Department of Surgery, Children's Hospital Boston and Harvard Medical School, Boston, MA, USA.
Ann Surg. 2010 Mar;251(3):555-8. doi: 10.1097/SLA.0b013e3181c0e5d7.
To determine the event-free survival (EFS) and overall survival (OS) of children with very low risk Wilms tumor (VLRWT) treated with surgery only.
Previous studies suggested that postoperative chemotherapy had not improved the prognosis of children with VLRWT. A total of 77 children <24 months of age with small (<550 g) Stage I favorable histology Wilms tumors were treated with surgery only. This study was closed based on stopping rules to ensure that the 2-year EFS was > or =90%.
A total of 77 children were assessed for EFS and OS. Of these patients, 21 enrolled at the time of closure were recalled, treated with dactinomycin and vincristine (regimen EE4A), and censored for analysis thereafter. About 111 children subsequently treated with EE4A were available for comparison.
Median follow-up of surviving patients was 8.2 years for surgery only (range, 1.9-11.8 years) and 5.2 years for the EE4A group (range, 1.6-8.9 years). The estimated 5-year EFS for surgery only was 84% (95% confidence interval [CI]: 73%, 91%); for the EE4A patients it was 97% (95% CI: 92%, 99%, P = 0.002). One death was observed in each treatment group. The estimated 5-year OS was 98% (95% CI: 87%, 99%) for surgery only and 99% (95% CI: 94%, 99%) for EE4A (P = 0.70).
The surgery-only EFS was lower than anticipated but, coupled with a much higher than anticipated salvage rate of the chemotherapy naive patients whose disease recurred, led to an observed long-term OS equivalent to that seen with 2-drug chemotherapy. This approach to the treatment of patients with VLRWT eliminates the toxic side-effects of chemotherapy for a large majority of patients. A follow-up study is underway to confirm these findings.
确定仅接受手术治疗的极低危 Wilms 肿瘤（VLRWT）患儿的无事件生存（EFS）和总生存（OS）。
先前的研究表明，术后化疗并未改善 VLRWT 患儿的预后。共有 77 名年龄小于 24 个月且肿瘤较小（<550g）、组织学良好的 I 期 Wilms 肿瘤患儿仅接受手术治疗。本研究根据终止规则关闭，以确保 2 年 EFS>或=90%。
对 77 例患儿进行 EFS 和 OS 评估。其中 21 例在关闭时入组，接受放线菌素 D 和长春新碱（EE4A 方案）治疗，并在此后进行分析。随后有 111 例患儿接受 EE4A 治疗，可用于比较。
仅手术组的存活患儿中位随访时间为 8.2 年（范围，1.9-11.8 年），EE4A 组为 5.2 年（范围，1.6-8.9 年）。仅手术组的 5 年 EFS 估计为 84%（95%置信区间[CI]：73%，91%）；EE4A 患儿为 97%（95% CI：92%，99%，P=0.002）。两组各有 1 例死亡。仅手术组的 5 年 OS 估计为 98%（95% CI：87%，99%），EE4A 组为 99%（95% CI：94%，99%）（P=0.70）。
仅手术组的 EFS 低于预期，但考虑到化疗初治患儿的复发挽救率远高于预期，观察到的长期 OS 与 2 药化疗相当。这种治疗极低危 Wilms 肿瘤患儿的方法消除了化疗对大多数患儿的毒副作用。一项随访研究正在进行中以确认这些发现。
