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KRAS inhibition in pancreatic cancer: pooled efficacy and safety signals from early-phase studies

Tiede, K. O. M.; Teixeira, M. F.; De Moura, M.; Chiaini, O.; Sonbol, M. B.; Borad, M.; Bekaii-Saab, T.; Uson Junior, P. L. S.

2025-12-04 oncology
10.64898/2025.12.03.25341547 medRxiv
Show abstract

Pancreatic ductal adenocarcinoma (PDAC) remains one of the most lethal cancers, driven by KRAS mutations long deemed "undruggable". We conducted a meta-analysis of five early phase cohorts (n=301) that evaluated KRAS-targeted therapies in patients with PDAC. The pooled objective response rate was 28% (95% CI 22-35%), indicating promising activity in refractory PDAC, with consistent estimates across the studies (I2=0%). Gastrointestinal toxicities were common (diarrhea, 47%; nausea, 43%). These findings validate direct KRAS inhibition as a breakthrough concept in PDAC but are tempered by modest durability, risk of bias, and limitations of early phase designs, underscoring the need for biomarker-guided, rigorously designed clinical trials.

Published in Scientific Reports (predicted rank #14) · training set

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