Starzl Unit of Abdominal Transplantation, St. Luc University Hospital, Catholic University of Louvain, Brussels, Belgium.
Department of Surgery, Oncology and Gastroenterology, University of Padua, Padua, Italy.
Hepatology. 2017 Dec;66(6):1910-1919. doi: 10.1002/hep.29342. Epub 2017 Nov 6.
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.
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).
本研究旨在确定最佳变量,以区分“高获益”和“低获益”患者。方法:创建肝移植(LT)意向治疗(ITT)生存获益的概念。对 1987 年至 2015 年间连续登记的 2103 例成人 HCC 患者的数据进行分析。采用 3 个严格的统计步骤来创建 LT 的 ITT 生存获益:开发 ITT LT 和非 LT 生存模型,并为每位入组患者计算基于第一个模型的 ITT 生存与不进行 LT(基于第二个模型)的 ITT 生存的差异,定义为 ITT LT 生存获益。4 个变量(终末期肝病模型、甲胎蛋白、米兰标准状态和影像学反应)在 delta 获益方面显示出高效应。根据这些危险因素,确定了 4 个获益组。有 3-4 个危险因素的患者(“无获益组”;2103 例患者中有 405 例,占 19.2%)与替代治疗相比,LT 无获益。相反,没有任何危险因素的患者(“大获益组”;n=108;5.1%)从 LT 中获得的获益最高,达到 60 个月。
本文提出的 ITT 移植生存获益可帮助医生更好地选择等待 LT 的 HCC 患者。获得的分层可能导致器官分配方法的改进和更公平。无获益患者应被取消 LT 等待名单,而获益比例大的患者应优先考虑 LT。