Quirino Lai1, Alessandro Vitale2, Samuele Iesari1, Armin Finkenstedt3, Gianluca Mennini4, Gabriele Spoletini5, Maria Hoppe-Lotichius6, Giovanni Vennarecci7, Tommaso M Manzia8, Daniele Nicolini9, Alfonso W Avolio10, Anna Chiara Frigo11, Ivo Graziadei12, Massimo Rossi4, Emmanouil Tsochatzis5, Gerd Otto6, Giuseppe M Ettorre7, Giuseppe Tisone8, Marco Vivarelli9, Salvatore Agnes10, Umberto Cillo2, Jan Lerut1. 1. Starzl Unit of Abdominal Transplantation, St. Luc University Hospital, Catholic University of Louvain, Brussels, Belgium. 2. Department of Surgery, Oncology and Gastroenterology, University of Padua, Padua, Italy. 3. Gastroenterology and Hepatology, Department of Internal Medicine II, Innsbruck Medical University, Innsbruck, Austria. 4. Department of General Surgery and Organ Transplantation, Umberto I Hospital, Sapienza University, Rome, Italy. 5. UCL Institute for Liver and Digestive Health and Royal Free Sheila Sherlock Liver Center, Royal Free Hospital and UCL, London, United Kingdom. 6. Department of Transplantation and Hepatobiliary Surgery, University of Mainz, Mainz, Germany. 7. Division of General Surgery and Liver Transplantation, San Camillo Hospital, Rome, Italy. 8. Department of Transplant Surgery, Polyclinic Tor Vergata Foundation, Tor Vergata University, Rome, Italy. 9. Unit of Hepatobiliary Surgery and Transplantation, Azienda Ospedaliero-Universitaria "Ospedali Riuniti", Torrette Ancona, Italy. 10. Liver Unit, Department of Surgery, Agostino Gemelli Hospital, Catholic University, Rome, Italy. 11. Biostatistics Unit, University of Padua, Padua, Italy. 12. Academic Teaching Hospital, Hall, Tirol, Austria.
Abstract
The debate about the best approach to select patients with hepatocellular cancer (HCC) waiting for liver transplantation (LT) is still ongoing. This study aims to identify the best variables allowing to discriminate between "high-" and "low-benefit" patients. To do so, the concept of intention-to-treat (ITT) survival benefit of LT has been created. Data of 2,103 adult HCC patients consecutively enlisted during the period 1987-2015 were analyzed. Three rigorous statistical steps were used in order to create the ITT survival benefit of LT: the development of an ITT LT and a non-LT survival model, and the individual prediction of the ITT survival benefit of LT defined as the difference between the median ITT survival with (based on the first model) and without LT (based on the second model) calculated for each enrolled patient. Four variables (Model for End-Stage Liver Disease, alpha-fetoprotein, Milan-Criteria status, and radiological response) displayed a high effect in terms of delta benefit. According to these risk factors, four benefit groups were identified. Patients with three to four factors ("no-benefit group"; n = 405 of 2,103; 19.2%) had no benefit of LT compared to alternative treatments. Conversely, patients without any risk factor ("large-benefit group"; n = 108; 5.1%) yielded the highest benefit from LT reaching 60 months. CONCLUSION: The ITT transplant survival benefit presented here allows physicians to better select HCC patients waiting for LT. The obtained stratification may lead to an improved and more equitable method of organ allocation. Patients without benefit should be de-listed, whereas patients with large benefit ratio should be prioritized for LT. (Hepatology 2017;66:1910-1919).
The debate about the best approach to select patients with hepatocellular cancer (HCC) waiting for liver transplantation (LT) is still ongoing. This study aims to identify the best variables allowing to discriminate between "high-" and "low-benefit" patients. To do so, the concept of intention-to-treat (ITT) survival benefit of LT has been created. Data of 2,103 adult HCC patients consecutively enlisted during the period 1987-2015 were analyzed. Three rigorous statistical steps were used in order to create the ITT survival benefit of LT: the development of an ITT LT and a non-LT survival model, and the individual prediction of the ITT survival benefit of LT defined as the difference between the median ITT survival with (based on the first model) and without LT (based on the second model) calculated for each enrolled patient. Four variables (Model for End-Stage Liver Disease, alpha-fetoprotein, Milan-Criteria status, and radiological response) displayed a high effect in terms of delta benefit. According to these risk factors, four benefit groups were identified. Patients with three to four factors ("no-benefit group"; n = 405 of 2,103; 19.2%) had no benefit of LT compared to alternative treatments. Conversely, patients without any risk factor ("large-benefit group"; n = 108; 5.1%) yielded the highest benefit from LT reaching 60 months. CONCLUSION: The ITT transplant survival benefit presented here allows physicians to better select HCC patients waiting for LT. The obtained stratification may lead to an improved and more equitable method of organ allocation. Patients without benefit should be de-listed, whereas patients with large benefit ratio should be prioritized for LT. (Hepatology 2017;66:1910-1919).
Authors: Arno Kornberg; Martina Schernhammer; Jennifer Kornberg; Helmut Friess; Katharina Thrum Journal: Dig Dis Sci Date: 2018-09-27 Impact factor: 3.199