摘要
目的分析儿童非转移性横纹肌肉瘤(RMS)分层治疗的疗效及预后影响因素。方法研究对象为2005年1月至2016年12月上海交通大学医学院附属上海儿童医学中心新诊断的非转移性RMS患儿,共129例。根据RMS术前、术后分期及病理类型将患儿分为低危组、中危组和高危组,综合治疗方案包括根据分组采用不同强度化疗、放疗治疗及适时肿瘤根治性切除术。采用Kaplan-Meier生存分析法对RMS进行生存率及影响因素分析。结果129例患儿中,最终119例进入统计,发病年龄7~191个月,中位发病年龄48个月,无事件患儿中位随访时间40个月,所有患儿中位随访时间36个月。实际分组中低危组8例,中危组49例,高危组62例。5年总体生存(OS)率为(92.1±2.9)%,5年无事件生存(EFS)率为(76.5±4.4)%;低危组、中危组及高危组患儿5-EFS均>70%,但组间比较差异无统计学意义(χ^2=2.679,P=0.262);单因素分析发现:发病年龄≤1岁或≥10岁、术前分期、早期手术完整切除明显影响预后,差异均有统计学意义(均P<0.05),而性别、病理分型、原发部位与EFS无明显相关性(均P>0.05)。腺泡型RMS(ARMS)患儿中叉头转录因子1(FOXO1)阳性者5-EFS明显低于FOXO1阴性者[(56.3±14.8)%比(83.3%±15.2)%],差异有统计学意义(χ^2=4.588,P=0.028)。结论RMS治疗需严格做好危险度分组;低危组相对中危组预后较差,方案需改进;中危组患儿可考虑优化化疗药物并适度减轻化疗剂量;FOXO1阳性可纳入危险度分组中进行分层治疗。
Objective To assess the efficacy of stratified treatment of pediatric non-distant metastatic rhabdomyosarcoma (RMS). Methods A retrospective review was conducted in 129 pediatric patients with non-distant metastatic RMS between January 2005 and December 2016 at Shanghai Children′s Medical Center Affiliated to Shanghai Jiaotong University School of Medicine.According to their pathological types, TNM stages and postoperative pathologic staging, the 129 patients were grouped a low-risk group, an intermediate-risk group and a high-risk group.Multimodality therapies were applied to all patients including chemotherapy, surgery and radiotherapy.The overall survival (OS) and event-free survival (EFS) rates were analyzed by using the Kaplan-Meier method. Results Of 129 patients, 119 cases were included in this study.In 119 patients, the age of onset for the RMS ranged from 7 to 191 months, with the median onset age of 48 months.The median follow-up time was 40 months for event-free patients with RMS, and 36 months for all the 119 patients.The 5-year OS and EFS for all patients were (92.1±2.9)% and (76.5±4.4)%, respectively.While the 5-year EFS for patients in the low-risk group, intermediate-risk group and high-risk group were all above 70%, and the difference among the three groups was not statistically significant (χ^2=2.679, P=0.262). A subsequent univariate analysis revealed that the onset age for RMS (≤1 year old or≥10 years old), TNM stage and postoperative pathologic stage were important predictors of EFS with statistical significance (all P<0.05), while gender, pathological type and primary site of RMS did not exhibit any significant impact on 5-EFS (all P>0.05). The 5-year EFS of RMS patients with Forkhead Box Protein O1(FOXO1)-positive was significantly lower than that of FOXO1-negative patients [(56.3%±14.8)% vs.(83.3±15.2)%], and the difference was statistically significant (χ^2=4.588, P=0.028). Conclusions It is important that the stratification treatment should be strictly implemented on RMS patients.First, further improvement is necessary for the treatment of patients in the low-risk group due to their poorer prognosis compared to that of their intermediate-risk counterparts, for whom one feasible option is to reduce the dose of chemotherapy drug.Furthermore, FOXO1 can be used as an indicator for poor prognosis, where stratified treatment is necessary for pediatric patients with RMS.
作者
韩亚丽
汤静燕
潘慈
胡文婷
高怡瑾
Han Yali;Tang Jingyan;Pan Ci;Hu Wenting;Gao Yijin(Shanghai Children's Medical Center Affiliated to Shanghai Jiaotong University School of Medicine,Shanghai 200127,China)
出处
《中华实用儿科临床杂志》
CSCD
北大核心
2019年第15期1146-1150,共5页
Chinese Journal of Applied Clinical Pediatrics
关键词
横纹肌肉瘤
分层治疗
预后
儿童
Rhabdomyosarcoma
Stratification treatment
Prognosis
Child
