Adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer: A systematic review and meta-analysis of randomized controlled trials.

<h4>Objectives</h4>Several randomized controlled trials compared adjuvant systemic chemotherapy with observation in patients with resected biliary tract cancer (BTC) have yielded inconsistent outcomes. In order to assess the efficacy of adjuvant therapy in these patients, we conducted th...

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Main Authors: Liying Tian, Qian Guo, Daidi Fu, Xiao Ma, Linjun Wang
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2025-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0295583
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author Liying Tian
Qian Guo
Daidi Fu
Xiao Ma
Linjun Wang
author_facet Liying Tian
Qian Guo
Daidi Fu
Xiao Ma
Linjun Wang
author_sort Liying Tian
collection DOAJ
description <h4>Objectives</h4>Several randomized controlled trials compared adjuvant systemic chemotherapy with observation in patients with resected biliary tract cancer (BTC) have yielded inconsistent outcomes. In order to assess the efficacy of adjuvant therapy in these patients, we conducted this systematic review and meta-analysis.<h4>Methods</h4>We conducted a thorough search in various databases, which included MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials, ASCO Abstracts, ESMO Abstracts and ClinicalTrials.gov. All relevant randomized controlled trials investigating the adjuvant chemotherapy compared with observation in resected biliary tract cancer were identified. The primary outcome of interest was overall survival (OS), while secondary outcome was relapse-free survival (RFS). Statistical analyses were conducted using Review Manager 5.3. Additionally, publication bias was evaluated using Egger's test in Stata 12.0.<h4>Results</h4>A total of 5 randomized controlled trials, involving 1406 patients, were included in this analysis. Compared with observation, adjuvant chemotherapy improved RFS [HR 0.84 (0.73-0.96), p=0.01] (I2=0%, p=0.89) but not OS [HR 0.89 (0.77-1.03), p=0.12] (I2=51%, p=0.09) in the entire population after BTC resection. Subgroup analyses revealed that adjuvant chemotherapy did improve both OS [HR 0.76 (0.62-0.93), p=0.009] (I2=7%, p=0.37) and RFS [HR 0.74 (0.58-0.95), p=0.02] (I2=0%, p=0.39) in patients with lymph node positivity. Furthermore, patients receiving oral fluoropyrimidine monotherapy showed benefit from the adjuvant therapy, with longer OS [HR 0.78 (0.65-0.94), p=0.009] (I2=2%, p=0.31) and RFS [HR 0.81 (0.68-0.95), p=0.01] (I2=0%, p=0.95).<h4>Conclusions</h4>To conclude, adjuvant chemotherapy have the potential to offer advantages in patients with resected BTC. Specifically, patients demonstrating positive lymph node status have a higher likelihood of benefiting from adjuvant therapy. Our analysis supports the current standard of care of adjuvant fluoropyrimidine. However, the recommendation of oral fluoropyrimidine monotherapy as the preferred option is not definitive, as it is based on limited studies. Further validation of these outcomes is necessary by conducting extensive randomized controlled trials.
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spelling doaj-art-cd27f534ca06436ebf59212da271b9ce2025-08-20T03:09:34ZengPublic Library of Science (PLoS)PLoS ONE1932-62032025-01-01204e029558310.1371/journal.pone.0295583Adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer: A systematic review and meta-analysis of randomized controlled trials.Liying TianQian GuoDaidi FuXiao MaLinjun Wang<h4>Objectives</h4>Several randomized controlled trials compared adjuvant systemic chemotherapy with observation in patients with resected biliary tract cancer (BTC) have yielded inconsistent outcomes. In order to assess the efficacy of adjuvant therapy in these patients, we conducted this systematic review and meta-analysis.<h4>Methods</h4>We conducted a thorough search in various databases, which included MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials, ASCO Abstracts, ESMO Abstracts and ClinicalTrials.gov. All relevant randomized controlled trials investigating the adjuvant chemotherapy compared with observation in resected biliary tract cancer were identified. The primary outcome of interest was overall survival (OS), while secondary outcome was relapse-free survival (RFS). Statistical analyses were conducted using Review Manager 5.3. Additionally, publication bias was evaluated using Egger's test in Stata 12.0.<h4>Results</h4>A total of 5 randomized controlled trials, involving 1406 patients, were included in this analysis. Compared with observation, adjuvant chemotherapy improved RFS [HR 0.84 (0.73-0.96), p=0.01] (I2=0%, p=0.89) but not OS [HR 0.89 (0.77-1.03), p=0.12] (I2=51%, p=0.09) in the entire population after BTC resection. Subgroup analyses revealed that adjuvant chemotherapy did improve both OS [HR 0.76 (0.62-0.93), p=0.009] (I2=7%, p=0.37) and RFS [HR 0.74 (0.58-0.95), p=0.02] (I2=0%, p=0.39) in patients with lymph node positivity. Furthermore, patients receiving oral fluoropyrimidine monotherapy showed benefit from the adjuvant therapy, with longer OS [HR 0.78 (0.65-0.94), p=0.009] (I2=2%, p=0.31) and RFS [HR 0.81 (0.68-0.95), p=0.01] (I2=0%, p=0.95).<h4>Conclusions</h4>To conclude, adjuvant chemotherapy have the potential to offer advantages in patients with resected BTC. Specifically, patients demonstrating positive lymph node status have a higher likelihood of benefiting from adjuvant therapy. Our analysis supports the current standard of care of adjuvant fluoropyrimidine. However, the recommendation of oral fluoropyrimidine monotherapy as the preferred option is not definitive, as it is based on limited studies. Further validation of these outcomes is necessary by conducting extensive randomized controlled trials.https://doi.org/10.1371/journal.pone.0295583
spellingShingle Liying Tian
Qian Guo
Daidi Fu
Xiao Ma
Linjun Wang
Adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer: A systematic review and meta-analysis of randomized controlled trials.
PLoS ONE
title Adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer: A systematic review and meta-analysis of randomized controlled trials.
title_full Adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer: A systematic review and meta-analysis of randomized controlled trials.
title_fullStr Adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer: A systematic review and meta-analysis of randomized controlled trials.
title_full_unstemmed Adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer: A systematic review and meta-analysis of randomized controlled trials.
title_short Adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer: A systematic review and meta-analysis of randomized controlled trials.
title_sort adjuvant chemotherapy compared with observation in patients with resected biliary tract cancer a systematic review and meta analysis of randomized controlled trials
url https://doi.org/10.1371/journal.pone.0295583
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