儿童横纹肌肉瘤分层化学治疗疗效及生存因素分析

Efficacy of stratified chemotherapy for rhabdomyosarcoma in children and factors related to prognosis of patients

目的探讨儿童横纹肌肉瘤(rhabdomyosarcoma, RMS)的分层化学治疗疗效及影响预后的相关因素。方法回顾单中心治疗的34例RMS患儿临床资料,分析各临床分层治疗组的疗效,并对诸临床因素与生存的相关性进行单因素分析。结果 34例患儿接受手术、化疗,其中30例接受放疗。临床分为低危组、中危组和高危组,对应于相应强度的化疗方案。本分组与国际横纹肌肉瘤研究组(the Intergroup Rhabdomyosarcoma Study Group, IRSG)分组标准相比略有差异,即部分IRSG低危组患儿划归至中危治疗组。低危组3例,完全缓解;中危组21例,11例完全缓解,8例部分缓解;高危组10例,3例完全缓解,1例部分缓解,最终4例死亡。各组5年总体生存率(overall survival, OS)分别是100%、78%和35%。手术后存在肉眼残留与无肉眼残留患儿5年OS分别为25%、90%(P<0.05)。诊断时是否发生远处转移患儿5年OS为35%、82%(P<0.05)。结论本研究的低危组有很好预后,适用于低强度化疗方案;将部分IRSG低危组患儿调整至中危组提高化疗强度有较好的长期生存率;高危组需探索新的治疗手段。是否存在远处转移及手术是否完整切除仍是影响预后的主要因素。

Objective To analyze the efficacy of stratified chemotherapy for rhabdomyosarcoma(RMS)in children and the factors related to prognosis of patients.Methods The clinical data of 34 RMS children treated by stratified chemotherapy were reviewed.The correlation between clinical factors and survival was analyzed by univariate regression method.Results Thirty four patients were classified into low-risk group(n=3),intermediate-risk group(n=21)and high-risk group(n=10)according the Intergroup Rhabdomyosarcoma Study Group(IRSG)with some modification,receiving corresponding intensity chemotherapy regimens.Three cases of low-risk group all had complete remission.Of 21 cases in intermediate-risk group,11 had complete remission,8 partial remission.In high-risk group,3 cases had complete remission and 1 case partial remission.The 5-year overall survival(OS)in low-,intermediate-and high-risk groups was 100%,78%and 35%,respectively.The 5-year OS was 25%and 90%in patients with or without gross residual tumor after operation,35%and 82%in patients with or without distant metastasis.Conclusion The low-risk group has a good prognosis and can be treated with low intensity chemotherapy.The exploration of new therapeutic strategies are needed for high-risk patients.Gross residual disease and distant metastasis are still related to poor prognosis.