Objective: To analyze the treatment outcomes and prognoses of children with head and neck non-parameningeal rhabdomyosarcoma (HNnPM RMS). Methods: A retrospective analysis was performed on the clinical data of children with HNnPM RMS admitted to Beijing Children's Hospital from September 2012 to September 2022. The clinical features, comprehensive treatment modes and prognoses of the patients were analyzed. The overall survival rate (OS) and event free survival rate (EFS) were calculated using the Kaplan-Meier method, and univariate analysis was performed using the Log-rank test. Results: A total of 70 children were included in this study, 38 males and 32 females, with a median age of 47 months (2-210 months). Pathological subtypes including the embryonal in 27 cases, the alveolar in 36 cases and the spindle cell and sclerosing in 7 cases. Thirty children (83.3%) with alveolar type were positive for FOXO1 gene fusion. All 70 children underwent chemotherapy, including 38 with neoadjuvant chemotherapy and 32 with adjuvant chemotherapy. Sixty of 70 children underwent surgery, of whom, 10 underwent two or more surgeries. There were 63 children underwent radiotherapy, including 54 with intensity-modulated radiation therapy, 4 with particle implantation and 5 with proton therapy. The median follow-up was 45 (5-113) months, the 5-year OS was 73.2%, and the 5-year EFS was 57.7%. Univariate analysis showed lymph node metastasis (χ2=5.022, P=0.025), distant metastasis (χ2=8.258, P=0.004), and high Intergroup Rhabdomyosarcoma Study (IRS) group (χ2=9.859, P=0.029) as risk factors for poor prognosis. Before June 2016, the 5-year OS based on BCH-RMS-2006 scheme was 63.6%, and after 2016, the 5-year OS based on CCCG-RMS-2016 scheme was 79.6%. Conclusion: Multidisciplinary combined standardized treatment can offer good treatment outcome and prognosis for children with HNnPM RMS. Local control is a key to the efficacy of comprehensive treatment.
目的： 总结分析头颈部非脑膜旁区横纹肌肉瘤（head and neck non-parameningeal rhabdomyosarcoma，HNnPM RMS）患儿的治疗效果及预后特点。 方法： 回顾性分析北京儿童医院自2012年9月至2022年9月收治的HNnPM RMS患儿的临床资料，分析患儿的临床特点、综合治疗模式和预后情况。总生存率及无事件生存率采用Kaplan‐Meier法计算，单因素分析采用Log‐rank检验。 结果： 共70例患儿纳入本研究，男38例，女32例，中位年龄47个月（范围：2~210个月）。病理亚型中胚胎型27例，腺泡型36例，梭形细胞/硬化型7例；腺泡型中FOXO1基因融合阳性30例（83.3%）。70例患儿均进行了化疗，新辅助化疗38例，辅助化疗32例。60例患儿进行了手术治疗，其中10例经历了二次及以上手术。63例患儿进行了放疗，其中调强适形54例，粒子植入4例，质子治疗5例。中位随访时间45个月（范围：5~113个月），5年总生存率为73.2%，5年无事件生存率为57.7%。单因素分析显示淋巴结转移（χ2=5.022，P=0.025）、远处转移（χ2=8.258，P=0.004）、术后病理分组级别较高（χ2=9.859，P=0.020）是预后不良的危险因素。2016年6月前基于北京儿童医院2006版横纹肌肉瘤治疗方案（BCH-RMS-2006）5年总生存率为63.6%，2016年6月后基于中国儿童及青少年横纹肌肉瘤诊疗建议（CCCG-RMS-2016）5年总生存率为79.6%。 结论： HNnPM RMS在多学科联合规范化治疗的基础上，可以实现较好的预后。综合治疗中局部控制是治疗的关键。.