El-Serag Hashem B, Mallat Damien B, Rabeneck Linda
Sections of Gastroenterology, Houston Veterans Affairs Medical Center, Baylor College of Medicine, Houston, TX 77030, USA.
J Clin Gastroenterol. 2005 Feb;39(2):152-9.
The widespread use of liver imaging in patients with cirrhosis results in the discovery of small (<3 cm) nodules. Although the subsequent management of these patients is variable, it is generally focused on the diagnosis and treatment of hepatocellular carcinoma (HCC). We aimed to compare the 3-year survival associated with several competing strategies used in the management of patients with compensated liver cirrhosis in whom a single small liver lesion is detected during surveillance. We constructed a decision analysis model using a decision tree and Markov model. We assumed that all patients undergo an initial "diagnostic phase" consisting of an imaging study and serum alpha-fetoprotein (AFP). Patients with a "positive initial diagnostic phase" for HCC are referred for either imaging-guided biopsy (IGB) or surgical resection or orthotopic liver transplantation (OLT) without preceding IGB. IGB, if positive for HCC, was followed by OLT, surgical resection, or local therapy. Patients with a "negative initial diagnostic phase" undergo either repeat diagnostic testing (imaging, AFP) every 4 months or are referred for either OLT, surgical resection, or IGB followed by interventions. Probability assumptions were estimated from the published literature. The outcomes compared were 3-year overall survival and recurrence-free survival. When the initial diagnostic phase is positive for HCC, OLT it is associated with the longest survival. In the sensitivity analysis, when the 3-year overall survival for patients referred to OLT is <54%, surgical resection or IGB preceding therapy become more favorable strategies. This 3-year overall survival (<54%) associated with OLT is reached after a waiting time of 4 months on the transplant list, if a 4% monthly dropout rate is assumed. When the initial diagnostic phase is negative for HCC, then performing IGB, before proceeding to therapeutic intervention, is associated with the longest 3-year overall survival. If the IGB is positive, subsequent OLT is associated with the longest survival. The higher the predictive value of the initial diagnostic phase for HCC, the more favorable is OLT (for the "positive results" arm), and follow-up testing (for the "negative results" arm). In conclusion, given a high pretest likelihood of HCC in a single liver nodule detected during surveillance in patients with cirrhosis, IGB may not be required in the presence of a positive noninvasive diagnostic testing. The long waiting time prior to OLT limits its advantage over surgical resection in the treatment of patients with early HCC.
在肝硬化患者中广泛使用肝脏成像检查会发现小(<3 cm)结节。尽管对这些患者的后续管理各不相同,但总体上侧重于肝细胞癌(HCC)的诊断和治疗。我们旨在比较在监测期间发现单个小肝脏病变的代偿期肝硬化患者管理中使用的几种相互竞争策略的3年生存率。我们使用决策树和马尔可夫模型构建了一个决策分析模型。我们假设所有患者都要经历一个由影像学检查和血清甲胎蛋白(AFP)组成的初始“诊断阶段”。HCC“初始诊断阶段呈阳性”的患者被转诊进行影像引导活检(IGB)或手术切除或原位肝移植(OLT),而无需先行IGB。如果IGB对HCC呈阳性,则继以OLT、手术切除或局部治疗。“初始诊断阶段呈阴性”的患者每4个月进行一次重复诊断检测(影像学检查、AFP),或者被转诊进行OLT、手术切除或IGB继以干预措施。概率假设是根据已发表的文献估计的。比较的结果是3年总生存率和无复发生存率。当HCC的初始诊断阶段呈阳性时,OLT与最长生存期相关。在敏感性分析中,当转诊进行OLT的患者的3年总生存率<54%时,手术切除或治疗前的IGB成为更有利的策略。如果假设每月退出率为4%,那么在移植名单上等待4个月后,与OLT相关的这一3年总生存率(<54%)就会达到。当HCC的初始诊断阶段呈阴性时,在进行治疗性干预之前进行IGB与最长的3年总生存率相关。如果IGB呈阳性,随后的OLT与最长生存期相关。HCC初始诊断阶段的预测价值越高,OLT(对于“阳性结果”组)和后续检测(对于“阴性结果”组)就越有利。总之,鉴于在肝硬化患者监测期间发现的单个肝结节中HCC的预测试验可能性较高,在非侵入性诊断检测呈阳性的情况下可能不需要IGB。OLT之前的漫长等待时间限制了其在早期HCC患者治疗中相对于手术切除的优势。