脑膜旁横纹肌肉瘤 (PM-RMS) 约占所有横纹肌肉瘤 (RMS) 病例的 20%。目前，PM-RMS的研究大部分集中在欧洲和美国，国内的研究非常有限。本研究旨在分析北京儿童医院 (BCH) 连续两次治疗方案中儿童和青少年 PM-RMS 的临床结果和预后因素。共有 80 名年龄不超过 18 岁、既往未接受过治疗的 PM-RMS 患者接受过治疗。两个连续方案[即BCH-RMS-2006方案或中国儿童癌症研究组(CCCG)-RMS-2016方案]下的治疗纳入统计分析。采用Kaplan-Meier法进行生存分析，采用Cox回归进行单因素和多因素分析。 在纳入研究的80例患者中，69例（86.2%）存在脑膜侵犯（MI）。在这 69 名 MI 患者中，18 名（22.5%）患有颅神经麻痹（CNP），64 名（80.0%）患有颅底骨侵蚀（CBBE），25 名（31.3%）患有颅内延伸（ICE），2 名（2.5%）患有颅内延伸（ICE）。 ）脑脊液（CSF）肿瘤细胞呈阳性。中位随访时间为 20.5 个月（范围 5-100 个月）。整个队列的 5 年总生存率 (OS) 和无进展生存率 (PFS) 分别为 51.7% 和 45.6%。接受BCH-RMS-2006方案（18/80，22.5%）和CCCG-RMS-2016方案（62/80，77.5%）的患者5年OS率分别为33.3%和57.0% (P<0.05)，而这些患者的PFS率分别为22.2%和53.6%(P<0.05)。对于伴有 MI 的 PM-RMS 患者，接受新旧方案的患者 5 年 OS 率分别为 21.4% 和 52.7%，5 年 PFS 率分别为 14.3% 和 51.1%（ P<0.05）。手术切除的范围对生存没有显着影响。多变量分析显示，CBBE与ICE并存、未放疗、诱导化疗反应不佳以及BCH-RMS-2006方案是影响PFS和OS的危险因素。在本研究检查的患者中，患有PM-带有 CBBE 并伴有 ICE 的 RMS 预后最差。 MI患者受益于强化化疗联合放射治疗，但手术效果非常有限。2024转化儿科。版权所有。

Parameningeal rhabdomyosarcoma (PM-RMS) accounts for about 20% of all rhabdomyosarcoma (RMS) cases. At present, most research on PM-RMS has been conducted in Europe and the United States of America, and research in China has been very limited. This study sought to analyze the clinical outcomes and prognostic factors of PM-RMS in children and adolescents from two consecutive protocols at Beijing Children's Hospital (BCH).A total of 80 patients aged up to 18 years with previously untreated PM-RMS who had received treatment under two consecutive protocols [i.e., either the BCH-RMS-2006 protocol or the Chinese Children Cancer Group (CCCG)-RMS-2016 protocol] were included in the statistical analysis. The Kaplan-Meier method was used for the survival analysis, and Cox regression was used for the univariate and multivariate analyses.Of the 80 patients enrolled in the study, 69 (86.2%) had meningeal invasion (MI). Of these 69 MI patients, 18 (22.5%) had cranial nerve palsy (CNP), 64 (80.0%) had cranial base bone erosion (CBBE), 25 (31.3%) had intracranial extension (ICE), and 2 (2.5%) had positive cerebrospinal fluid (CSF) tumor cells. The median follow-up time was 20.5 months (range, 5-100 months). The 5-year overall survival (OS) and progression-free survival (PFS) rates for the entire cohort were 51.7% and 45.6%, respectively. The 5-year OS rates of the patients who received the BCH-RMS-2006 protocol (18/80, 22.5%) and the CCCG-RMS-2016 protocol (62/80, 77.5%) were 33.3% and 57.0%, respectively (P<0.05), while the PFS rates of these patients were 22.2% and 53.6%, respectively (P<0.05). In relation to the PM-RMS patients with MI, the 5-year OS rates were 21.4% and 52.7%, and the 5-year PFS rates were 14.3% and 51.1% for the patients who received the old and new regimens, respectively (P<0.05). The extent of surgical resection had no significant effect on survival. The multivariate analysis showed that the coexistence of CBBE and ICE, no radiotherapy, a poor response to induction chemotherapy, and the BCH-RMS-2006 protocol were risk factors affecting PFS and OS.Of the patients examined in this study, those with PM-RMS with CBBE accompanied by ICE had the worst prognosis. The patients with MI benefited from intensive chemotherapy combined with radiation therapy, but the effect of surgery was very limited.2024 Translational Pediatrics. All rights reserved.