Department of Pediatrics, Wake Forest University School of Medicine, Winston-Salem, North Carolina 27157, USA.
Curr Opin Oncol. 2010 May;22(3):268-73. doi: 10.1097/CCO.0b013e32833841a1.
To review the 2008-2009 literature on pediatric genitourinary tumors and highlight the most significant publications.
New techniques such as gene expression profiling, PET, nephron-sparing surgery, and stem cell transplantation are being incorporated into contemporary treatments for pediatric patients with genitourinary tumors. The WTX gene is the most commonly mutated gene in Wilms tumor, and its product enhances Wilms tumor gene 1-mediated transcription. Germline WTX mutations cause an X-linked sclerosing bone dysplasia but do not appear to predispose to Wilms tumor formation. Protocadherin gene clusters on chromosome 5q31 may act as tumor suppressors. In rhabdomyosarcoma, ILK and platelet-derived growth factor receptor-A join the paired box gene 7 and 3-forkhead box O1 fusions as potential therapeutic targets, and muscle-specific microRNAs offer promise as adjuvant therapy. Despite the high cure rate of Wilms tumor, long-term survivors remain at risk of death from various causes.
In general, the prognosis for patients with pediatric genitourinary tumors is favorable. The elucidation of the molecular abnormalities in these tumors is determining risk stratification, treatment strategies, and candidates for new drug development.
回顾 2008-2009 年小儿泌尿生殖系统肿瘤的文献，重点介绍最具意义的出版物。
新的技术，如基因表达谱分析、正电子发射断层扫描、保肾手术和干细胞移植，正被纳入小儿泌尿生殖系统肿瘤患者的当代治疗方法中。WTX 基因是 Wilms 瘤中最常见的突变基因，其产物增强 Wilms 瘤基因 1 介导的转录。胚系 WTX 突变导致 X 连锁硬化性骨发育不良，但似乎不会导致 Wilms 瘤形成。位于 5q31 染色体上的原钙黏蛋白基因簇可能作为肿瘤抑制因子。在横纹肌肉瘤中，整合素连接激酶和血小板衍生生长因子受体-A 与配对盒基因 7 和 3-叉头框 O1 融合，成为潜在的治疗靶点，肌肉特异性 microRNAs 作为辅助治疗具有一定的应用前景。尽管 Wilms 瘤的治愈率很高，但长期存活者仍有死于各种原因的风险。
一般来说，小儿泌尿生殖系统肿瘤患者的预后良好。这些肿瘤的分子异常的阐明正在确定风险分层、治疗策略和新药开发的候选者。
