有利的组织学 Wilms 瘤 5 年总生存率非常高(大于 90%)。[1]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007;13:463-470.http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com[178]Green DM, Breslow NE, Beckwith JB, et al. Treatment outcomes in patients less than 2 years of age with small, stage I, favorable-histology Wilms tumors: a report from the National Wilms Tumor Study. J Clin Oncol. 1993;11:91-95.http://www.ncbi.nlm.nih.gov/pubmed/8380295?tool=bestpractice.com肿瘤小于 550 g 且年龄小于 2 岁的患者也有极好的治疗效果。[90]Green DM. The treatment of stages I-IV favorable histology Wilms' tumor. J Clin Oncol. 2004;22:1366-1372.http://www.ncbi.nlm.nih.gov/pubmed/15084612?tool=bestpractice.com[173]Breslow NE, Beckwith JB, Haase GM, et al. Radiation therapy for favorable histology Wilms tumor: prevention of flank recurrence did not improve survival on National Wilms Tumor Studies 3 and 4. Int J Radiat Oncol Biol Phys. 2006;65:203-209.http://www.pubmedcentral.nih.gov/articlerender.fcgi?tool=pubmed&pubmedid=16542795http://www.ncbi.nlm.nih.gov/pubmed/16542795?tool=bestpractice.com但诊断后 25 年的治疗相关性致残率和死亡率的风险增加。[207]Termuhlen AM, Tersak JM, Liu Q, et al. Twenty-five year follow-up of childhood Wilms tumor: a report from the Childhood Cancer Survivor Study. Pediatr Blood Cancer. 2011;57:1210-1216.http://www.ncbi.nlm.nih.gov/pubmed/21384541?tool=bestpractice.com
如果存在间变、非整倍性、远处转移性疾病(尤其是淋巴结和/或肝转移和/或血管内瘤栓)以及 1p 和 16q 处杂合性缺失 (LOH),则预后不良。[1]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007;13:463-470.http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com[40]Wittmann S, Zirn B, Alkassar M, et al. Loss of 11q and 16q in Wilms tumors is associated with anaplasia, tumor recurrence, and poor prognosis. Genes Chromosomes Cancer. 2007;46:163-170.http://www.ncbi.nlm.nih.gov/pubmed/17099873?tool=bestpractice.com[59]Osterheld MC, Caron L, Meagher-Villemure K. Role of DNA content analysis and immunohistochemistry in the evaluation of the risk of unfavourable outcome in Wilms' tumours. Anticancer Res. 2008;28:751-756.http://www.ncbi.nlm.nih.gov/pubmed/18507016?tool=bestpractice.com[66]Malogolowkin M, Cotton CA, Green DM, et al. Treatment of Wilms tumor relapsing after initial treatment with vincristine, actinomycin D, and doxorubicin. A report from the National Wilms Tumor Study Group. Pediatr Blood Cancer. 2008;50:236-241.http://www.ncbi.nlm.nih.gov/pubmed/17539021?tool=bestpractice.com[116]Dome JS, Cotton CA, Perlman EJ, et al. Treatment of anaplastic histology Wilms' tumor: results from the fifth National Wilms' Tumor Study. J Clin Oncol. 2006;24:2352-2358.http://www.ncbi.nlm.nih.gov/pubmed/16710034?tool=bestpractice.com[127]Skotnicka-Klonowicz G, Rieske P, Bartkowiak J, et al. 16q heterozygosity loss in Wilms' tumour in children and its clinical importance. Eur J Surg Oncol. 2000;26:61-66.http://www.ncbi.nlm.nih.gov/pubmed/10718182?tool=bestpractice.com[144]Grundy PE, Breslow NE, Li S, et al. Loss of heterozygosity for chromosomes 1p and 16q is an adverse prognostic factor in favorable-histology Wilms tumor: a report from the National Wilms Tumor Study Group. J Clin Oncol. 2005;23:7312-7321.http://www.ncbi.nlm.nih.gov/pubmed/16129848?tool=bestpractice.com[208]Nakadate H, Yokomori K, Watanabe N, et al. Mutations/deletions of the WT1 gene, loss of heterozygosity on chromosome arms 11p and 11q, chromosome ploidy and histology in Wilms' tumors in Japan. Int J Cancer. 2001;94:396-400.http://onlinelibrary.wiley.com/doi/10.1002/ijc.1475/fullhttp://www.ncbi.nlm.nih.gov/pubmed/11745420?tool=bestpractice.com[209]Messahel B, Williams R, Ridolfi A, et al. Allele loss at 16q defines poorer prognosis Wilms tumour irrespective of treatment approach in the UKW1-3 clinical trials: a Children's Cancer and Leukaemia Group (CCLG) study. Eur J Cancer. 2009;45:819-826.http://www.ncbi.nlm.nih.gov/pubmed/19231157?tool=bestpractice.com
淋巴结累及和存在显微镜下残留疾病可高度预测 III 期有利组织学疾病患者的无事件生存和总生存率。[210]Ehrlich PF, Anderson JR, Ritchey ML, et al. Clinicopathologic findings predictive of relapse in children with stage III favorable-histology Wilms tumor. J Clin Oncol. 2013;31:1196-1201.http://www.ncbi.nlm.nih.gov/pubmed/23382471?tool=bestpractice.com
根据 NWTS-5 治疗的所有 IV 期有利组织学 Wilms 瘤患者的四年无事件生存率为 75%。[1]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007;13:463-470.http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com参与 SIOP-9 试验、接受术前化疗的 IV 期患者的 5 年无复发生存率为 73%。[121]Reinhard H, Semler O, Burger D, et al. Results of the SIOP 93-01/GPOH trial and study for the treatment of patients with unilateral nonmetastatic Wilms Tumor. Klin Padiatr. 2004;216:132-140.http://www.ncbi.nlm.nih.gov/pubmed/15175957?tool=bestpractice.com如果 IV 期患者组织学有利、转移性疾病局限于肝或肺,且转移对术前化疗有反应,则视为预后极好。[187]Berger M, Fernandez-Pineda I, Cabello R, et al. The relationship between the site of metastases and outcome in children with stage IV Wilms tumor: data from 3 European pediatric cancer institutions. J Pediatr Hematol Oncol. 2013;35:518-524.http://www.ncbi.nlm.nih.gov/pubmed/23588334?tool=bestpractice.com
复发率低。[1]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007;13:463-470.http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com复发性肿瘤患者的不良预后因素包括之前用多柔比星治疗、诊断后不到 12 个月出现复发以及腹部照射后出现腹内复发。[1]D'Angio GJ. The National Wilms Tumor Study: a 40 year perspective. Lifetime Data Anal. 2007;13:463-470.http://www.ncbi.nlm.nih.gov/pubmed/18027087?tool=bestpractice.com[17]Grundy P, Breslow N, Green DM, et al. Prognostic factors for children with recurrent Wilms' tumor: results from the Second and Third National Wilms' Tumor Study. J Clin Oncol. 1989;7:638-647.http://www.ncbi.nlm.nih.gov/pubmed/2540289?tool=bestpractice.com[193]Speafico F, Pritchard Jones K, Malogolowkin MH, et al. Treatment of relapsed Wilms tumors: lessons learned. Expert Rev Anticancer Ther. 2009;9:1807-1815.http://www.ncbi.nlm.nih.gov/pubmed/19954292?tool=bestpractice.com复发患者的预后较差。
研究观察到 Denys-Drash 综合征患者出现不良治疗效果,因为该疾病通常为双侧且与进行性肾功能衰竭相关。[6]Heppe RK, Koyle MA, Beckwith JB. Nephrogenic rests in Wilms tumor patients with the Drash syndrome. J Urol. 1991;145:1225-1228.http://www.ncbi.nlm.nih.gov/pubmed/1851891?tool=bestpractice.com[83]Breslow NE, Collins AJ, Ritchey ML, et al. End stage renal disease in patients with Wilms tumor: results from the National Wilms Tumor Study Group and the United States Renal Data System. J Urol. 2005;174:1972-1975.http://www.pubmedcentral.nih.gov/articlerender.fcgi?tool=pubmed&pubmedid=16217371http://www.ncbi.nlm.nih.gov/pubmed/16217371?tool=bestpractice.com[84]Breslow NE, Takashima JR, Ritchey ML, et al. Renal failure in the Denys-Drash and Wilms tumor-aniridia syndromes. Cancer Res. 2000;60:4030-4032.http://cancerres.aacrjournals.org/cgi/content/full/60/15/4030http://www.ncbi.nlm.nih.gov/pubmed/10945603?tool=bestpractice.com但成功的肾移植可提高总生存率。[211]Kist-van Holthe JE, Ho PL, Stablein D, et al. Outcome of renal transplantation for Wilms' tumor and Denys-Drash syndrome: a report of the North American Pediatric Renal Transplant Cooperative Study. Pediatr Transplant. 2005;9:305-310.http://www.ncbi.nlm.nih.gov/pubmed/15910385?tool=bestpractice.com
一项研究发现西班牙人的预后可能更差;但 Wilms 瘤的种族差异仍需进一步的研究。[212]Amirian ES. The role of Hispanic ethnicity in pediatric Wilms' tumor survival. Pediatr Hematol Oncol. 2013;30:317-327.http://www.ncbi.nlm.nih.gov/pubmed/23484868?tool=bestpractice.com