小儿肿瘤协会儿童肾母细胞瘤WT-2003协作方案初步报告

Preliminary results of WT-2003 Wilms’ tumor protocol by the chinese pediatric oncology society

目的 探讨建立儿童肿瘤协作组可能性，改善我国儿童肾母细胞瘤预后。方法 在中国小儿肿瘤协会下建立儿童肿瘤协作组，制定儿童肾母细胞瘤WT-2003协作方案，方案包括诊断与分期标准，分组标准、各组包括外科手术、内科化疗、选择性放疗的治疗规则。协作组WT-2003方案先在上海儿童医学中心试行20例，证明安全性和有效性后2003年起在协作组内应用。根据患儿分组术后接受18-24周不同强度化疗。方案 规定Ⅰ期及Ⅱ期病理分型为预后良好型不放疗，其余病例治疗均包含有手术、不同强度的放疗和化疗，估计手术不能完全切除时给予2个疗程术前化疗。结果 15家医院参加协作组，7家医院上报汇总资料，累计病例52例。最大年龄12岁，最小6个月，中位29个月。病理分型为预后良好型（FH）35例，预后不良型（UFH）8例，透明细胞肉瘤（CS）5例，肾未分化肉瘤（US）2例，横纹肌样肉瘤（RS）1例，典型肾母细胞瘤特征影像学诊断1例（拒绝手术）。分期：Ⅰ期17例，Ⅱ期11例，Ⅲ期17例，Ⅳ期6例，Ⅴ期1例。48例获完全缓解，占92．3％。目前无肿瘤生存45例，占本组病例的86．5％，时间为3～72个月，中位13个月。结论 中国儿童肿瘤多中心协作临床研究有可行性，通过这一方式可较快积累病例，使治疗更合理，提高我国儿童肿瘤整体诊治水平，本组患儿所采用诊断治疗方案有效，安全性好。

Objective To establish a multi-center Wilms＇ Tumor cooperative group in China in association with the Chinese Pediatric Society. Methods The diagnosis and staging criteria, the surgery, chemotherapy and radiation were standardized in the WT-2003 Wilms tumor protocol. This protocol was first tested in Shanghai Children＇s Medical Center. After the approval of its efficacy and safety, the protocol was adopted by the cooperative group. The protocol was based on the National Wilms＇ Tumor Study （NWTS） criteria of U. S. A, aiming to reduce treatment of low-stage, favorablehistology （FH） tumors without impairing survival and to improve prognosis of stage Ⅲand Ⅳ （FH） and unfavorable-histology （UFH） tumors with more intensive chemotherapy. Chemotherapy duration ranges from 18 weeks to 24 weeks with different intensity depends on individual criteria. For those unresectable tumors, two courses of Ifosfamide, Vcr and VP-16 are recommended as pre-surgery therapy. No radiation therapy is recommended for all stage Ⅰ and FH stage Ⅱ. No randomized comparison was attempted. Results Patients were recruited from 7 hospitals. Four patients were excluded from the final analysis because they did not meet the selection criteria. A total 52 patients, with age between 7 months to 12 years old （median 29 months） were enrolled. Pathology revealed 35 FH, eight UFH, five clear cell,one rhabdoid and two un-differentiated. There were seventeen patients with stage Ⅰ, eleven with stage Ⅱ, seventeen with stage Ⅲ, six with stage Ⅳ and one stage Ⅴtumors. Forty-eight （93%） patients achieved complete remission （CR）. One relapsed after CR 24 months and reached second CR after intensive chemotherapy and ABMT. No therapy-related death occurred. Disease free survival （DFS） is seen in 86% of patients and its duration ranged from 3 to 72 months （average = 13 months）. All but five have stopped the treatment according to the protocol. Conclusions Multi-centercooperative group model for childhood cancer is feasible in China. The medical care for childhood cancer can be improved through this model.