Clinical Trials Unit, National Cancer Institute of Naples, Naples, Italy.
Ann Oncol. 2013 Apr;24 Suppl 2(Suppl 2):ii6-14. doi: 10.1093/annonc/mdt052.
Despite the fact that the hepatocellular carcinoma (HCC) represents a major health problem, very few interventions are available for this disease, and only sorafenib is approved for the treatment of advanced disease. Of note, only very few interventions have been thoroughly evaluated over time for HCC patients compared with several hundreds in other, equally highly lethal, tumours. Additionally, clinical trials in HCC have often been questioned for poor design and methodological issues. As a consequence, a gap between what is measured in clinical trials and what clinicians have to face in daily practice often occurs. As a result of this scenario, even the most recent guidelines for treatment of HCC patients use low strength evidence to make recommendations. In this review, we will discuss some of the potential methodological issues hindering a rational development of new treatments for HCC patients.
尽管肝细胞癌 (HCC) 是一个主要的健康问题，但针对这种疾病的干预措施非常有限，只有索拉非尼被批准用于治疗晚期疾病。值得注意的是，与其他同样致命的肿瘤相比，只有极少数干预措施经过时间的考验，在 HCC 患者中得到了彻底的评估。此外，HCC 的临床试验经常因设计和方法学问题而受到质疑。因此，临床试验中测量的结果与临床医生在日常实践中面临的结果之间经常存在差距。由于这种情况，即使是治疗 HCC 患者的最新指南也使用低强度的证据来提出建议。在这篇综述中，我们将讨论一些可能阻碍 HCC 患者新治疗方法合理发展的方法学问题。
