International Kidney Cancer Coalition, Duivendrecht, The Netherlands; Department Of Nephrology and Hypertension, University Medical Center Utrecht, Regenerative Medicine Center Utrecht, Uppsalalaan, Utrecht, The Netherlands.
Lank Center for Genitourinary Oncology, Department of Medical Oncology, Dana Farber Cancer Institute, Boston, MA, USA.
Eur Urol. 2017 Dec;72(6):974-983. doi: 10.1016/j.eururo.2017.06.040. Epub 2017 Jul 16.
The European Association of Urology Renal Cell Carcinoma Guideline Panel recently conducted a systematic review of treatment options for patients with advanced non-clear-cell renal cell carcinomas (RCCs), which showed a substantial lack of evidence for management recommendations.
To improve the outcomes of patients with rare kidney cancers (RKCs), we performed a subsequent unstructured review to determine current treatment strategies and druggable pathways, involving key stakeholders with a global perspective to generate recommendations.
Based on the systematic review, literature was queried in Pubmed, Medline, and abstracts from proceedings of European Society for Medical Oncology and American Society of Clinical Oncology, in addition to consulting key opinion leaders and stakeholders. A conventional narrative review strategy was adopted to summarize the data.
The systematic review showed an absence of evidence for treating RKCs, with data only supporting sunitinib or MET inhibitors for some specific subtypes. However, a growing body of evidence implicates druggable pathways in specific RKC subtypes. To test hypotheses, the small patient numbers in each subtype require coordinated multicenter efforts. Many RKC patients are currently excluded from studies or are not analyzed using subtype-specific parameters, despite their unmet medical need.
We recognize the need for additional multicenter studies and subtype-specific analyses; however, we present management recommendations based on the data available. Web-based tools facilitating subtype-specific global registries and shared translational research resources will help generate sufficient data to formulate evidence-based recommendations for guidelines.
Patients confronted with rare kidney cancers are often treated the same way as clear-cell renal cell carcinoma patients, despite little evidence from randomized trials. Molecular characterization of tumors to stratify patients may improve outcomes. Availability of potential agents and trials remain a problem. Collaboration among medical centers is important to pool scarce data.
欧洲泌尿外科学会肾癌指南小组最近对晚期非透明细胞肾细胞癌（RCC）患者的治疗选择进行了系统评价，结果显示缺乏管理建议的大量证据。
为了改善罕见肾癌（RKC）患者的预后，我们进行了随后的非结构化综述，以确定当前的治疗策略和可用药靶点，涉及具有全球视野的关键利益相关者，以生成建议。
基于系统评价，在 PubMed、Medline 以及欧洲肿瘤内科学会和美国临床肿瘤学会会议摘要中检索文献，此外还咨询了主要意见领袖和利益相关者。采用传统的叙述性综述策略来总结数据。
系统评价显示，缺乏治疗 RKC 的证据，只有针对某些特定亚型的数据支持舒尼替尼或 MET 抑制剂。然而，越来越多的证据表明特定 RKC 亚型存在可用药靶点。为了验证假说，每个亚型中的患者数量较少，需要协调多中心的努力。尽管存在未满足的医疗需求，但许多 RKC 患者目前被排除在研究之外，或者没有使用亚型特异性参数进行分析。
我们认识到需要更多的多中心研究和亚型特异性分析；然而，我们根据现有数据提出了管理建议。基于网络的工具可以促进特定亚型的全球注册和共享转化研究资源，有助于生成足够的数据，为指南制定基于证据的建议。
患有罕见肾癌的患者通常与透明细胞肾细胞癌患者采用相同的治疗方法，尽管随机试验的证据很少。对肿瘤进行分子特征分析以分层患者可能会改善预后。潜在药物和试验的可用性仍然是一个问题。医疗机构之间的合作对于汇集稀缺数据很重要。
