Blastemal-type Wilms tumour (BT-WT) has been identified as a high risk histological subgroup in WT assessed after pre-nephrectomy chemotherapy in trials of the International Society of Paediatric Oncology (SIOP) Renal Tumour Study Group. Therefore, in SIOPWT2001, post-operative chemotherapy for BT-WT was intensified aiming to improve survival. Survival analysis of all unilateral BT-WT patients (SIOPWT2001) (n=238), was compared with historical BT-WT controls (SIOP93-01) (n=113). 351/4061 (8.6%) unilateral non-metastatic BT-WT patients (SIOP93-01/SIOPWT2001) were studied. Median age at diagnosis was 43 months (Inter Quartile Range (IQR) 24-68 months), stages: I (n=140, 40%), II (n=106, 30%), III (n=105, 30%). BT-WTs were higher staged, showed greater volume decrease after pre-operative chemotherapy and were diagnosed at an older median age compared to other WT patients. Patient characteristics did not differ substantially between SIOP93-01 and SIOPWT2001. Univariate analysis showed a 5-year event-free survival (EFS) of 80% (95% confidence interval (CI): 75-86%) (SIOPWT2001) compared to 67% in SIOP93-01 (95% CI: 59-76%; p=0.006) and overall survival (OS) of 88% (95% CI: 83-93%) (SIOPWT2001) compared to 84% (95% CI: 77-91%; p=0.4) in SIOP93-01. 95% of relapses were distant metastases (SIOP93-01/SIOPWT2001). Treatment protocol, age at diagnosis, tumour stage (III versus I/II) and volume (at surgery), were prognostic variables for EFS (uni- and multivariate Cox regression analysis). Independent prognosticators for OS were age at diagnosis, tumour stage and volume (at surgery). The most significant survival benefit of intensified treatment, was observed in Stage I (EFS 96% in SIOPWT2001 (OS 100%), 71% in SIOP93-01 (OS 90%)). BT-WT derived benefits from more intensive chemotherapy as reflected by a reduction in relapse risk. However, the benefit of the more intensive chemotherapy to improve OS was only observed in stage I BT-WTs, by adding doxorubicin.

译文

在国际儿科肿瘤学会 (SIOP) 肾肿瘤研究小组的试验中,囊胚型Wilms肿瘤 (bt-wt) 已被确定为肾切除术前化疗后评估的WT中的高风险组织学亚组。因此,在SIOPWT2001中,加强了对BT-WT的术后化疗,以提高生存率。将所有单侧BT-WT患者的生存分析 (SIOPWT2001) (n = 238) 与历史BT-WT对照 (SIOP93-01) (n = 113) 进行了比较。研究了351/4061 (8.6%) 单侧非转移性BT-WT患者 (SIOP93-01/SIOPWT2001)。诊断时的中位年龄为43个月 (四分位距离 (IQR) 24-68个月),阶段: I (n = 140,40%),II (n = 106,30%),III (n = 105,30%)。与其他WT患者相比,bt-wts分期更高,术前化疗后显示出更大的体积减少,并且在中位年龄较大的情况下被诊断出。SIOP93-01和siopwt2001之间的患者特征没有显着差异。单因素分析显示5年无事件生存率 (EFS) 为80% (95% 置信区间 (CI): 75-86%) (SIOPWT2001),而SIOP93-01为67% (95% CI: 59-76%; p = 0.006),88% 总生存率 (OS) 为95% CI: 83-93%) (SIOPWT2001) 与SIOP93-01中的84% (95% CI: 77-91%; p = 0.4) 相比。95% 的复发是远处转移 (SIOP93-01/SIOPWT2001)。治疗方案,诊断年龄,肿瘤分期 (III与I/II) 和体积 (手术时) 是EFS的预后变量 (单和多变量Cox回归分析)。OS的独立预后指标是诊断时的年龄,肿瘤分期和体积 (手术时)。强化治疗的最显着生存益处是在I期观察到的 (SIOPWT2001中的EFS 96% (OS 100%),SIOP93-01中的71% (OS 90%))。Bt-wt从更密集的化疗中获益,这反映在复发风险降低上。然而,通过添加阿霉素,仅在I期bt-wts中观察到更强化的化疗改善OS的益处。

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