单中心儿童肾肿瘤SCMC WT-99方案临床报告

Protocol SCMC WT-99 for childhood Wilms' tumor

目的改善儿童肾母细胞瘤预后。方法对1998年10月至2007年12月住院明确诊断为肾母细胞瘤及肾肉瘤患儿,采用外科手术、内科化疗、选择性放疗,病理科、影像学科协作诊断综合治疗的上海儿中心WT-99方案。按方案中条件根据分期及其他危险因素进行分组,并按分组给予不同药物组合和强度的化疗。Ⅰ期及Ⅱ期病理分型预后良好型不放疗。估计手术不能完全切除时给予2个疗程术前化疗。结果全组54例,年龄3个月至13岁。病理分类预后良好型39例,预后不良型4例,透明细胞肉瘤5例,横纹肌肉瘤样1例,分型困难或未分化型5例。临床结合病理分期为Ⅰ期19例,Ⅱ期7例,Ⅲ期19例,Ⅳ7例,Ⅴ期2例。54例中获完全缓解51例(94.4%),3例初治失败。缓解后复发9例。随访1～104个月,中位31.5个月,估计5年无病生存率(EFS)和总生存率(SR)分别为75.7%和83.9%。结论多专业联合诊断治疗工作模式及儿中心WT-99方案对儿童肾母细胞瘤有效。

Objectives To improve the survival rate of pediatric Wilms＇ tumor by optimizing the therapeutic protocol. Methods Diagnosis am] treatment was decided by the multi-disciplinary team including oncologists, surgeons, pathologists, radiologists and diagnostic radiologists. WT-99 protocol was applied to 54 patients aged from 3 months to 13 years oht hospitalized from Oct. 1998 to Dec. 2007. The regimen for FH stage Ⅰ , Ⅱ and anaplastie stage I patients was vineristine （Vcr） and daetinomycin （Act-D）, while patients with focal anaplastie stage Ⅱ to Ⅳ and FH stage Ⅲ , Ⅳ were given Ver, Aet-D and adriamycin （Adr）. Diffuse anaplastic stage Ⅱ to Ⅳ and clear cell stage Ⅰ to Ⅳ patients received four-drug regimen including Vcr, etoposide （VP-16） , Adr and cytoxan （CTX）. Rhabdoid stage I to 1V received earboplatin, VP-16 and CTX. For those patients with tumor unable to be completely resected, 2 courses of ifos- famide, Ver and VP-16 as pre-surgery therapy were conducted. No radiation therapy was performed to all stage Ⅰ and FH stage Ⅱ patients. No randomized comparisons were attempted. Results Among 54 patients, pathological examination showed 39 of FH, 4 of UFH, 5 of clear cell and 1 of rhabdoid and the other 5 undetermined. Nineteen patients were stage Ⅰ , 7 stage Ⅱ , 19 stage Ⅲ , 7 stage Ⅳ and 2 stage Ⅴ. Fifty-one patients reached complete response （CR, 94.4%）. Nine patients relapsed anti 3 of them reached second CR while 6 refused further treatment. No therapy related death happened. Estimated 5-year overall survival rate （SR） is 83.9% and event-free survival is 75.7%. Conclusions Muhi-disciplinary team work model and protocol WT-99 are safe and effective for childhood Wilms＇ tumor.