Survival Benefit Relative to Treatment Modalities Among Patients with Very Early Intrahepatic Cholangiocarcinoma: an Analysis of the National Cancer Database

Background Liver transplantation (LT) has been considered a potential curative treatment for patients with very early intrahepatic cholangiocarcinoma (ICC) and cirrhosis, yet the survival benefit of LT has not been well defined. This study aimed to compare the long-term survival outcomes of patients who underwent LT with that of individuals who received resection and non-curative intent treatment (non-CIT).Methods Patients who underwent LT, hepatectomy, and non-CIT between 2004 and 2018 were included in the National Cancer Database. Survival benefits of LT over resection and non-CIT were analyzed relative to overall survival (OS).Results Among 863 patients, 54 (6.3%) underwent LT, while 342 (39.6%) underwent surgical resection, and 467 (54.1%) received non-CIT, respectively. While the rates of non-CIT increased over time, the percentages of LT remained consistent during the study period. LT patients had similar 5-year OS to individuals who underwent resection (referent, resection: LT, HR 0.95, 95%CI 0.84-1.58, p=0.84). In contrast, 5-year OS was better among patients who underwent LT versus individuals who had non-CIT after controlling other variables using propensity score overlapping weighting (5-year OS, LT 57.1% vs. LR 25.8%, p<0.001).Conclusions The outcomes of very early ICC patients who underwent LT were similar to individuals who underwent hepatectomy, but better than patients treated with non-CIT. LT should be may be a consideration as a treatment option for patients with early stage ICC who are unsuitable candidates for resection.

Automated summary

This study compared the long-term survival outcomes of liver transplantation (LT) with surgical resection and non-curative intent treatment (non-CIT) for patients with very early intrahepatic cholangiocarcinoma (ICC) and cirrhosis. The results showed that the 5-year overall survival rate of LT patients was similar to those who underwent resection, but better than patients treated with non-CIT. Therefore, LT may be a treatment option for patients with early stage ICC who are unsuitable candidates for resection.