Tamaro P
Ospedale Inf. Burlo Garofolo, Clinica Pediatrica Università di Trieste.
Arch Ital Urol Androl. 1997 Sep;69(4):227-32.
Wilms tumor, although rare, is the most frequent malignant renal tumor in children. With approximately 70 new cases diagnosed annually in Italy, it is important to collect these rare cases and to treat them in a uniform way. At the dawn of this century virtually all children with Wilms tumors died: today 95% of them survive; the treatment is carefully tailored to well defined risk factors (histology-stage-molecular markers) to minimize short and long-term toxicities. The object of this review is to describe the pathway to the progress. The most important step was to recognize that a single institution could not collect enough patients to answer complex questions, whereas this was possible for super-groups like the National Wilms Tumor Study Group (NWTSG) in the USA, the multinational SIOP study group in Europe, the United Kingdom group, the Italian group (CNR-AIEOP) and more recently the Brazilian one. Wilms tumor is the paradigm for the multimodal treatment of a pediatric malignant solid tumor, the development in surgical technique, the recognition of the sensitivity of Wilms tumor to irradiation and the availability of several chemotherapeutic agents led to a dramatic change in the prognosis for most patients. Much progress has been made in the study of histopatology of childhood renal tumors: the patients must be stratified into two groups, the favourable and the unfavourable histology, further subdivided into anaplasic tumors (focal or diffuse), clear cell tumors and rhabdoid tumors. The past few years have provided a breakthrough in understanding some of the genetic factors involved in Wilms' tumor: moreover, possible chromosomal prognostic factors have been identified: loss of heterozygosity of 16q markers and 1p markers. Today the results of the treatment of Wilms tumors are very good. In the NWTS-3 the four year relapse-free survival rate in stage 1 with favourable and with anaplastic histology was 92%, in stage 2 with favourable histology 88%, in stage 3 with favourable histology 79%, in stage 4 and in stage 2, 3 and 4 with unfavourable histology 71%. The Italian group has obtained less impressive results in the ¿80, but similar results in the first stage with the ¿92 protocol. There is a debate about the immediate nephrectomy preferred by NWTS and the preoperative strategies adopted by SIOP group. Successful treatment may be associated with many late effect: in patients cured of Wilms tumor the risk of congestive heart failure has been less than 1.7%, the risk of a second tumor less than 1%. The must important late effect remains the relapse of the disease: the risk is about 14-20%.
肾母细胞瘤虽然罕见，但却是儿童中最常见的恶性肾肿瘤。在意大利，每年约有70例新确诊病例，因此收集这些罕见病例并以统一方式进行治疗非常重要。在本世纪初，几乎所有患肾母细胞瘤的儿童都会死亡：如今，95%的患儿得以存活；治疗方案会根据明确的风险因素（组织学、分期、分子标志物）进行精心调整，以尽量减少短期和长期毒性。本综述的目的是描述取得进展的历程。最重要的一步是认识到单个机构无法收集到足够多的患者来回答复杂问题，而像美国的国家肾母细胞瘤研究组（NWTSG）、欧洲的跨国SIOP研究组、英国研究组、意大利研究组（CNR - AIEOP）以及最近的巴西研究组这样的超级团队则能够做到。肾母细胞瘤是小儿恶性实体瘤多模式治疗的典范，手术技术的发展、对肾母细胞瘤放射敏感性的认识以及多种化疗药物的可得性，使大多数患者的预后发生了巨大变化。儿童肾肿瘤组织病理学研究取得了很大进展：患者必须分为两组，即组织学良好组和不良组，不良组再进一步细分为间变肿瘤（局灶性或弥漫性）、透明细胞肿瘤和横纹肌样肿瘤。过去几年在理解肾母细胞瘤相关的一些遗传因素方面取得了突破：此外，还确定了可能的染色体预后因素：16q标志物和1p标志物的杂合性缺失。如今，肾母细胞瘤的治疗效果非常好。在NWTS - 3研究中，组织学良好且为间变的1期患者四年无复发生存率为92%，组织学良好的2期患者为88%，组织学良好的3期患者为79%，4期以及组织学不良的2期、3期和4期患者为71%。意大利研究组在20世纪80年代取得的结果不太理想，但在90年代采用92方案时1期的结果与之相似。关于NWTS所倾向的直接肾切除术和SIOP组采用的术前策略存在争议。成功治疗可能会带来许多晚期影响：在治愈肾母细胞瘤的患者中，充血性心力衰竭的风险低于1.7%，患第二种肿瘤的风险低于1%。最重要的晚期影响仍然是疾病复发：风险约为14% - 20%。
