Context

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.

Objective

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.

Evidence acquisition

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.

Evidence synthesis

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.

Conclusions

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.

Patient summary

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.

中文翻译：

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罕见肾脏癌的治疗建议

语境

欧洲泌尿外科协会肾细胞癌指南小组最近对晚期非透明细胞肾细胞癌（RCC）患者的治疗方案进行了系统的回顾，结果表明，缺乏足够的管理建议证据。

客观的

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审查，以确定当前的治疗策略和可药物化途径，涉及具有全球视野的关键利益相关者，以提出建议。

取证

在系统评价的基础上，除了咨询关键意见领袖和利益相关者外，还从Pubmed，Medline和欧洲医学肿瘤学会和美国临床肿瘤学会的论文摘要中查询了文献。采用常规叙事回顾策略对数据进行汇总。

证据综合

系统评价显示缺乏治疗RKC的证据，数据仅支持某些特定亚型的舒尼替尼或MET抑制剂。然而，越来越多的证据表明在特定的RKC亚型中可能存在药物通路。为了检验假设，每种亚型的患者人数少，需要多中心协作。尽管他们的医疗需求未得到满足，但许多RKC患者目前仍被排除在研究之外或未使用亚型特异性参数进行分析。

结论

我们认识到需要更多的多中心研究和特定于亚型的分析；但是，我们会根据可用数据提出管理建议。基于Web的工具可促进特定于子类型的全球注册表和共享的翻译研究资源，将有助于生成足够的数据，以制定基于证据的指南建议。

病人总结

尽管随机试验的证据很少，但面对罕见肾癌的患者通常会与透明细胞肾细胞癌患者以相同的方式进行治疗。对患者进行分层的肿瘤分子特征可能会改善预后。潜在代理商和试验的可用性仍然是一个问题。医疗中心之间的合作对于汇总稀缺的数据非常重要。