I 期上皮或间质型肾母细胞瘤为低危肿瘤：英国-CCLG 和 GPOH 研究中（2001-2020 年）采用 SIOP-WT-2001 方案治疗患者的分析。

Stage I epithelial or stromal type Wilms tumors are low risk tumors: An analysis of patients treated on the SIOP-WT-2001 protocol in the UK-CCLG and GPOH studies (2001-2020).

机构信息

Department of Pathology, Oslo University Hospital, Rikshospitalet, Oslo, Norway.

Department of Paediatric Haematology and Oncology, Saarland University Hospital, Homburg, Germany.

出版信息

Cancer. 2023 Jun 15;129(12):1930-1938. doi: 10.1002/cncr.34734. Epub 2023 Mar 17.

DOI:10.1002/cncr.34734

PMID:36929497

Abstract

BACKGROUND

Patients treated with preoperative chemotherapy with stage I intermediate-risk Wilms tumor (IR-WT) represent the largest group of patients with Wilms tumor (WT), and they have excellent outcomes.

METHODS

The authors performed a retrospective analysis of patients with stage I epithelial (ET-WT) or stromal type WT (ST-WT) treated pre- and postoperatively according to the International Society of Paediatric Oncology-WT-2001 protocol in the UK Children's Cancer and Leukaemia Group and Gesellschaft für Pädiatrische Onkologie und Hämatologie groups' participation in the relevant WT trials and studies (2001-2020).

RESULTS

There were 880 patients with stage I IR-WT, including 124 with ET-WT, 156 with ST-WT, and 600 with other IR-WT (oIR-WT). Patients with stage I ET-WT or ST-WT were significantly younger than patients with oIR-WT, represented a large proportion of stage I WTs in their groups, and tumors showed poor histologic response to preoperative chemotherapy. The 5-year event-free survival (EFS) estimates for patients with stage I ET-WT (96.8% ± 1.8 SE) or ST-WT (96.8% ± 1.6 SE) were significantly better than for patients with oIR-WT (90.3% ± 1.3 SE) (p = .014 and p = .009, respectively). A multivariate analysis showed that histologic type (ET-WT or ST-WT) remained a significant factor for EFS when adjusted for age and gender (p = .032 and p = .022, respectively). In both groups, relapses occurred in 3.2% of patients, and the overall survival was 99.2%.

CONCLUSIONS

The results suggest that stage I ET-WT or ST-WT could be regarded as low-risk WT, for which omission of postoperative chemotherapy should be considered.

PLAIN LANGUAGE SUMMARY

Patients with pretreated intermediate-risk Wilms tumor (WT) represent the largest group of patients with WT. This study reports the outcomes of patients with stage I epithelial type (ET-WT) or stromal type WT (ST-WT). These patients were significantly younger and had a larger proportion of stage I cases than patients with other intermediate-risk WT (oIR-WT). The event-free survival for patients with stage I ET-WT and ST-WT was significantly better than for patients with oIR-WT. Rare relapses were curable resulting in 99.2% overall survival.

摘要

背景

接受 I 期中危 Wilms 瘤（IR-WT）术前化疗的患者是 Wilms 瘤（WT）患者中最大的群体，他们的预后极好。

方法

作者对英国儿童癌症和白血病组（UK Children's Cancer and Leukaemia Group）及 Gesellschaft für Pädiatrische Onkologie und Hämatologie 组（Gesellschaft für Pädiatrische Onkologie und Hämatologie groups）按照国际小儿肿瘤协会-WT-2001 方案（International Society of Paediatric Oncology-WT-2001 protocol）进行术前和术后治疗的 I 期上皮型（ET-WT）或间质型 WT（ST-WT）患者进行回顾性分析，这些患者参加了 WT 相关试验和研究（2001-2020 年）。

结果

共有 880 例 I 期 IR-WT 患者，其中 124 例为 ET-WT，156 例为 ST-WT，600 例为其他 IR-WT（oIR-WT）。I 期 ET-WT 或 ST-WT 患者明显比 oIR-WT 患者年轻，在各自组中占很大比例，且肿瘤对术前化疗的组织学反应不佳。I 期 ET-WT（96.8%±1.8 SE）或 ST-WT（96.8%±1.6 SE）患者的 5 年无事件生存率（EFS）估计明显优于 oIR-WT 患者（90.3%±1.3 SE）（p=.014 和 p=.009）。多变量分析显示，组织学类型（ET-WT 或 ST-WT）在调整年龄和性别后仍然是 EFS 的显著因素（p=.032 和 p=.022）。在这两组中，分别有 3.2%的患者出现复发，总生存率为 99.2%。