PD‐1/PD‐L1 Inhibitors Plus Chemotherapy Versus Chemotherapy Alone as First‐Line Therapy for Patients With Unfavorable Cancer of Unknown Primary: A Multicenter, Retrospective Cohort Study

ABSTRACT This multicenter study aimed to investigate the efficacy and safety of PD‐1/PD‐L1 inhibitors plus chemotherapy (ICI‐Chemo group) versus chemotherapy alone (Chemo group) for patients with cancer of unknown primary (CUP) in the first‐line setting. We included patients with unfavorable CUP acr...

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Main Authors: Riqing Huang, Haifeng Li, Shuo Li, Ditian Shu, Rishang Chen, Zhousan Zheng, Tinghua Gao, Meiting Chen, Anqi Hu, Yunjie Huang, Qiufan Zheng, Xin An, Cong Xue, Yuchen Cai, Yanxia Shi
Format: Article
Language:English
Published: Wiley 2025-03-01
Series:MedComm
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Online Access:https://doi.org/10.1002/mco2.70124
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Summary:ABSTRACT This multicenter study aimed to investigate the efficacy and safety of PD‐1/PD‐L1 inhibitors plus chemotherapy (ICI‐Chemo group) versus chemotherapy alone (Chemo group) for patients with cancer of unknown primary (CUP) in the first‐line setting. We included patients with unfavorable CUP across four medical centers in China. Between January 2014 and December 2023, 117 patients were enrolled: 46 patients in the ICI‐Chemo group and 71 patients in the Chemo group. After a median follow‐up of 28.1 months, the ICI‐Chemo group exhibited a significant improvement over the Chemo group in median PFS (9.10 months vs. 6.37 months; hazard ratio [HR] 0.46; 95% CI: 0.30–0.71; p < 0.001) and OS (35.67 months vs. 10.2 months; HR 0.37; 95% CI: 0.22–0.64; p < 0.001). Similarly, among patients who received TP (taxane plus platinum)‐based chemotherapies, OS and PFS benefits were observed in the ICI‐Chemo group. The objective response rate was higher in the ICI‐Chemo group than in the Chemo group (54.35% vs. 22.53%, p < 0.001). Grade 3 or higher drug‐related adverse events occurred in 11 patients (23.91%) in the ICI‐Chemo group and 28 patients (39.44%) in the Chemo group. Thus, PD‐1/PD‐L1 inhibitors plus chemotherapy could be the preferred first‐line treatment for patients with unfavorable CUP, providing improved efficacy and manageable toxicity.
ISSN:2688-2663