Therapeutic responses in patients with advanced NSCLC enrolled in early-phase clinical trials at MD Anderson.

We evaluated clinical outcomes and safety profiles in patients with non-small cell lung cancer (NSCLC) treated in early-phase trials. A retrospective review of 546 NSCLC cases treated from January 2016 to December 2024 at The University of Texas MD Anderson Cancer Center was performed using the MD Anderson CHIMERA database. Patients were categorized into seven groups based on treatment regimen. The overall objective response rate (ORR) was 19.9%, and the highest ORRs were in the targeted combination (30.8%) and targeted monotherapy (29.7%) groups. Gene alteration-matched therapy, serum albumin level, metastatic burden, treatment group, and liver metastasis were independently associated with progression-free survival. For overall survival, albumin level, metastatic burden, liver metastasis, and treatment regimen group showed independent significant associations. While overall safety profiles were tolerable, combination regimens were associated with increased proportions of grade ≥3 adverse events and dose modifications.