Natural history of untreated nonsurgical hepatocellular carcinoma:: Rationale for the design and evaluation of therapeutic trials
Natural history of untreated nonsurgical hepatocellular carcinoma:: Rationale for the design and evaluation of therapeutic trials
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DOI：
10.1002/hep.510290145
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发表时间：
1999-01-01
期刊：
影响因子：
13.5
通讯作者：
Bruix, J
中科院分区：
文献类型：
--
作者：
Llovet, JM;Bustamante, J;Bruix, J
本研究分析了非手术肝细胞癌（HCC）患者的自然病史和预后因素。通过单因素和多因素分析，研究了102例前瞻性随机对照试验（RCT）中未接受治疗的HCC患者的20个变量。这些患者均不适合接受根治性治疗（手术切除、肝移植或乙醇注射），也不存在Okuda 3级或体力状态≥ 3级所反映的终末期疾病。Child-Pugh A级65例，B级34例，C级3例。他们中的大多数人表现出保留的性能状态测试（PST）（0 = 56; 1 = 38; 2 = 8例），26例为单发中位随访17个月后，79例患者死亡，1年、2年和3年生存率分别为54%、40%和28%，多变量研究确定PST（P = 0.01）、体质综合征（P = 0.04）、血管侵犯（P = 0.001）和肝外扩散（P = 0.04）是死亡率的独立预测因素。48例无不良因素的患者（0期）的1年、2年和3年生存率分别为80%、65%和50%，54例至少有一个不良参数的患者（I期）的1年、2年和3年生存率分别为29%、16%和8%。因此，阶段0将对应于中期阶段，而阶段I将代表在达到末期阶段之前的高级状态。总之，非手术肝细胞癌的预后并不是单一的严峻，可以通过评估症状和侵袭性肿瘤模式的存在来预测。治疗性试验的设计和评价应考虑到这些特点。
This study analyzed the natural history and prognostic factors of patients with nonsurgical hepatocellular carcinoma (HCC). Twenty variables from 102 cirrhotic patients with HCC who mere not treated within prospective randomized controlled trials (RCT) were investigated through uni- and multivariate analyses. None of them was suitable for radical therapies (surgical resection, liver transplantation, or ethanol injection) or presented end-stage disease as reflected by an Okuda stage 3 or a Performance Status greater than or equal to 3. Sixty-five patients were Child-Pugh A, 34 were B, and 3 were C. Most of them exhibited a preserved Performance Status Test (PST) (0 = 56; 1 = 38; 2 = 8), Tumor was solitary in 26 (less than or equal to 5 cm in 16) and multinodular/massive in 76, After a median follow-up of 17 months, 79 patients died, the 1-, 2-, and 3-year survival being 54%, 40%, and 28%, The multivariate study identified PST (P =.01), constitutional syndrome (P =.04), vascular invasion (P =.001), and extrahepatic spread (P =.04) as independent predictors for mortality. The 1-, 2-, and 3-year survival for the 48 patients without adverse factors (Stage 0) was 80%, 65%, and 50%, respectively, and 29%, 16%, and 8% in the 54 patients with at least one adverse parameter (Stage I). Therefore, Stage 0 would correspond to an intermediate stage, while Stage I would represent an advanced status, before reaching an end-stage phase. In conclusion, the outcome of nonsurgical HCC is not homogeneously grim and may be predicted by assessing the presence of symptoms and of an invasive tumoral pattern. Therapeutic trials should be designed and evaluated considering these characteristics.