Biomarker study of pembrolizumab in patients with advanced rare cancers.

doi:10.1016/j.xcrm.2026.102827

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Article

Biomarker study of pembrolizumab in patients with advanced rare cancers.

Cell reports. Medicine - 16 Jun 2026

Naing Aung, Li Xiqi, Wang Qi, Tian Xiangjun Jun, Sabir Sharjeel, Bhosale Priya R, Derbala Mohamed H, Stephen Bettzy, Xu Mingxuan, Hajjar Joud, Gurses Serdar A, Yang Yali, Momin Hassan Ahmed, Wistuba Ignacio Ivan, Alshawa Anas, Shaw Kenna R, Zarifa Abdulrazzak, Singh Gopal, Pandurengan Renganayaki Krishna, Raso Gabriela Maria, Parra Edwin Roger, Song Juhee, Mohammad Mohammad Moustaf, Rodon Ahnert Jordi, Fu Siqing, Subbiah Vivek, Piha-Paul Sarina Anne, Wang Jing, Woodman Scott Eric, Meric-Bernstam Funda

Abstract excerpt

Rare cancers lack robust, evidence-based treatment options. We conduct a prospective, multi-histology phase 2 clinical trial of pembrolizumab in 154 patients (142 evaluable), observing an objective response rate of 14.8% and clinical benefit (CB) in 26.8%. Multi-modal profiling is performed on baseline, on-treatment, and progression samples. CB associates with high microsatellite instability (MSI-H)/high tumor...

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Identifiers

PMID: 42167248
DOI: 10.1016/j.xcrm.2026.102827
PMCID: PMC13293929

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