Klimo Paul, Pai Panandiker Atmaram S, Thompson Clinton J, Boop Frederick A, Qaddoumi Ibrahim, Gajjar Amar, Armstrong Gregory T, Ellison David W, Kun Larry E, Ogg Robert J, Sanford Robert A
Semmes-Murphey Neurologic & Spine Institute, TN, USA.
J Neurosurg Pediatr. 2013 Mar;11(3):274-81. doi: 10.3171/2012.11.PEDS12317. Epub 2013 Jan 4.
Whereas diffuse intrinsic pontine gliomas generally have a short symptom duration and more cranial nerve involvement, focal brainstem gliomas are commonly low grade, with fewer cranial neuropathies. Although these phenotypic distinctions are not absolute predictors of outcome, they do demonstrate correlation in most cases. Because there is a limited literature on focal brainstem gliomas in pediatric patients, the objective of this paper was to report the management and outcome of these tumors.
The authors reviewed the records of all children diagnosed with radiographically confirmed low-grade focal brainstem gliomas from 1986 to 2010. Each patient underwent biopsy or resection for tissue diagnosis. Event-free survival (EFS) and overall survival were evaluated. Univariate analysis was conducted to identify demographic and treatment variables that may affect EFS.
Fifty-two patients (20 girls, 32 boys) with follow-up data were identified. Median follow-up was 10.0 years, and the median age at diagnosis was 6.5 years (range 1-17 years). The tumor locations were midbrain (n = 22, 42%), pons (n = 15, 29%), and medulla (n = 15, 29%). Surgical extirpation was the primary treatment in 25 patients (48%). The 5- and 10-year EFS and overall survival were 59%/98% and 52%/90%, respectively. An event or treatment failure occurred in 24 patients (46%), including 5 deaths. Median time to treatment failure was 3.4 years. Disease progression in the other 19 patients transpired within 25.1 months of diagnosis. Thirteen of these patients received radiation, including 11 within 2 months of primary treatment failure. Although children with intrinsic tumors had slightly better EFS at 5 years compared with those with exophytic tumors (p = 0.054), this difference was not significant at 10 years (p = 0.147). No other variables were predictive of EFS.
Surgery suffices in many children with low-grade focal brainstem gliomas. Radiation treatment is often reserved for disease progression but offers comparable disease control following biopsy. In the authors' experience, combining an assessment of clinical course, imaging, and tumor biopsy yields a reasonable model for managing children with focal brainstem tumors.
弥漫性脑桥内胶质瘤通常症状持续时间短且更多累及脑神经，而局灶性脑干胶质瘤一般为低级别，累及的脑神经较少。尽管这些表型差异并非预后的绝对预测指标，但在大多数情况下确实显示出相关性。由于关于儿童局灶性脑干胶质瘤的文献有限，本文的目的是报告这些肿瘤的治疗及预后情况。
作者回顾了1986年至2010年期间所有经影像学确诊为低级别局灶性脑干胶质瘤的儿童记录。每位患者均接受活检或切除以进行组织诊断。评估无事件生存期（EFS）和总生存期。进行单因素分析以确定可能影响EFS的人口统计学和治疗变量。
确定了52例有随访数据的患者（20例女孩，32例男孩）。中位随访时间为10.0年，诊断时的中位年龄为6.5岁（范围1 - 17岁）。肿瘤位于中脑（n = 22，42%）、脑桥（n = 15，29%）和延髓（n = 15，29%）。25例患者（48%）以手术切除作为主要治疗方法。5年和10年的EFS及总生存期分别为59%/98%和52%/90%。24例患者（46%）发生了事件或治疗失败，包括5例死亡。治疗失败的中位时间为3.4年。其他19例患者在诊断后25.1个月内病情进展。其中13例患者接受了放疗，包括11例在初次治疗失败后2个月内接受放疗。尽管内生型肿瘤患儿5年时的EFS略优于外生型肿瘤患儿（p = 0.054），但10年时这种差异无统计学意义（p = 0.147）。没有其他变量可预测EFS。
对于许多低级别局灶性脑干胶质瘤患儿，手术治疗足矣。放疗通常留待病情进展时使用，但活检后也能提供相当的疾病控制效果。根据作者的经验，综合临床病程、影像学和肿瘤活检评估可为治疗局灶性脑干肿瘤患儿提供一个合理的模式。
