Pachnis A, Pritchard J, Gaze M, Levitt G, Michalski A
Department of Haematology and Oncology, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom.
Med Pediatr Oncol. 1998 Sep;31(3):150-2. doi: 10.1002/(sici)1096-911x(199809)31:3<150::aid-mpo4>3.0.co;2-a.
Current treatment of stage III favorable histology (FH) Wilms tumor is surgery, radiotherapy to residual disease, and "triple" chemotherapy (vincristine, dactinomycin, and doxorubicin) for 12 months. This study tests the hypothesis that some stage III patients, especially very young children with minimal residual abdominal disease, might be successfully treated without radiotherapy, thereby avoiding the adverse late effects associated with radiotherapy.
From 1984, radiotherapy was omitted from the treatment of 8 carefully selected children who were younger than 3 years of age at diagnosis with stage III Wilms tumor by virtue of microscopic residual disease after surgery and whose lymph nodes were not involved by tumors. They were followed with bimonthly abdominal ultrasound examinations to assess local control.
Follow-up is now from 2 to 12 years (median 6 years) and 7 of the 8 children are alive and well with no abdominal recurrence. One child relapsed in the lungs and despite further treatment died of progressive disease. The disease-free survival (DFS) and overall survival (OS) are therefore both 87.5%.
The DFS and OS in this admittedly small sample are consistent with the survival rates for stage III FH Wilms tumor in the first United Kingdom Children's Cancer Study Group (UKCCSG), North American (NWTS), and European (SIOP) Wilms Tumor studies, Larger numbers of patients are needed to determine whether or not this treatment approach is generally applicable, but we conclude that some children in this stage III "substage" may be treated successfully without radiotherapy.
目前对Ⅲ期组织学类型良好（FH）的肾母细胞瘤的治疗方法是手术、对残留病灶进行放疗以及进行为期12个月的“三联”化疗（长春新碱、放线菌素D和阿霉素）。本研究检验了这样一个假设，即一些Ⅲ期患者，尤其是腹部残留病灶极少的幼儿，或许可以在不进行放疗的情况下成功治愈，从而避免放疗带来的不良晚期效应。
自1984年起，对于8名经仔细挑选的儿童，在其诊断为Ⅲ期肾母细胞瘤时（诊断依据为术后显微镜下可见残留病灶且淋巴结未受肿瘤累及），若年龄小于3岁，则在治疗中省略放疗。每两个月对他们进行腹部超声检查以评估局部控制情况。
目前随访时间为2至12年（中位时间为6年），8名儿童中有7名存活且状况良好，无腹部复发。1名儿童肺部复发，尽管接受了进一步治疗，但仍死于疾病进展。因此，无病生存率（DFS）和总生存率（OS）均为87.5%。
在这个样本量较小的研究中，DFS和OS与英国儿童癌症研究组（UKCCSG）、北美（NWTS）以及欧洲（SIOP）的肾母细胞瘤研究中Ⅲ期FH肾母细胞瘤的生存率一致。需要更多患者来确定这种治疗方法是否普遍适用，但我们得出结论，一些处于Ⅲ期“亚阶段”的儿童或许可以在不进行放疗的情况下成功治愈。
