The development of the new clinical trial based on the quality assurance of treatment process in the childhood rhabdomyosarcoma.

本研究的目的是准备新的儿童横纹肌肉瘤临床试验，以改善日本横纹肌肉瘤的不良预后。对日本1991 ~ 2002年间331例横纹肌肉瘤的治疗情况进行回顾性分析。总体5年生存率为60.7%，比irs低10-20%。2)对既往大剂量化疗试验的随访研究。+干细胞移植未提供任何结论。3)小儿外科医生对手术切缘、根治性手术时机、淋巴结切除范围等尚未达成共识。新的横纹肌肉瘤临床试验的发展基于上述调查，在儿童中准备了新的临床试验。1)在此基础上介绍了手术和放疗指南。2)建立了PAX3 - 7-FKHR的病理中心评价系统和嵌合基因。3)根据风险分级制定研究方案。低A和低B组，VAC剂量和治疗时间缩短。中间组采用儿童剂量调整的VAC。大剂量化疗。高危组采用干细胞抢救。研究的主要终点确定为3年无事件，无进展生存期。4)各研究建立数据中心；神户TRI低风险中心，JRSG中等风险中心办公室和国家癌症中心高风险协议。5)建立儿童肿瘤组织库，进行进一步的基础研究。6) 2004年开始临床试验，入组24例。其中14例符合入组临床试验条件。

The purpose of the present study is to prepare the new clinical trial for childhood rhabdomyosarcoma to improve poor outcome of the rhabdomyosarcoma in Japan.1.The survey of the treatment of the rhabdomyosarcoma in Japan1)331 cases treated during the period of 1991 to 2002 were assessed by the retrospective chart review. Overall 5 year survival revealed 60.7% that was 10-20% lower that IRS.2)The follow up study of the previous trial for high-dose chemo. + stem cell transplantation did not provide any conclusion.3)There is no consensus among pediatric surgeons for the surgical margin, timing of the radical operation and the range of lymph node resection.2.Development of the new clinical trial for the rhabdomyosarcoma Based upon the survey described above, the new clinical trial was prepared in children.1)The guideline for surgery and radiation was introduced based on the IRS.2)The system for central review for pathology and the chimeric gene of PAX3,7-FKHR were developed.3)Study protocol were prepared according to the risk classification. For Low A and B, dose of VAC and its treatment period were diminished. For intermediate group, VAC with dose adjustment for children was adopted. High dose chemo. and stem cell rescue was applied for high risk group. Primary endpoint of the study is determined as 3 year event free, no progression survival.4)Data center was established in each study ; Kobe TRI center for Low Risk, JRSG central office for Intermediate Risk and National Cancer Center for High Risk protocol.5)Tissue bank of the childhood tumor was developed for further basic study.6)The clinical trial started in 2004 and 24 cases were entered. Among these, 14 cases were qualified to enrolle for clinical trial.