Background The National Wilms Tumor Study (NWTS) approach to treating stage III favorable-histology Wilms tumor (FHWT) is Regimen DD4A (vincristine, dactinomycin, and doxorubicin) and radiation therapy. Further risk stratification is required to improve outcomes and reduce late effects. We evaluated clinical and biologic variables for patients with stage III FHWT without combined loss of heterozygosity (LOH) at chromosomes 1p and 16q treated in the Children's Oncology Group protocol AREN0532. Methods From October 2006 to August 2013, 588 prospectively treated, centrally reviewed patients with stage III FHWT were treated with Regimen DD4A and radiation therapy. Tumor LOH at 1p and 16q was determined by microsatellite analysis. Ineligible patients (n = 5) and those with combined LOH 1p/16q (n = 40) were excluded. Results A total of 535 patients with stage III disease were studied. Median follow-up was 5.2 years (range, 0.2 to 9.5). Four-year event-free survival (EFS) and overall survival estimates were 88% (95% CI, 85% to 91%) and 97% (95% CI, 95% to 99%), respectively. A total of 58 of 66 relapses occurred in the first 2 years, predominantly pulmonary (n = 36). Eighteen patients died, 14 secondary to disease. A better EFS was associated with negative lymph node status ( P < .01) and absence of LOH 1p or 16q ( P < .01), but not with gross residual disease or peritoneal implants. In contrast, the 4-year EFS was only 74% in patients with combined positive lymph node status and LOH 1p or 16q. A total of 123 patients (23%) had delayed nephrectomy. Submitted delayed nephrectomy histology showed anaplasia (n = 8; excluded from survival analysis); low risk/completely necrotic (n = 7; zero relapses), intermediate risk (n = 63; six relapses), and high-risk/blastemal type (n=7; five relapses). Conclusion Most patients with stage III FHWT had good EFS/overall survival with DD4A and radiation therapy. Combined lymph node and LOH status was highly predictive of EFS and should be considered as a potential prognostic marker for future trials.

中文翻译：

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III 期组织学良好的肾母细胞瘤的结果和预后因素：来自儿童肿瘤学小组研究 AREN0532 的报告

背景 国家 Wilms 肿瘤研究 (NWTS) 治疗 III 期组织学良好的 Wilms 肿瘤 (FHWT) 的方法是方案 DD4A（长春新碱、更生霉素和多柔比星）和放射治疗。需要进一步的风险分层以改善结果并减少后期影响。我们评估了在儿童肿瘤组协议 AREN0532 中治疗的染色体 1p 和 16q 处无杂合性缺失 (LOH) 的 III 期 FHWT 患者的临床和生物学变量。方法 2006 年 10 月至 2013 年 8 月，588 例前瞻性治疗、集中审查的 III 期 FHWT 患者接受了 DD4A 方案和放射治疗。通过微卫星分析确定 1p 和 16q 处的肿瘤 LOH。不符合条件的患者 (n = 5) 和合并 LOH 1p/16q 的患者 (n = 40) 被排除在外。结果共研究了 535 名 III 期疾病患者。中位随访时间为 5.2 年（范围 0.2 至 9.5）。四年无事件生存率 (EFS) 和总生存率估计值分别为 88%（95% CI，85% 至 91%）和 97%（95% CI，95% 至 99%）。前 2 年共有 66 次复发，其中 58 次复发，主要是肺部复发（n = 36）。18 名患者死亡，14 名继发于疾病。较好的 EFS 与淋巴结阴性 ( P < .01) 和 LOH 1p 或 16q 缺失 ( P < .01) 相关，但与明显残留病灶或腹膜植入物无关。相比之下，联合阳性淋巴结状态和 LOH 1p 或 16q 的患者的 4 年 EFS 仅为 74%。共有 123 名患者 (23%) 进行了延迟肾切除术。提交的延迟肾切除术组织学显示发育不全（n = 8；从生存分析中排除）；低风险/完全坏死（n = 7；零复发）、中等风险（n = 63；6 次复发）和高风险/胚细胞型（n=7；5 次复发）。结论 大多数 III 期 FHWT 患者在接受 DD4A 和放射治疗后具有良好的 EFS/总生存率。联合淋巴结和 LOH 状态对 EFS 具有高度预测性，应被视为未来试验的潜在预后标志物。