Objective: To analyze the clinical characteristics of children with head and neck rhabdomyosarcoma (RMS) and to summarize the mid-long term efficacy of Beijing Children's Hospital Rhabdomyosarcoma 2006 (BCH-RMS-2006) regimen and China Children's Cancer Group Rhabdomyosarcoma 2016 (CCCG-RMS-2016) regimen. Methods: A retrospective cohort study. Clinical data of 137 children with newly diagnosed head and neck RMS at Beijing Children's Hospital, Capital Medical University from March 2013 to December 2021 were collected. Clinical characteristic of patients at disease onset and the therapeutic effects of patients treated with the BCH-RMS-2006 and CCCG-RMS-2016 regimens were compared. The treatments and outcomes of patients with recurrence were also summarized. Survival analysis was performed by Kaplan-Meier method, and Log-Rank test was used for comparison of survival rates between groups. Results: Among 137 patients, there were 80 males (58.4%) and 57 females (41.6%), the age of disease onset was 59 (34, 97) months. The primary site in the orbital, non-orbital non-parameningeal, and parameningeal area were 10 (7.3%), 47 (34.3%), and 80 (58.4%), respectively. Of all patients, 32 cases (23.4%) were treated with the BCH-RMS-2006 regimen and 105 (76.6%) cases were treated with the CCCG-RMS-2016 regimen. The follow-up time for the whole patients was 46 (20, 72) months, and the 5-year progression free survival (PFS) and overall survival (OS) rates for the whole children were (60.4±4.4)% and (69.3±4.0)%, respectively. The 5-year OS rate was higher in the CCCG-RMS-2016 group than in BCH-RMS-2006 group ((73.0±4.5)% vs. (56.6±4.4)%, χ2=4.57,P=0.029). For the parameningeal group, the 5-year OS rate was higher in the CCCG-RMS-2016 group (61 cases) than in BCH-RMS-2006 group (19 cases) ((57.3±7.6)% vs. (32.7±11.8)%, χ2=4.64,P=0.031). For the group with meningeal invasion risk factors, the 5-year OS rate was higher in the CCCG-RMS-2016 group (54 cases) than in BCH-RMS-2006 group (15 cases) ((57.7±7.7)% vs. (30.0±12.3)%, χ2=4.76, P=0.029). Among the 10 cases of orbital RMS, there was no recurrence. In the non-orbital non-parameningeal RMS group (47 cases), there were 13 (27.6%) recurrences, after re-treatment, 7 cases survived. In the parameningeal RMS group (80 cases), there were 40 (50.0%) recurrences, with only 7 cases surviving after re-treatment. Conclusions: The overall prognosis for patients with orbital and non-orbital non-parameningeal RMS is good. However, children with parameningeal RMS have a high recurrence rate, and the effectiveness of re-treatment after recurrence is poor. Compared with the BCH-RMS-2006 regimen, the CCCG-RMS-2016 regimen can improve the treatment efficacy of RMS in the meningeal region.
目的： 分析头颈部横纹肌肉瘤（RMS）的临床特征，总结北京儿童医院横纹肌肉瘤2006（BCH-RMS-2006）方案和中国儿童癌症协作组横纹肌肉瘤2016（CCCG-RMS-2016）方案的中长期随访数据。 方法： 回顾性队列研究。收集首都医科大学附属北京儿童医院2013年3月至2021年12月收治的新诊断的头颈部RMS 137例患儿的临床资料，分析纳入BCH-RMS-2006和CCCG-RMS-2016两个方案患儿的临床特征，比较两组患儿的生存数据，总结复发患儿的再治疗情况和结局，采用Kaplan-Meier方法进行生存分析，组间生存率比较采用Log-Rank检验。 结果： 137例RMS患儿中男80例（58.4%）、女57例（41.6%），就诊年龄59（34，97）月龄。发生在眼眶区、非眶非脑膜旁区、脑膜旁区的分别为10例（7.3%）、47例（34.3%）、80例（58.4%）。32例（23.4%）化疗方案为BCH-RMS-2006，105例（76.6%）患儿化疗方案为CCCG-RMS-2016。137例患儿随访时间为46（20，72）个月，5年无进展生存率（PFS）及总生存率（OS）分别为（60.4±4.4）%和（69.3±4.0）%。RMS-BCH-2006方案组与CCCG-RMS-2016组患儿5年OS分别为（56.6±4.4）% 及（73.0±4.5）%，差异有统计学意义（χ2=4.57，P=0.029）；脑膜旁区RMS应用BCH-RMS-2016方案（19例）与CCCG-RMS-2016（61例）患儿5年OS分别为（32.7±11.8）%及（57.3±7.6）%，差异有统计学意义（χ2=4.64，P=0.031）。合并脑膜侵犯危险因素组应用BCH-RMS-2006方案组（15例）与CCCG-RMS-2016方案组（54例）患儿5年OS分别为（30.0±12.3）%及（57.7±7.7）%，差异有统计学意义（χ2=4.76，P=0.029）。原发眼眶区的10例患儿无复发；原发非眶非脑膜旁区的47例患儿复发进展13例（27.6%），经过再治疗后7例生存；原发脑膜旁区的80例患儿复发40例（50.0%），经再次治疗后仅7例生存。 结论： 眼眶及非眶非脑膜旁区RMS患儿总体预后良好。脑膜旁区RMS复发率高且复发后再治疗效果差。与BCH-RMS-2006方案相比，CCCG-RMS-2016方案可以提高脑膜旁区RMS的疗效。.