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非临床试验环境下复发性 favorable-histology Wilms 肿瘤的结局。

Outcomes of relapsed favorable-histology Wilms tumor in non-clinical trial setting.

机构信息

Department of Pediatrics, Texas Children's Cancer and Hematology Centers, Baylor College of Medicine, Houston, Texas, USA.

Department of Pediatrics, MD Anderson Cancer Center, Houston, Texas, USA.

出版信息

Pediatr Blood Cancer. 2025 Jan;72(1):e31347. doi: 10.1002/pbc.31347. Epub 2024 Oct 12.

Abstract

BACKGROUND

The last major North American cooperative group clinical trial for relapsed favorable-histology Wilms tumor (FHWT) was completed in 2002. The outcomes of patients with relapsed Wilms tumor subsequently treated outside of clinical trials are unknown. The aim of this study was to assess the efficacy and toxicity of salvage therapies used for patients with FHWT suffering first relapse.

METHODS

We conducted a retrospective chart review of patients treated for first relapse of FHWT at six large North American institutions from January 2002 through August 2018.

RESULTS

Ninety-four patients were identified. Thirty-six patients were classified as standard-risk relapse (SRR), 49 patients as high-risk relapse (HRR), and seven patients as very high-risk relapse (VHRR). Two patients were unable to be classified. Twenty-one patients with SRR were treated with Regimen I. The 4-year event-free survival (EFS) and overall survival (OS) for SRR were 82.4% and 93.3%, respectively, with median follow-up of 72 months. Twenty-eight HRR/VHRR patients were treated with ICE therapy, while 13 received National Wilms Tumor Study (5th) (NWTS-5) Stratum C. No patient completed protocol therapy per Stratum C; median maintenance cycles administered were two cycles. The 4-year EFS and OS for HRR/VHRR were 32.6% and 58.3%, respectively, with median follow-up of 33 months.

CONCLUSIONS

Outcomes for all strata of relapsed WT patients treated in a non-clinical setting appear to have similar outcomes to historical cohorts treated on NWTS-5. Improved strategies are urgently needed for HRR and VHRR relapses.

摘要

背景

2002 年完成了最后一项针对复发性良好组织学 Wilms 肿瘤(FHWT)的北美合作组临床试验。此后,在临床试验之外接受治疗的复发性 Wilms 肿瘤患者的结果尚不清楚。本研究旨在评估接受 FHWT 首次复发患者使用挽救治疗的疗效和毒性。

方法

我们对 2002 年 1 月至 2018 年 8 月期间,六家北美大型机构治疗的 FHWT 首次复发患者进行了回顾性图表审查。

结果

共确定了 94 名患者。36 名患者被归类为标准风险复发(SRR),49 名患者为高风险复发(HRR),7 名患者为非常高风险复发(VHRR)。有两名患者无法分类。21 名 SRR 患者接受了 I 方案治疗。SRR 的 4 年无事件生存率(EFS)和总生存率(OS)分别为 82.4%和 93.3%,中位随访时间为 72 个月。28 名 HRR/VHRR 患者接受 ICE 治疗,而 13 名患者接受了国家 Wilms 肿瘤研究(第 5 次)(NWTS-5)C 层治疗。没有患者按照 C 层完成协议治疗;中位数给予的维持周期为两个周期。HRR/VHRR 的 4 年 EFS 和 OS 分别为 32.6%和 58.3%,中位随访时间为 33 个月。

结论

在非临床环境中治疗的所有复发 WT 患者的分层结果似乎与接受 NWTS-5 治疗的历史队列相似。对于 HRR 和 VHRR 复发,迫切需要改进策略。

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