Abstract

No prospective randomized trial has evaluated the efficacy of no treatment with palliation of pain or jaundice, definitive concurrent chemoradiotherapy (CCRT), sequential chemotherapy (CT) and radiotherapy (RT), or CT alone for unresectable intrahepatic cholangiocarcinoma (ICC). Using propensity score (PS) matching with the Mahalanobis metric (PSM-MM), we designed a nationwide, population-based, cohort study to determine the effects of different treatments on patients with unresectable ICC. We minimized confounding of different treatment outcomes among the following unresectable ICC groups of patients from the Taiwan Cancer Registry database: group 1, definitive CCRT; group 2, sequential CT and RT; group 3, no treatment (palliative therapy; relief of pain, pruritus, or jaundice); and group 4, CT alone. The matching process yielded a final cohort of 936 patients (234, 234, 234, and 234 patients in groups 1, 2, 3, and 4, respectively). In both univariate and multivariate Cox regression analyses, adjusted hazard ratios (aHRs; 95% confidence interval [CI]) derived for the definitive CCRT and sequential CT and RT cohorts compared with the CT alone cohort were 0.54 (0.46-0.64) and 0.73 (0.62-0.87), respectively. An aHR (95% CI) of 1.95 (1.51-2.51) was derived for significant independent prognostic risk factors for poor overall survival for the no treatment cohort compared with the CT alone cohort. Adding RT to CT for unresectable ICC is more beneficial instead of CT alone. Definitive CCRT is the optimal therapy for patients with unresectable ICC without distant metastasis.

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