Bellmunt Joaquim, Guix Marta
University Hospital del Mar, Barcelona, Spain.
BJU Int. 2009 Mar;103(5):572-7. doi: 10.1111/j.1464-410X.2008.08336.x.
There are now five targeted agents, i.e. sorafenib, sunitinib, temsirolimus, bevacizumab (in combination with interferon) and everolimus, that have been shown to improve the outcome in patients with metastatic clear cell renal cell carcinoma (mRCC), in randomized controlled trials (RCTs). Compared with the period when cytokines were the only systemic intervention known to have any activity, decisions on medical management are now complex. Clinicians must seek to adjust therapy to the circumstances of the individual patient, and consider the sequencing of agents. In this context, several expert groups have sought to provide treatment guidelines. As in other diseases, guidelines for mRCC seek to establish evidence-based recommendations for best clinical practice and to encourage their widespread use. Data from phase III trials (level 1 evidence) are an essential element in this process, and guidelines need continual updating in the light of new findings. However, there are inevitably questions that large RCTs have not directly addressed. This is the case for major subgroups of the mRCC population, e.g. the elderly and those with comorbidities. In these circumstances, less well-controlled sources of data, and clinical experience, have a role to play. Certain guidelines (although not all) acknowledge the contribution that such sources of evidence can make.
目前已有五种靶向药物，即索拉非尼、舒尼替尼、替西罗莫司、贝伐单抗（与干扰素联合使用）和依维莫司，在随机对照试验（RCT）中已证明可改善转移性透明细胞肾细胞癌（mRCC）患者的预后。与细胞因子是唯一已知有任何活性的全身干预措施的时期相比，现在的药物治疗决策变得复杂。临床医生必须根据个体患者的情况调整治疗方案，并考虑药物的使用顺序。在这种情况下，几个专家组试图提供治疗指南。与其他疾病一样，mRCC的指南旨在为最佳临床实践建立基于证据的建议，并鼓励广泛使用。来自III期试验的数据（1级证据）是这一过程的重要组成部分，并且指南需要根据新发现不断更新。然而，不可避免地存在一些大型RCT尚未直接解决的问题。mRCC人群的主要亚组，如老年人和患有合并症的患者就是这种情况。在这种情况下，控制较差的数据来源和临床经验也能发挥作用。某些指南（并非全部）承认这些证据来源可以做出的贡献。
