Division of Neuro-oncology, St. Jude Children's Research Hospital, 262 Danny Thomas Place - Mailstop 260, Memphis, TN, 38105, USA.
Curr Treat Options Oncol. 2012 Dec;13(4):465-77. doi: 10.1007/s11864-012-0205-5.
Survival rates for patients with ependymoma, a glial tumor arising from the ependymal cells lining the ventricles of the brain and spinal cord canal, have changed little during the past decade. Contemporary "standard" therapy for children and adults with ependymoma consists of maximal surgical resection followed by focal irradiation except in cases of disseminated disease. Despite refinements in radiotherapy techniques and improvements in survival for patients with gross totally resected, nonanaplastic disease, many therapeutic challenges remain, especially for patients with unresectable, macroscopic, metastatic, or anaplastic disease. Moreover, radiotherapy to the developing central nervous system, especially in patients younger than age 5 years, can have potential long-term neurocognitive and neuroendocrine sequelae. Chemotherapy has not played a role in most treatment regimens for ependymoma to date, but due to the ongoing therapeutic challenges for a subset of patients, this modality is being reinvestigated in a few ongoing studies. Early recognition of patients who will not respond to primary therapy is imperative to modify treatment regimens, such as intensification with the addition of adjuvant chemotherapy, the use of novel experimental therapies, or their combination. Refinements in patient stratification schemes that are based on a combination of clinical variables and molecular profiles also require improved knowledge of tumor biology. Several molecular alterations have been identified already, some of which may be of prognostic significance. Furthermore, disruption of molecular alterations in signaling pathways involved in the development and maintenance of ependymoma by using novel molecularly targeted therapies may improve outcomes and reduce toxicity for patients with ependymoma.
室管膜瘤患者的存活率在过去十年中变化不大，室管膜瘤是一种源自脑和脊髓管脑室衬里的室管膜细胞的神经胶质瘤。目前，儿童和成人室管膜瘤的“标准”治疗方法是最大限度地进行手术切除，然后进行局部放疗，但弥散性疾病除外。尽管放射治疗技术有所改进，且完全切除、非间变疾病患者的生存率有所提高，但仍存在许多治疗挑战，尤其是对于无法切除的、肉眼可见的、转移性或间变疾病的患者。此外，对发育中的中枢神经系统进行放疗，尤其是对年龄小于 5 岁的患者，可能会导致潜在的长期神经认知和神经内分泌后遗症。迄今为止，化疗在大多数室管膜瘤治疗方案中并未发挥作用，但由于一部分患者仍存在治疗挑战，因此这种治疗方式正在少数正在进行的研究中重新研究。早期识别那些对初始治疗无反应的患者至关重要，这有助于修改治疗方案，例如通过添加辅助化疗来强化治疗，使用新的实验性治疗方法或联合使用这些方法。基于临床变量和分子特征组合的患者分层方案的改进也需要提高对肿瘤生物学的认识。已经确定了几种分子改变，其中一些可能具有预后意义。此外，通过使用新型的分子靶向治疗来破坏参与室管膜瘤发生和维持的信号通路中的分子改变，可能会改善室管膜瘤患者的预后并降低毒性。
