Department of General Surgery, Singapore General Hospital, Outram Road, 169608 Singapore.
Br J Cancer. 2011 Sep 27;105(7):945-52. doi: 10.1038/bjc.2011.333. Epub 2011 Aug 23.
Hepatocellular carcinoma (HCC) is the third leading cause of cancer deaths worldwide. We tested megestrol acetate (MA) against placebo in the treatment of advanced HCC.
From 2002 through 2007, this randomised double-blind trial enrolled 204 patients with treatment-naive advanced HCC (Eastern Cooperative Oncology Group (ECOG) performance rating of 0-3) from specialist care centres in six Asia-Pacific nations. Patients received placebo or MA (320 mg day(-1)). End points were overall survival (OS) and quality of life.
An adverse but not statistically significant difference in OS was found for MA vs placebo: median values 1.88 and 2.14 months, respectively (hazard ratio (HR)=1.25, 95% CI=0.92-1.71, P=0.16). However, OS was similar among patients of good functional status (Child-Pugh A and ECOG 0, 1 or 2) (44.3%) in both treatment groups, with the adverse effect of MA confined to those of poor status. Megestrol acetate patients had a worse global health status (not statistically significant) but reduced levels of appetite loss and nausea/vomiting.
Megestrol acetate has no role in prolonging OS in advanced treatment-naive HCC. Overall survival with placebo differed markedly from that in similar trials conducted elsewhere, suggesting therapeutic outcomes may be strongly dependent on ECOG status and Child-Pugh score.
肝细胞癌（HCC）是全球癌症死亡的第三大主要原因。我们测试了醋酸甲地孕酮（MA）与安慰剂在治疗晚期 HCC 中的疗效。
2002 年至 2007 年，这项随机双盲试验纳入了来自六个亚太国家的专科治疗中心的 204 例未经治疗的晚期 HCC 患者（东部肿瘤协作组（ECOG）表现评分为 0-3）。患者接受安慰剂或 MA（320mg/天）治疗。终点为总生存期（OS）和生活质量。
MA 与安慰剂相比，OS 出现不良但无统计学意义的差异：中位数分别为 1.88 个月和 2.14 个月（风险比（HR）=1.25，95%CI=0.92-1.71，P=0.16）。然而，在功能状态良好的患者（Child-Pugh A 和 ECOG 0、1 或 2）中，两组的 OS 相似（44.3%），MA 的不良影响仅限于状态较差的患者。MA 患者的总体健康状况较差（无统计学意义），但食欲丧失和恶心/呕吐的发生率较低。
醋酸甲地孕酮在延长晚期 HCC 患者的 OS 方面没有作用。安慰剂组的总生存期与其他地方进行的类似试验明显不同，这表明治疗结果可能强烈依赖于 ECOG 状态和 Child-Pugh 评分。
