Duan C, He S D, Wang S C, Jin M, Zhao W, Wang X S, Liu Z K, Yu T, He L J, Wang X M, Cui C Y, Ni X, Su Y
Department of Medical Oncology, Pediatric Oncology Center,Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, National Key Clinical Discipline of Pediatric Oncology, Laboratory for Clinical Medicine, Capital Medical University, Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing100045, China.
Department of Otorhinolaryngology, Head and Neck Surgery, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing100045, China.
Zhonghua Er Ke Za Zhi. 2025 Jan 2;63(1):62-69. doi: 10.3760/cma.j.cn112140-20241115-00833.
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. 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. 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 patients 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)% (56.6±4.4)%, =4.57,=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)% (32.7±11.8)%, =4.64,=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)% (30.0±12.3)%, =4.76, =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. 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.
分析头颈部横纹肌肉瘤(RMS)患儿的临床特征,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集团横纹肌肉瘤2016(CCCG-RMS-2016)方案的中长期疗效。一项回顾性队列研究。收集了2013年3月至2021年12月在首都医科大学附属北京儿童医院新诊断的137例头颈部RMS患儿的临床资料。比较了疾病初发时患者的临床特征以及接受BCH-RMS-2006和CCCG-RMS-2016方案治疗患者的疗效。还总结了复发患者的治疗及转归情况。采用Kaplan-Meier法进行生存分析,用Log-Rank检验比较组间生存率。137例患者中,男性80例(58.4%),女性57例(41.6%),发病年龄为59(34,97)个月。眼眶、非眼眶非脑膜旁和脑膜旁区域的原发部位分别为10例(7.3%)、47例(34.3%)和80例(58.4%)。所有患者中,32例(23.4%)接受BCH-RMS-2006方案治疗,105例(76.6%)接受CCCG-RMS-2016方案治疗。全体患者的随访时间为46(20,72)个月,全体患者的5年无进展生存率(PFS)和总生存率(OS)分别为(60.4±4.4)%和(69.3±4.0)%。CCCG-RMS-2016组的5年OS率高于BCH-RMS-2006组((73.0±4.5)% (56.6±4.4)%,χ² =4.57,P =0.029)。对于脑膜旁组,CCCG-RMS-2016组(61例)的5年OS率高于BCH-RMS-2006组(19例)((57.3±7.6)% (32.7±11.8)%,χ² =4.64,P =0.031)。对于有脑膜侵犯危险因素的组,CCCG-RMS-2016组(54例)的5年OS率高于BCH-RMS-2006组(15例)((57.7±7.7)% (30.0±12.3)%,χ² =4.76,P =0.029)。10例眼眶RMS患者均无复发。在非眼眶非脑膜旁RMS组(47例)中,有13例(27.6%)复发,再次治疗后7例存活。在脑膜旁RMS组(80例)中,有40例(50.0%)复发,再次治疗后仅7例存活。眼眶和非眼眶非脑膜旁RMS患者的总体预后良好。然而,脑膜旁RMS患儿复发率高,复发后再次治疗的效果较差。与BCH-RMS-2006方案相比,CCCG-RMS-2016方案可提高RMS在脑膜区域的治疗效果。