儿童晚期肾母细胞瘤12例的疗效与预后

Clinical efficacy and prognosis with advanced Wilms＇ tumor in children

目的总结2009年2月至2012年6月收治的12例经病理确诊及I临床分期为晚期的儿童肾母细胞瘤的诊疗资料，分析临床疗效及总结诊疗经验。方法12例肾母细胞瘤患儿中，男10例，女2例；中位年龄2．54岁（9个月～15岁）。根据美国国家肾母细胞瘤研究组（NWTSG）制定的临床病理分期标准，12例患儿中，Ⅲ期5例，Ⅳ期7例。同时根据NWTSG的病理组织分化程度不同，12例患儿中，组织分化良好型及组织分化不良型各6例。根据NWTSG制定诊疗标准选择不同化疗及放疗方案。对于单纯手术及放疗、化疗未达到完全缓解并反复复发病例可行自体外周血造血干细胞移植（APBSCT）治疗。分析12例患儿晚期肾母细胞瘤的临床特点、疗效及预后分析。结果1．首发症状：腹部包块8例（66．6％），发热腹痛2例（12．7％），血尿2例（12．7％）。2．随访至2013年1月，中位随访时间31．5个月（8～131个月），失访放弃治疗1例，获随访ll例患儿完全缓解4例，部分缓解1例，病情进展5例，复发1例，其中死亡4例（即部分缓解及病情进展病例）。总生存率63．63％（7／11例），病死率36．37％（4／11例），无事件生存率为36．37％（4／11例），其中1例Ⅳ期患儿常规放、化疗后3次复发，经马法兰（Melphalan，M）＋卡铂（Carboplatin，C）＋足叶乙甙（Etoposide，E）即CEM方案结合APBSCT治疗后达到完全缓解。3年估计生存率达到51．4％。结论儿童肾母细胞晚期预后较差，病死率仍较高。APBSCT治疗可作为晚期病例的治疗方案之一。

Objective To analyze the clinical data of 12 children with advanced Wlims＇ tumor in children from Feb. 2009 to Jan. 2012. All cases were diagnosed by pathology and to analysis the clinic efficacy and treatment ex- perience were analyzed. Methods Of 12 patients, 10 cases were male and 2 cases were female. The medium age of 12 patients was 2.54 years old（9 months - 15 years old）. According to pathological stage and clinical stage of The Natio- nal Wilms＇ Tumor Study Group（NWTSG） ,5 cases belonged to stage HI ,and 7 cases stage IV. Six cases were well-diffe- rentiated tissue type, and 6 cases were poorly differentiated tissue type according to NWTSG. In all patients, different ways of chemotherapy and radiotherapy were selected according to clinical stage and tissue type differentiation. If a pa- tient had repeated recurrence after common surgery and chemotherapy, would treated by autologous peripheral blood stem cell transplantation（APBSCT）. Statistic analysis was used to analyze the clinical characters and efficacy and prog- nosis for 12 patients. Results 1. Initial symptoms：in 12 cases, 8 cases presented abdominal mass （66.6%）, 2 cases with abdominal pain and fever（ 12.7% ） ,and 2 cases with hematuria（ 12.7% ）. 2. Eleven cases followed up to Jan. 2013, the medium time was 3 I. 5 months（8 -131 months）. Of 12 cases, 1 case give up therapy and follow-up and 11 cases were followed up. Of those 11 cases followed-up,4 cases had complete remission（CR） ,and 1 ease had remission in part（PR） ,the conditions of 5 cases were progressively worse, 1 case replapsed, and 4 patients died. Total survival rate was 63.63% （7/11 cases） ,and mortality was 36.37% （4/11 cases） ,and free survival rate was 36.37% （4/11 ca- ses）, of that, 1 patient of stage IV, relapsed 3 times after common radiotherapy and chemotherapy, achieved complete re- mission after high dose chemotherapy（ Melphalan ＋ Carboplatin ＋ Etoposide, CEM） and APBSCT. The estimated 3-year- survival rate was 51.4%. Conclusions The prognosis of advanced Wilms＇ tumor is poor, and the mortality is still high. High dose chemotherapy with APBSCT may be a valuable method for advanced cases.