本研究的目的是报告参加儿童肿瘤学组 AREN0534 的儿童的长期复发时间和模式，这是一项 2009 年至 2015 年进行的多中心 III 期临床试验。参与者包括患有双侧肾母细胞瘤 (BWT) 或单侧肾母细胞瘤的儿童具有发展 BWT 遗传倾向的 WT 随访长达 10 年。绘制了无事件生存 (EFS) 的平滑危险（风险）函数，以便可以可视化总体和预先指定组内的事件发生时间。对 222 名儿童（190 名 BWT 和 32 名具有 BWT 倾向的单侧 WT）进行了随访，中位时间为 8.6 年。报告了 50 起事件，其中 48 起为复发/进展。总体 8 年 EFS 为 75%（95% 置信区间：69%-83%）。 EFS 事件的最高风险是在诊断后立即发生，并且该风险在 2 年内不断下降。诊断后约 4 年观察到第二个事件高峰，直到随访期结束才报告少量事件。在子集分析中，在女性、组织学未变、淋巴结或切缘阴性、组织学良好的肾母细胞瘤患者中，化疗后存在中等风险的患者中，更常见到后来的风险增加。两年后发生的复发中，大多数是肾脏复发。这些模式表明，晚期事件可能是女性中更常见的第二原发肿瘤，尽管还需要更多的研究。临床医生可以考虑对诊断后 4 年以上的 BWT 患者进行观察。© 2024 UICC。

The objective of this study is to report the long-term timing and patterns of relapse for children enrolled in Children's Oncology Group AREN0534, a multicenter phase III clinical trial conducted from 2009 to 2015. Participants included children with bilateral Wilms tumor (BWT) or unilateral WT with genetic predisposition to develop BWT followed for up to 10 years. Smoothed hazard (risk) functions for event-free survival (EFS) were plotted so that the timing of events could be visualized, both overall and within pre-specified groups. Two hundred and twenty-two children (190 BWT and 32 unilateral WT with BWT predisposition) were followed for a median of 8.6 years. Fifty events were reported, of which 48 were relapse/progression. The overall 8-year EFS was 75% (95% confidence interval: 69%-83%). The highest risk for an EFS event was immediately after diagnosis with a declining rate over 2 years. A second peak of events was observed around 4 years after diagnosis, and a small number of events were reported until the end of the follow-up period. In subset analyses, later increases in risk were more commonly observed in patients with female sex, anaplastic histology, negative lymph nodes or margins, and favorable histology Wilms tumor patients with post-chemotherapy intermediate risk. Among relapses that occurred after 2 years, most were to the kidney. These patterns suggest that late events may be second primary tumors occurring more commonly in females, although more investigation is required. Clinicians may consider observation of patients with BWT beyond 4 years from diagnosis.© 2024 UICC.