with marginal distributions of trial baseline covariates. Imbalances of known covariates were adjusted with inverse odds weighting. Median OS and PFS were compared between trial and real-world cohorts for all tumor types. Weighted Cox …