PMID- 35272564 OWN - NLM STAT- MEDLINE DCOM- 20220321 LR - 20220321 IS - 1365-2060 (Electronic) IS - 0785-3890 (Print) IS - 0785-3890 (Linking) VI - 54 IP - 1 DP - 2022 Dec TI - Real-world outcomes of patients with advanced intrahepatic cholangiocarcinoma treated with programmed cell death protein-1-targeted immunotherapy. PG - 803-811 LID - 10.1080/07853890.2022.2048416 [doi] AB - OBJECTIVE: There is a lack of effective treatment to improve the prognosis of intrahepatic cholangiocarcinoma (ICC). Programmed cell death protein-1 (PD-1)-targeted immunotherapy has shown promising results in a variety of malignant tumours. However, in patients with advanced ICC, the safety and efficacy of anti-PD-1 agents remain unclear. METHODS: Forty-two advanced ICC patients treated with anti-PD-1 agents from August 2018 to December 2020 were retrospectively analyzed. Tumour response, overall survival (OS), progression-free survival (PFS), and time to tumour progression (TTP) were evaluated. Adverse events were also recorded. RESULTS: The median duration of follow-up was 12.1 months, and the median time of treatment was 6.7 months for all patients. The median OS, median PFS, and median TTP for the whole cohort were 19.3 months, 11.6 months, and 11.6 months, respectively. The overall response rate (ORR) and disease control rate (DCR) for the whole cohort were 23.8% and 85.7%, respectively. Of the 42 evaluable individuals, two (4.8%) had hyperprogressive disease. The most common adverse events (AEs) were pain (n = 6; 14.3%), anorexia (n = 4; 9.5%), hypertension (n = 4; 9.5%), pyrexia (n = 3; 7.1%), cough (n = 3; 7.1%), and hypothyroidism (n = 3; 7.1%). The median OS of patients with albumin-bilirubin (ALBI) grade 1 was longer than that of patients with ALBI grade 2 (19.3 months vs. 14.7 months). The median PFS did not show a significant difference between ALBI grade 1 and grade 2 patients (13.6 months vs. 6.9 months). CONCLUSIONS: PD-1-targeted immunotherapy showed promising efficacy and safety in advanced ICC patients.Key messagesPD-1-targeted immunotherapy is a safe and effective treatment for advanced ICC patients.This study provides therapeutic strategy for advanced ICC patients. FAU - Deng, Min AU - Deng M AD - Department of Liver Surgery, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. FAU - Li, Shaohua AU - Li S AD - Department of Liver Surgery, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. FAU - Wang, Qiaoxuan AU - Wang Q AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. AD - Department of Radiation Oncology, Sun Yat-sen University Cancer Center, Guangzhou, China. FAU - Zhao, Rongce AU - Zhao R AD - Department of Liver Surgery, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. FAU - Zou, Jingwen AU - Zou J AD - Department of Liver Surgery, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. FAU - Lin, Wenping AU - Lin W AD - Department of Liver Surgery, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. FAU - Mei, Jie AU - Mei J AD - Department of Liver Surgery, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. FAU - Wei, Wei AU - Wei W AD - Department of Liver Surgery, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. FAU - Guo, Rongping AU - Guo R AD - Department of Liver Surgery, Sun Yat-sen University Cancer Center, Guangzhou, China. AD - State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PL - England TA - Ann Med JT - Annals of medicine JID - 8906388 RN - 0 (Apoptosis Regulatory Proteins) RN - 0 (Programmed Cell Death 1 Receptor) SB - IM MH - Apoptosis Regulatory Proteins/therapeutic use MH - *Bile Duct Neoplasms/drug therapy MH - Bile Ducts, Intrahepatic MH - *Cholangiocarcinoma/drug therapy MH - Humans MH - Immunotherapy/adverse effects MH - Programmed Cell Death 1 Receptor MH - Retrospective Studies PMC - PMC8920361 OTO - NOTNLM OT - Intrahepatic cholangiocarcinoma OT - PD-1 OT - immunotherapy OT - outcome OT - real-world COIS- None of the authors does not have any conflicts of interests. This study was supported by the National Natural Science Foundation of China [No. 82172579, No. 81871985]; Natural Science Foundation of Guangdong Province [No. 2018A0303130098]; Science and Technology Planning Project of Guangdong Province [No. 2017A020215112]; Science and Technology Planning Project of Guangzhou [No. 201903010017 and No. 201904010479]; Clinical Trials Project [5010 Project] of Sun Yat-sen University [No. 5010-2017009]; Clinical Trials Project [308 Project] of Sun Yat-sen University Cancer Centre [No. 308-2015-014]; and Medical Innovation Project of the First Affiliated Hospital of Guangzhou University of Chinese Medicine [2019IIT18]. EDAT- 2022/03/12 06:00 MHDA- 2022/03/22 06:00 PMCR- 2022/03/11 CRDT- 2022/03/11 05:31 PHST- 2022/03/11 05:31 [entrez] PHST- 2022/03/12 06:00 [pubmed] PHST- 2022/03/22 06:00 [medline] PHST- 2022/03/11 00:00 [pmc-release] AID - 2048416 [pii] AID - 10.1080/07853890.2022.2048416 [doi] PST - ppublish SO - Ann Med. 2022 Dec;54(1):803-811. doi: 10.1080/07853890.2022.2048416.