Comparative efficacy of sorafenib versus best supportive care in recurrent hepatocellular carcinoma after liver transplantation: A case-control study

Background & Aims: The efficacy of sorafenib in the post-liver transplantation (LT) setting has been scarcely studied. The aim of this study was to evaluate the efficacy of sorafenib, compared to best supportive care (BSC), in two cohorts of patients which presented with hepatocellular carcinoma (HCC) recurrence after LT. Methods: Data from patients who developed presentation or progression of HCC recurrence after LT not amenable to surgical/locoregional treatments (untreatable presentation/progression, UP) were retrieved. The cohort of patients receiving sorafenib starting from 2007 was compared to that of patients receiving BSC in the previous era. Disease outcome was investigated in terms of survival from recurrence or from UP by means of univariate and multivariate Cox regression models with event times left-truncated at the date of UP. Results: Of a total of 39 patients, 24 received BSC and 15 sorafenib. The two groups were well matched at baseline, with time-related imbalances regarding mTOR-based immunosuppression and median time from LT to recurrence, significantly higher in the sorafenib group. Patients' outcome in the sorafenib group was significantly improved (median survival from recurrence 21.3 vs.11.8 months, HR = 5.2, p = 0.0009; median survival from UP 10.6 vs. 2.2 months, HR = 21.1, p <0.0001). The only factor associated with survival after HCC recurrence in multivariate analysis was treatment with sorafenib (HR = 4.0; p = 0.0325). No severe adverse event was registered in this post-LT setting. Conclusions: Although the use of historical controls weakens final interpretation, sorafenib seems to be associated with an acceptable safety profile and benefit in survival in HCC patients suffering recurrence after LT.