Neurosurgery and Pediatrics, George Washington University School of Medicine, Washington, DC, USA.
Division of Neurosurgery, Children's National Hospital, Washington, DC, USA.
Adv Tech Stand Neurosurg. 2024;53:93-118. doi: 10.1007/978-3-031-67077-0_7.
Ependymomas are the third most common intracranial tumor in children, presenting in both the supratentorial and infratentorial compartments. They may present in infants, young children, and adolescents with symptoms depending on size, location, and the age of the patient. The ideal imaging for evaluation and treatment is MRI. This is crucial for preoperative evaluation and planning, as well as postoperative assessment and evaluating the efficacy of treatment. Essentially without exception, aggressive surgery aimed at complete resection is the initial and most important factor in the long-term outcome of all these children. Histopathologic diagnosis for intracranial pediatric ependymoma has been narrowed to grade II and grade III, no longer characterized as classic and anaplastic. Subsequent conformal photon or proton beam irradiation is an established post-surgical therapy, with solid evidence that it benefits survival and offers lower toxicity to the normal brain of the young child. Although chemotherapeutic treatment has not been generally impactful, immunotherapeutic interventions may be on the horizon. Updated molecular subgrouping of ependymoma is changing the post-resection approach of these tumors with regard to both treatment and outcome. Excluding spinal ependymoma and subependymoma, there are four subtypes that are defined by genetic characteristics, two found in the supratentorial compartment, ST-EPN-YAP1 and ST-EPN-ZFTA, and two in the posterior fossa, PF-EPN-A and PF-EPN-B. Younger children harboring ZFTA fusion-positive supratentorial and type A posterior fossa tumors, regardless of histology, tend toward the poorest outcomes. On the contrary, older children with supratentorial YAP1 fusion-positive ependymomas and type B posterior fossa tumors may survive with surgery alone. The paradigm shift regarding the behavior of the various childhood ependymoma subtypes will hopefully lead to targeted, individualized therapies and improved outcomes.
室管膜瘤是儿童中第三常见的颅内肿瘤,可发生于幕上和幕下部位。它们可发生于婴儿、幼儿和青少年,症状取决于肿瘤的大小、位置和患者的年龄。评估和治疗的理想影像学方法是 MRI。这对于术前评估和计划、术后评估以及评估治疗效果至关重要。几乎无一例外,旨在完全切除的积极手术是所有这些儿童长期预后的初始和最重要因素。颅内儿科室管膜瘤的组织病理学诊断已缩小为 2 级和 3 级,不再被认为是经典和间变性的。随后的适形光子或质子束照射是一种已确立的术后治疗方法,有确凿的证据表明它有益于生存,并降低了对幼儿正常大脑的毒性。尽管化疗治疗总体上没有明显效果,但免疫治疗干预可能即将出现。室管膜瘤的更新分子亚组正在改变这些肿瘤在手术后的治疗和预后方法。除了脊髓室管膜瘤和室管膜下瘤,还有四种亚型,它们是根据遗传特征定义的,两种位于幕上,ST-EPN-YAP1 和 ST-EPN-ZFTA,两种位于后颅窝,PF-EPN-A 和 PF-EPN-B。携带 ZFTA 融合阳性幕上和 A 型后颅窝肿瘤的年幼儿童,无论组织学如何,往往预后最差。相反,年龄较大的儿童如果存在幕上 YAP1 融合阳性的室管膜瘤和 B 型后颅窝肿瘤,仅通过手术就可能存活。各种儿童室管膜瘤亚型行为的范式转变有望导致靶向、个体化治疗和改善预后。