Boccon-Gibod L, Rey A, Sandstedt B, Delemarre J, Harms D, Vujanic G, De Kraker J, Weirich A, Tournade M F
Department of Pathology, Hôpital d'Enfants Armand-Trousseau, Paris, France.
Med Pediatr Oncol. 2000 Mar;34(3):183-90. doi: 10.1002/(sici)1096-911x(200003)34:3<183::aid-mpo4>3.0.co;2-o.
The SIOP Nephroblastoma therapeutic protocols include a period of preoperative chemotherapy followed by nephrectomy and a period of postoperative chemotherapy. From the outset, identification of low-risk groups has been an aim of the SIOP Nephroblastoma Trials and Studies. Now that 90% of children with Wilms tumor can be cured, attention is even more focused on the identification of patients who could benefit from less aggressive postoperative therapy, thus minimizing the morbidity and late effects associated with treatment. The prognostic implications of total necrosis in nephroblastoma after chemotherapy have not been investigated hitherto.
Between November 1, 1987 and June 30, 1993, 599 patients referred to the SIOP-9 Nephroblastoma Trial and Study were preoperatively treated and classified as stages I-IV nonanaplastic Wilms tumor.
Of these 599 patients, pathologic examination of the nephrectomy specimen revealed a completely necrotic Wilms tumor (CNWT) with no viable tumor remaining in 59 (10%): these comprised 37 stages I-III and 22 stage IV. Of these patients, 58 (98%) had no evidence of disease at 5 years vs. 90% for the rest of the cohort (P < 0.05). Stages I-III patients represented 63% of CNWT and had a 97% overall survival rate. The only death was related to veno-occlusive disease and occurred in a stage I patient in the month following nephrectomy. Stage IV patients represented 37% of CNWT (vs. only 10% of all other cases of unilateral nonanaplastic Wilms tumor) and had a 100% rate of survival. Children with CNWT were older (mean 59 months vs. 43 months); their tumor at diagnosis was larger and had regressed more significantly at subsequent ultrasound examination. The data also uphold the hypothesis that Wilms tumors of blastemic pattern are most aggressive, but also are extremely responsive to chemotherapy.
Patients with unilateral nonanaplastic WT that showed total necrosis following preoperative chemotherapy had excellent outcome and should benefit from less aggressive postoperative treatment in further trials. Other very responsive tumors, such as Wilms with <10% viable tumor, should also be assessed.
国际小儿肿瘤学会（SIOP）肾母细胞瘤治疗方案包括术前化疗期，随后进行肾切除术以及术后化疗期。从一开始，识别低风险组就是SIOP肾母细胞瘤试验与研究的目标。鉴于90%的肾母细胞瘤患儿能够治愈，现在更加关注识别那些能够从强度较低的术后治疗中获益的患者，从而将与治疗相关的发病率和远期影响降至最低。化疗后肾母细胞瘤中完全坏死的预后意义迄今尚未得到研究。
在1987年11月1日至1993年6月30日期间，599名转诊至SIOP-9肾母细胞瘤试验与研究的患者接受了术前治疗，并被分类为I-IV期非间变肾母细胞瘤。
在这599名患者中，肾切除标本的病理检查显示有59例（10%）为完全坏死的肾母细胞瘤（CNWT），无存活肿瘤残留：其中包括37例I-III期和22例IV期。在这些患者中，58例（98%）在5年时无疾病证据，而队列其余患者为90%（P<0.05）。I-III期患者占CNWT的63%，总生存率为97%。唯一的死亡与肝静脉闭塞病有关，发生在一名I期患者肾切除术后的当月。IV期患者占CNWT的37%（而在所有其他单侧非间变肾母细胞瘤病例中仅占10%），生存率为100%。CNWT患儿年龄较大（平均59个月对43个月）；他们诊断时的肿瘤较大，且在随后的超声检查中消退更为显著。数据还支持以下假设，即胚芽型肾母细胞瘤最具侵袭性，但对化疗也极为敏感。
术前化疗后显示完全坏死的单侧非间变肾母细胞瘤患者预后极佳，在进一步试验中应从强度较低的术后治疗中获益。其他反应性很强的肿瘤，如存活肿瘤<10%的肾母细胞瘤，也应进行评估。
