INTRODUCTION High-risk (HR) metastatic (stage IV) Wilms tumours (WTs) have a particular poor outcome. METHODS Here, we report the results of HR (diffuse anaplastic [DA] or blastemal type [BT]) stage IV WT treated patients according to the HR arm in the SIOP2001 prospective study. RESULTS From January 2002 to August 2014, 3559 patients with WT were included in the SIOP2001 trial. Among the 525 patients (15%) with metastatic WT, 74 (14%) had stage IV HR-WT. The median age at diagnosis was 5.5 years (range: 1.4-18.3). Thirty-four patients (47%) had BT-WT and 40 (53%) had DA-WT. Five-year event-free survival rates were 44 ± 17% and 28 ± 15% for BT-WT and DA-WT, respectively (p = 0.09). Five-year overall survival rates were 53 ± 17% and 29 ± 16% for BT-WT and DA-WT, respectively (p = 0.03). Metastatic complete response after preoperative treatment was significantly associated with outcome in univariate and multivariate analyses (hazards ratio = 0.3; p = 0.01). Postoperative radiotherapy of metastatic sites might also be beneficial. Forty-three of 74 patients experienced a relapse or progression predominantly in the lungs (80%). The median time to relapse/progression after diagnosis was 7.3 months (range: 1.6-33.3) and 4.9 months (range: 0.7-28.4) for BT-WT and DA-WT, respectively (p = 0.67). This is the first prospective evidence of inferior survival of stage IV BT-WT as compared with historical intermediate-risk WT. Survival of patients with stage IV DA-WT has not improved compared to the previous SIOP93-01 study. CONCLUSION These results call for new treatment approaches for patients with HR stage IV WT.

中文翻译：

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根据SIOP2001方案治疗的IV期高危Wilms肿瘤患者的治疗结果：SIOP肾脏肿瘤研究小组的报告。

简介高危（HR）转移性（IV期）威尔姆斯肿瘤（WTs）的预后特别差。方法在这里，我们根据SIOP2001前瞻性研究中的HR部门报告了HR（弥散性间变性[DA]或母细胞型[BT]）IV期WT治疗患者的结果。结果从2002年1月到2014年8月，SIOP2001试验纳入了3559例WT患者。在525例转移性WT患者中，有74例（14％）患有IV期HR-WT。诊断时的中位年龄为5.5岁（范围：1.4-18.3）。BT-WT患者34例（47％），DA-WT患者40例（53％）。BT-WT和DA-WT的五年无事件生存率分别为44±17％和28±15％（p = 0.09）。BT-WT和DA-WT的五年总生存率分别为53±17％和29±16％（p = 0.03）。术前治疗后转移完全反应与单因素和多因素分析的结果显着相关（危险比= 0.3； p = 0.01）。转移部位的术后放疗也可能有益。74例患者中有43例主要在肺部复发或进展（80％）。BT-WT和DA-WT诊断后复发/进展的中位时间分别为7.3个月（范围：1.6-33.3）和4.9个月（范围：0.7-28.4）（p = 0.67）。与历史中危WT相比，这是IV期BT-WT生存期较差的第一个前瞻性证据。与先前的SIOP93-01研究相比，IV期DA-WT患者的生存率没有改善。结论这些结果要求对HR IV期WT患者进行治疗。