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一项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。1.日本横纹肌肉瘤治疗的调查1）对1991年至2002年期间治疗的331例病例进行回顾性病历分析。总的5年生存率为60.7%，比IRS低10-20%。2）既往大剂量化疗试验的随访研究。+ 干细胞移植没有提供任何结论。3）儿科外科医生对手术切缘、根治性手术的时机和淋巴结切除的范围没有达成共识。2.横纹肌肉瘤新临床试验的发展基于上述调查，新的临床试验是在儿童中准备的。1）手术和放射治疗指南是根据IRS介绍的。2）建立病理学和PAX 3，7-FKHR嵌合基因的中心审查系统。3）根据风险分类制定研究方案。对于低A和B，VAC剂量和治疗时间减少。中间组采用儿童剂量调整的VAC。大剂量化疗。高危组行干细胞治疗。研究的主要终点确定为3年无事件，无进展生存期。4）每个研究都建立了数据中心;科比TRI中心为低风险，JRSG中心办公室为中等风险，国家癌症中心为高风险方案。5）建立了儿童肿瘤组织库，用于进一步的基础研究。6）临床试验于2004年开始，共有24例病例入组。其中14例符合临床试验条件。

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.