Background: Studies comparing orthotopic liver transplantation to margin negative resection for patients with small unifocal hepatocellular carcinoma have not controlled for degree of cirrhosis. Methods: The National Cancer Database was used to identify patients with preserved liver function (Model for End-stage Liver Disease score ≤12) who underwent orthotopic liver transplantation or margin negative resection for American Joint Committee on Cancer stage I hepatocellular carcinoma lesions <3 cm between 2010 and 2013. Multivariable and Cox regression adjusting for age, demographics, comorbid disease burden, Model for End-stage Liver Disease score, tumor size, and operation were used to compare overall survival between cohorts. Results: In the study, 241 (53%) patients underwent orthotopic liver transplantation. In addition, 219 (47%) underwent margin negative resection. On multivariable regression, patients having a Charlson comorbidity score ≥2 were more likely to undergo orthotopic liver transplantation, (odds ratio 1.94, P =.03). African American patients (odds ratio 0.44, P =.02), and patients of advanced age (odds ratio 0.92, P <.001) were more likely to undergo margin negative resection. Patients undergoing orthotopic liver transplantation had longer overall survival than those undergoing margin negative resection (median OS not reached versus 67.6 months, P <.001). Multivariable Cox regression identified surgical procedure as the only independent determinant of survival with margin negative resection conferring a nearly 3-fold increased risk of death (hazard ratio 2.86, P <.001). Conclusion: Orthotopic liver transplantation offers a survival advantage relative to margin negative resection for patients with small unifocal hepatocellular carcinoma and preserved liver function.
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