Department of General Surgery and Organ Transplantation, Hepatobiliary Surgery and Liver Transplantation Unit, University Hospital of Padua, Padua, Italy.
Liver and Multi-Organ Transplantation Unit, St. Orsola Hospital, Alma Mater Studiorum - University of Bologna, Bologna, Italy.
J Hepatol. 2014 Jun;60(6):1165-71. doi: 10.1016/j.jhep.2014.01.022. Epub 2014 Feb 6.
BACKGROUND & AIMS: Number-needed-to-treat is used in assessing the effectiveness of a health-care intervention, and reports the number of patients who need to be treated to prevent one additional bad outcome. Although largely used in medical literature, there are no studies measuring the benefit of liver transplantation (LT) over hepatic resection (HR) for hepatocellular carcinoma (HCC) in terms of "Number of patients needed to transplant (NTT)."
Child-Turcotte-Pugh (CTP) Classes B-C, very large (>10 cm) and multi-nodular (>2 nodules) tumours, macroscopic vascular invasion and extra-hepatic metastases.
1028 HCC cirrhotic patients from one Eastern (n=441) and two Western (n=587) surgical units. Patient survival observed after HR by proportional hazard regression model was compared to that predicted after LT by the Metroticket calculator. The benefit obtainable from LT compared to resection was analysed in relationship with number of nodules (modelled as ordinal variable: single vs. oligonodular), size of largest nodule (modelled as a continuous variable), presence of microscopic vascular invasion (MVI), and time horizon from surgery (5-year vs. 10-year).
330 patients were beyond the Milan criteria (32%) and 597 (58%) had MVI. The prevalence of MVI was 52% in patients within Milan criteria and 71% in those beyond (p<0.0001). In the 5-year transplant benefit analysis, nodule size and HCC number were positive predictors of transplant benefit, while MVI had a strong negative impact on NTT. Transplantation performed as an effective therapy (NTT <5) only in oligonodular HCC with largest diameter >3cm (beyond conventional LT criteria) when MVI was absent. The 10-year scenario increased drastically the transplant benefit in all subgroups of resectable patients, and LT became an effective therapy (NTT <5) for all patients without MVI whenever tumor extension and for oligonodular HCC with MVI within conventional LT criteria.
Based on NTT analysis, the adopted time horizon (5-year vs. 10-year scenario) is the main factor influencing the benefit of LT in patients with resectable HCC and Child A cirrhosis.
需要治疗的人数（number-needed-to-treat，NNT）用于评估医疗干预措施的效果，它表示预防额外不良结局所需治疗的患者数量。尽管在医学文献中广泛使用，但尚无研究根据“需要移植的患者数量（number-needed-to-transplant，NNT）”来衡量肝移植（LT）与肝切除术（HR）治疗肝细胞癌（HCC）的获益。
Child-Turcotte-Pugh（CTP）分级 B-C、肿瘤非常大（>10cm）和多结节（>2 个结节）、肉眼血管侵犯和肝外转移。
来自东部（n=441）和西部两个外科单位的 1028 例 HCC 肝硬化患者。通过比例风险回归模型观察 HR 后患者的生存情况，并与 Metroticket 计算器预测的 LT 后生存情况进行比较。分析 LT 与切除相比的获益与结节数量（建模为有序变量：单发 vs. 寡结节）、最大结节大小（建模为连续变量）、微血管侵犯（MVI）的存在以及手术时间（5 年 vs. 10 年）的关系。
330 例患者超出米兰标准（32%），597 例（58%）有 MVI。在符合米兰标准的患者中，MVI 的患病率为 52%，在不符合米兰标准的患者中为 71%（p<0.0001）。在 5 年 LT 获益分析中，结节大小和 HCC 数量是 LT 获益的正预测因素，而 MVI 对 NTT 有强烈的负面影响。只有当 MVI 不存在时，最大直径>3cm（超出传统 LT 标准）的寡结节 HCC 才是有效的移植治疗（NTT<5）。10 年方案显著增加了所有可切除患者亚组的 LT 获益，并且只要肿瘤范围扩大且存在 MVI，LT 就成为所有无 MVI 患者的有效治疗方法（NTT<5），无论 HCC 为多结节还是单结节。
根据 NTT 分析，所采用的时间范围（5 年与 10 年方案）是影响可切除 HCC 和 Child A 肝硬化患者 LT 获益的主要因素。
