Tumor Mutational Burden as a Biomarker for Advanced Biliary Tract Cancer

Background : High tumor mutational burden (TMB-H) has been reported as a predictive marker to immunotherapy or prognostic marker in various tumor types. However, there has been little study of the role of TMB-H in advanced biliary tract cancer (BTC). Methods : We analyzed 119 advanced BTC patients w...

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Autores principales: Hongsik Kim MD, Hana Kim MD, Ryul Kim MD, PhD, Hyunji Jo MD, Hye Ryeon Kim MD, Joohyun Hong MD, PhD, Joon Oh Park MD, PhD, Young Suk Park MD, PhD, Seung Tae Kim MD, PhD
Formato: article
Lenguaje:EN
Publicado: SAGE Publishing 2021
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Acceso en línea:https://doaj.org/article/83fd42d3d7f54068817202539c732b95
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Sumario:Background : High tumor mutational burden (TMB-H) has been reported as a predictive marker to immunotherapy or prognostic marker in various tumor types. However, there has been little study of the role of TMB-H in advanced biliary tract cancer (BTC). Methods : We analyzed 119 advanced BTC patients who received Gemcitabine/Cisplatin (GP) as a first-line treatment between November 2019 and April 2021. Next-generation sequencing (NGS), including TMB analysis, as a routine clinical practice was performed in 119 patients. The TruSight TM Oncology 500 assay from Illumina was used as a cancer panel. Results : Among 119 patients, 18 (18.5%) had a tumor with high TMB (≥ 10 Muts/Mb). There were no significant differences between the status of TMB and clinical outcomes with GP, including objective response rate (ORR) ( P  = .126), disease control rate (DCR) ( p  = .454), and median progression-free survival (PFS) ( p  = .599). The median overall survival (OS) was not different between patients with TMB-H and no TMB-H ( p  = .430). In subgroup analysis of 32 patients receiving immune checkpoint inhibitor (ICIs), there were significant differences in ORR ( p  = .034) and median PFS ( p   =  .025) with ICIs between patients with and without TMB-H. Conclusions : This study revealed that TMB-H in advanced BTCs did not have a prognostic or role in the standard first-line treatment. However, TMB-H might be a predictive biomarker for response to ICIs in advanced BTC.