Digestive Disease Research Centre, Tehran University of Medical Science, Tehran, Iran.
Hepatology. 2011 Dec;54(6):1998-2004. doi: 10.1002/hep.24581.
Screening for hepatocellular carcinoma (HCC) is commonly practiced and recommended in published guidelines, but evidence for its efficacy has been controversial. We tested the feasibility of conducting a randomized controlled trial (RCT) of HCC surveillance in patients with cirrhosis and followed up those offered screening to detect clinical outcomes. Participation was offered to patients with cirrhosis attending liver clinics at three university hospitals. Following discussion, patients received a decision aid (DA) that outlined the risks and benefits of surveillance. The proposed screening program comprised ultrasonography 6-monthly and serum alpha-fetoprotein every 3 months. We envisaged five groups of patients: those who agreed to randomization, those choosing nonrandomized screening, those wanting continuation of usual care, those who were undecided, and those refusing participation. Among 205 patients, 204 (99.5%) declined randomization. Of these, 181 (88%) elected for a nonrandomized screening program, 10% chose usual care (which typically included ad hoc screening), and two were undecided. Among 176 patients fluent in English communication skills, 160 (91%) preferred nonrandomized screening compared with 22/29 (76%) patients needing an interpreter (P < 0.026). Of 173 patients in nonrandomized screening followed up for a mean 13.5 ± 6.04 months, three developed HCC, two died from nonliver-related causes, and one underwent liver transplantation for liver failure. Eighteen of 21 patients in "usual care" received ad hoc screening. A simultaneous survey on the quality of the DA showed that the majority of participants believed that the information provided was unbiased.
Although an RCT is theoretically ideal for determining the efficacy, efficiency, and cost-effectiveness of HCC screening, informed patients prefer surveillance. A randomized study of HCC screening is not feasible when informed consent is imparted.
肝细胞癌（HCC）的筛查在已发表的指南中被广泛实践和推荐，但关于其疗效的证据一直存在争议。我们测试了在肝硬化患者中进行 HCC 监测的随机对照试验（RCT）的可行性，并对接受筛查以检测临床结果的患者进行了随访。该研究在三家大学医院的肝脏诊所为肝硬化患者提供了参与机会。讨论后，患者会收到一份决策辅助工具（DA），其中概述了监测的风险和收益。拟议的筛查方案包括每 6 个月进行一次超声检查和每 3 个月进行一次血清甲胎蛋白检查。我们设想了五组患者：同意随机分组的患者、选择非随机筛查的患者、希望继续接受常规治疗的患者、犹豫不决的患者和拒绝参与的患者。在 205 名患者中，有 204 名（99.5%）患者拒绝随机分组。其中，181 名（88%）患者选择了非随机筛查方案，10%选择了常规治疗（通常包括临时筛查），还有 2 名患者犹豫不决。在 176 名英语沟通能力流利的患者中，有 160 名（91%）患者更喜欢非随机筛查，而需要口译员的 29 名患者中有 22 名（76%）患者（P < 0.026）。在进行了平均 13.5 ± 6.04 个月随访的 173 名非随机筛查患者中，有 3 名患者发生 HCC，2 名患者死于非肝脏相关原因，1 名患者因肝功能衰竭接受肝移植。在“常规治疗”的 21 名患者中，有 18 名患者接受了临时筛查。同时进行的关于 DA 质量的调查显示，大多数参与者认为提供的信息是无偏见的。
虽然 RCT 在理论上是确定 HCC 筛查的疗效、效率和成本效益的理想方法，但知情患者更喜欢监测。当给予知情同意时，进行 HCC 筛查的随机研究是不可行的。
