Zheng Jing-Jie, Zhang Gui-Jun, Huo Xu-Lei, Wang Liang, Hao Shu-Yu, Zhang Li-Wei, Wu Zhen, Wu Yu-Mei, Zhang Jun-Ting, Li Da
1Department of Gynecologic Oncology, Beijing Obstetrics and Gynecology Hospital, Capital Medical University; and.
2Department of Neurosurgery, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
J Neurosurg. 2019 Sep 13;133(5):1302-1312. doi: 10.3171/2019.6.JNS19752. Print 2020 Nov 1.
Primary intracranial rhabdomyosarcoma (PIRMS) is rare, and the effects of the treatment strategy on overall survival (OS) are unclear. This study aimed to evaluate risk factors pertinent to OS and to propose an optimal treatment strategy.
Clinical data of patients with PIRMS treated at Beijing Tiantan Hospital and from the English-language literature between 1946 and 2018 were reviewed. A literature review was performed via Ovid, MEDLINE, Embase, PubMed, Web of Science, and Cochrane databases using the terms "rhabdomyosarcoma," "intracranial," "cerebral," and "brain." Previously published data were processed and used according to Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines.
There were 8 males (66.7%) and 4 females with PIRMS at our institution, with a mean age of 24.3 years. Gross-total resection was achieved in 4 patients (33.3%), and adjuvant radiation and chemotherapy were administered in 5 (45.5%) and 3 (27.3%) patients, respectively. After a mean follow-up period of 13.7 months, all patients developed local-regional recurrence and died of the disease. Twenty-nine cases (14 female and 15 male) were reported in the literature with a median age of 9.0 years. After a mean follow-up duration of 18.6 months, 13 patients (44.8%) developed recurrences, 7 patients (24.1%) had extracranial metastasis, and 14 patients (48.3%) died. In the pooled cases, adjuvant radiation (hazard ratio [HR] 0.089, 95% confidence interval [CI] 0.027-0.288, p < 0.001) and age < 10 years (HR 0.227, 95% CI 0.077-0.666, p = 0.007) were independent predictors of good local-regional progression-free survival (LR-PFS). Adjuvant radiation therapy (HR 0.301, 95% CI 0.110-0.828, p = 0.020) and age < 10 years (HR 0.359, 95% CI 0.131-0.983, p = 0.046) were significant predictors for favorable OS in the multivariate model.
Due to the rarity of the disease, a poor outcome of PIRMS was demonstrated based on the pooled cohort. Use of radiation was associated with improved outcomes and should be considered to improve OS/LR-PFS. Further study is required to identify the optimal treatment regimen.Systematic review no.: CRD42019121249 (crd.york.ac.uk/PROSPERO/).
原发性颅内横纹肌肉瘤（PIRMS）较为罕见，治疗策略对总生存期（OS）的影响尚不清楚。本研究旨在评估与OS相关的危险因素，并提出最佳治疗策略。
回顾了在北京天坛医院接受治疗的PIRMS患者的临床资料以及1946年至2018年期间的英文文献。通过Ovid、MEDLINE、Embase、PubMed、Web of Science和Cochrane数据库进行文献检索，检索词为“横纹肌肉瘤”“颅内”“脑的”和“脑”。根据系统评价和Meta分析的首选报告项目指南对先前发表的数据进行处理和使用。
我院有8例男性（66.7%）和4例女性患有PIRMS，平均年龄为24.3岁。4例患者（33.3%）实现了肿瘤全切，分别有5例（45.5%）和3例（27.3%）患者接受了辅助放疗和化疗。平均随访13.7个月后，所有患者均出现局部区域复发并死于该疾病。文献报道了29例（14例女性和15例男性），中位年龄为9.0岁。平均随访18.6个月后，13例患者（44.8%）出现复发，7例患者（24.1%）发生颅外转移，14例患者（48.3%）死亡。在汇总病例中，辅助放疗（风险比[HR]0.089，95%置信区间[CI]0.027 - 0.288，p < 0.001）和年龄<10岁（HR 0.227，95%CI 0.077 - 0.666，p = 0.007）是局部区域无进展生存期（LR - PFS）良好的独立预测因素。在多变量模型中，辅助放疗（HR 0.301，95%CI 0.110 - 0.828，p = 0.020）和年龄<10岁（HR 0.359，95%CI 0.131 - 0.983，p = 0.046）是OS良好的显著预测因素。
由于该疾病罕见，基于汇总队列显示PIRMS预后较差。放疗的使用与改善预后相关，应考虑用于改善OS/LR - PFS。需要进一步研究以确定最佳治疗方案。系统评价编号：CRD42019121249（crd.york.ac.uk/PROSPERO/）
