Yeonghak Bang1, Changhoon Yoo1, Mario Scartozzi2, Stefano Cascinu3,4, Andrea Casadei-Gardini5,6, Sara Lonardi7,8, Hyung-Don Kim1, Caterina Vivaldi9, Margherita Rimini10, Giovanni Luca Frassineti11, Sook Ryun Park1, Mario Domenico Rizzato8, Min-Hee Ryu1, Francesca Salani9, Ilario Giovanni Rapposelli11, Baek-Yeol Ryoo1, Vittorina Zagonel8, Valentina Massa9, Martina Valgiusti11, Valentina Burgio3. 1. Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea. 2. Department of Medical Oncology, University Hospital of Cagliari, Cagliari, Italy. 3. Department of Medical Oncology, San Rafaele Scientific Institute IRCCS, Milan, Italy. 4. Vita-Salute San Raffaele University, Milan, Italy. 5. Department of Medical Oncology, San Rafaele Scientific Institute IRCCS, Milan, Italy. casadeigardini@gmail.com. 6. Vita-Salute San Raffaele University, Milan, Italy. casadeigardini@gmail.com. 7. Early Phase Clinical Trial Unit, Department of Oncology, Veneto Institute of Oncology IOV-IRCCS, Padua, Italy. 8. Medical Oncology Unit 1, Department of Oncology, Veneto Institute of Oncology IOV- IRCCS, Padua, Italy. 9. Department of Translational Research and New Surgical and Medical Technologies, University of Pisa, Via Savi 6, 56126, Pisa, Italy. 10. Department of Oncology and Hematology, Division of Oncology, University Hospital of Modena, Via del Pozzo 71, Modena, Italy. 11. Department of Medical Oncology, Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IRST) IRCCS, Meldola, Italy.
Abstract
BACKGROUND: Regorafenib has been shown to improve clinical outcomes compared to placebo, becoming a standard second-line therapy for sorafenib-progressed and -tolerated hepatocellular carcinoma (HCC) patients. OBJECTIVE: We performed a multicentre, retrospective study in Italy and Korea to evaluate the effectiveness of the treatment sequence sorafenib-regorafenib compared with sorafenib and physician's choice in a real-life setting. PATIENTS AND METHODS: A propensity score model was developed to control the results for baseline variable imbalances between the arm treated with sorafenib and regorafenib (S-R) and the arm treated with sorafenib and physician's choice (S-P). Survival analysis was conducted on the matched population. RESULTS: After the application of propensity score matching, we analysed 99 patients in the arm treated with S-R and 99 patients in the arm treated with S-P. For the S-R group, the median overall survival was 22.2 months (95% CI 17.1-27.4), compared to 17.9 months (95% CI 15.1-50.0) for the S-P group. The results of the univariate analysis showed a 31% reduction of death risk for patients treated with S-R (p = 0.0382) compared to patients treated with S-P. Interaction tests highlighted the predictive role of alpha-fetoprotein (AFP), neutrophil-to-lymphocyte ratio (NLR), and extrahepatic spread. CONCLUSION: This study provides additional proof of the superiority of the S-R treatment over the S-P treatment approach in advanced HCC patients from a real-life setting.
BACKGROUND:Regorafenib has been shown to improve clinical outcomes compared to placebo, becoming a standard second-line therapy for sorafenib-progressed and -tolerated hepatocellular carcinoma (HCC) patients. OBJECTIVE: We performed a multicentre, retrospective study in Italy and Korea to evaluate the effectiveness of the treatment sequence sorafenib-regorafenib compared with sorafenib and physician's choice in a real-life setting. PATIENTS AND METHODS: A propensity score model was developed to control the results for baseline variable imbalances between the arm treated with sorafenib and regorafenib (S-R) and the arm treated with sorafenib and physician's choice (S-P). Survival analysis was conducted on the matched population. RESULTS: After the application of propensity score matching, we analysed 99 patients in the arm treated with S-R and 99 patients in the arm treated with S-P. For the S-R group, the median overall survival was 22.2 months (95% CI 17.1-27.4), compared to 17.9 months (95% CI 15.1-50.0) for the S-P group. The results of the univariate analysis showed a 31% reduction of death risk for patients treated with S-R (p = 0.0382) compared to patients treated with S-P. Interaction tests highlighted the predictive role of alpha-fetoprotein (AFP), neutrophil-to-lymphocyte ratio (NLR), and extrahepatic spread. CONCLUSION: This study provides additional proof of the superiority of the S-R treatment over the S-P treatment approach in advanced HCCpatients from a real-life setting.