The role of neoadjuvant-chemotherapy (NADJ) in cholangiocarcinoma is unknown. The purpose of this study is to evaluate whether NADJ improves overall survival (OS) in cholangiocarcinoma compared to adjuvant-chemotherapy (ADJ).
Using the National Cancer Database, we identified patients who underwent surgery and chemotherapy for stage I-III cholangiocarcinoma between 2006 and 2014. Patients with metastatic disease at diagnosis or unknown chemotherapy sequence with surgery were excluded. Propensity score for NADJ was calculated by multivariate logistic regression method. Matching with patients who received ADJ was then performed at the ratio of 1:3 using nearest neighbor method with a caliper width of 0.2. Covariates included in matching were: age at diagnosis, sex, race, insurance status, Charlson score, year of diagnosis, location of tumor, tumor grade, clinical stage, and use of radiation.
1450 patients met our inclusion criteria, 299 (20.6%) received NADJ while 1151 (79.3%) received ADJ. The median age at diagnosis was 63 years. Factors associated with higher (p < 0.05) use of NADJ compared to ADJ were: ages <54 (35% vs. 23%), white race (91% vs. 86%), year of diagnosis 2012-2014 (48% vs. 40%), intrahepatic tumor location (74% vs. 54%), clinical stage I (46% vs. 38%), and unknown grade of tumor (36% vs. 14%). 279 patients in NADJ group were matched to 698 patients in ADJ group, with resulting standardized mean difference of < 0.1 for all covariates. In the matched cohort, patients who received NADJ had a significantly better OS compared to those who received ADJ (HR: 0.79; 95% CI: 0.65 – 0.96, p = 0.01). The 1- and 5-year OS was 85.9% and 42.1% respectively for NADJ, while it was 85.0% and 32.7% respectively for ADJ.
In this large national database study, NADJ compared to ADJ improved OS in a select group of patients with cholangiocarcinoma.
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All authors have declared no conflicts of interest.