Prescrire Int. 2014 Jan;23(145):8-11.
Patients with metastatic colorectal cancer who have exhausted their therapeutic options have a life expectancy of only a few months. Treatment is then based on tailored supportive care, without cytotoxic drugs. Regorafenib, an inhibitor of multiple protein kinases, has been authorised in the European Union for the treatment of patients with metastatic colorectal cancer who have no other therapeutic options. Clinical evaluation is based on a double-blind, randomised, placebo-controlled trial in patients who had already received multiple lines of cytotoxic chemotherapy but who remained in good overall condition. Adding regorafenib to best supportive care led to median overall survival, albeit of only a few weeks (6.4 months with regorafenib versus 5 months with placebo). Regorafenib has many adverse effects, including hepatic, cardiovascular, cutaneous, gastrointestinal, thyroid, neurological and haematological disorders, as well as infections and bleeding. These adverse effects are severe in about 40% of patients. Some patients died from these adverse effects, notably liver damage and bleeding. In practice, according to the only available trial, regorafenib appears to prolong overall survival by a few weeks in some cases, but at a cost of serious adverse effects in about 40% of patients, including premature death. Pending further evaluation, it is preferable to recommend best supportive care.
转移性结直肠癌患者若已用尽治疗方案，预期寿命仅几个月。此时治疗基于个体化的支持性护理，不使用细胞毒性药物。瑞戈非尼是一种多蛋白激酶抑制剂，已在欧盟获批用于治疗无其他治疗选择的转移性结直肠癌患者。临床评估基于一项双盲、随机、安慰剂对照试验，试验对象为已接受多线细胞毒性化疗但总体状况良好的患者。在最佳支持性护理基础上加用瑞戈非尼可延长中位总生存期，尽管仅延长几周（瑞戈非尼组为6.4个月，安慰剂组为5个月）。瑞戈非尼有许多不良反应，包括肝脏、心血管、皮肤、胃肠道、甲状腺、神经和血液系统疾病，以及感染和出血。约40%的患者不良反应严重。一些患者死于这些不良反应，尤其是肝损伤和出血。实际上，根据唯一可用的试验，瑞戈非尼在某些情况下似乎可将总生存期延长几周，但代价是约40%的患者出现严重不良反应，包括过早死亡。在进一步评估之前，最好推荐最佳支持性护理。
