Objectives Patients with bilateral Wilms tumor (BWT) initially receive neoadjuvant chemotherapy to shrink the tumors and increase the likelihood of successful nephron-sparing surgery. Biopsy of poorly responding tumors is often done to better understand therapy resistance. The purpose of this retrospective, single-institution study was to determine whether initial chemotherapy response is associated with tumor histology, potentially obviating the need for biopsy or change in chemotherapy. Methods Patients with synchronous BWT who underwent surgery at St Jude Children’s Research Hospital from January 2000 to March 2022 were considered for this study. A mixed-effects logistic regression model was used to evaluate the likelihood of the tumor being stromal predominant, as predicted by tumor response to neoadjuvant chemotherapy. Results Sixty-eight patients were eligible for this study. Tumors that increased in size had an odds ratio of 19.5 (95% CI: 2.46-155.03) for being stromal-predominant vs any other histologic subtype. Age at diagnosis was youngest in patients with stromal-predominant tumors, with a mean age of 18.8 months (SD = 14.1 months), compared to all other histologic subtypes (χ2=7.05, p = .07). The predictive value of a tumor growing, combined with patient age less than 18 months, for confirming stromal-predominant histology was 85.7% (95% CI: 57.18%-93.5%). Conclusions Tumors that increased in size during neoadjuvant chemotherapy were most frequently stromal-predominant BWT, especially in younger patients. Therefore, nephron-sparing surgery, rather than biopsy, or extension or intensification of neoadjuvant chemotherapy, should be considered for bilateral BWT that increase in volume during neoadjuvant chemotherapy, particularly in patients younger than 18 months of age.

中文翻译：

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双侧肾母细胞瘤对化疗的反应提示组织学亚型并指导治疗

目的 双侧肾母细胞瘤 (BWT) 患者最初接受新辅助化疗以缩小肿瘤并增加成功保留肾单位手术的可能性。通常对反应不佳的肿瘤进行活检，以更好地了解治疗耐药性。这项回顾性、单机构研究的目的是确定初始化疗反应是否与肿瘤组织学相关，从而可能避免活检或改变化疗的需要。方法 本研究考虑2000年1月至2022年3月在圣裘德儿童研究医院接受手术的同步BWT患者。使用混合效应逻辑回归模型来评估肿瘤以间质为主的可能性，正如肿瘤对新辅助化疗的反应所预测的那样。结果 68 名患者符合本研究的条件。与任何其他组织学亚型相比，增大的肿瘤以间质为主的优势比为 19.5 (95% CI: 2.46-155.03)。与所有其他组织学亚型相比，间质为主的肿瘤患者的诊断年龄最年轻，平均年龄为 18.8 个月（SD = 14.1 个月）（χ2=7.05，p = .07）。肿瘤生长与患者年龄小于 18 个月相结合，对于确认间质为主的组织学的预测价值为 85.7%（95% CI：57.18%-93.5%）。结论 新辅助化疗期间体积增大的肿瘤最常见的是间质为主的 BWT，尤其是在年轻患者中。因此，对于新辅助化疗期间体积增加的双侧 BWT，尤其是年龄小于 18 个月的患者，应考虑保留肾单位手术，而不是活检或延长或强化新辅助化疗。