Boucher Eveline, Forner Alejandro, Reig Maria, Bruix Jordi
BCLC Group, Liver Unit, Hospital Clinic, IDIBAPS, Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas, University of Barcelona, Barcelona, Spain.
Liver Int. 2009 Jan;29 Suppl 1:148-58. doi: 10.1111/j.1478-3231.2008.01929.x.
Treatment of hepatocellular carcinoma has dramatically changed in the last years. The better knowledge of the molecular mechanisms responsible of tumor initiation and progression has allowed the development of molecular targeted therapies that specifically block the disrupted pathways. Among all these new agents, Sorafenib is the only one that has shown efficacy in terms of survival in advanced stage in two randomized, double-blind, controlled trials. The positive result of these two trials are the proof of the efficacy of molecular targeted therapies in hepatocellular carcinoma and opens the door to multipathway blockade and the use of these targeted therapies in the adjuvant setting. Other agents have shown promising results in phase 1-2 trials but further studies are needed to demonstrate their efficacy. In the next years, efforts should be directed to identifying genomic and proteomic profiling that will help us to assess the prognosis and to define what treatment benefits whom, ultimately giving way to personalized medicine.
在过去几年中，肝细胞癌的治疗发生了巨大变化。对肿瘤起始和进展相关分子机制的更深入了解，使得能够开发出特异性阻断失调通路的分子靶向疗法。在所有这些新药物中，索拉非尼是唯一一种在两项随机、双盲、对照试验中显示出对晚期患者生存有效的药物。这两项试验的阳性结果证明了分子靶向疗法在肝细胞癌中的疗效，并为多途径阻断以及在辅助治疗中使用这些靶向疗法打开了大门。其他药物在1-2期试验中显示出有前景的结果，但需要进一步研究来证明其疗效。在未来几年，应致力于确定基因组和蛋白质组图谱，这将有助于我们评估预后，并确定哪种治疗对谁有益，最终走向个性化医疗。
