Memorial Sloan-Kettering Cancer Center, 1275 York Ave., New York, NY, 10065, USA,
Curr Treat Options Neurol. 2013 Oct;15(5):593-606. doi: 10.1007/s11940-013-0255-4.
Medulloblastoma and central nervous system (CNS) primitive neuroectodermal tumor (PNET) are primary pediatric brain tumors that require multidisciplinary therapies. Although often treated similarly in clinical trials, they are biologically different diseases. Even within medulloblastomas and CNS PNETs, there are molecularly distinct subgroups with differing presentations and prognoses. Overall, prognosis is better for medulloblastomas. Specific treatments for these types of cancer are continuously evolving to maximize survival and minimize long-term sequelae of treatment. Patients should be treated on a clinical trial, if eligible, as they may gain benefit with minimal risk over current standard of care. The amount of residual disease after surgery better correlates with survival for medulloblastomas than for CNS PNETs. Maximal surgical resection of tumor should be done, only if additional permanent, neurologic deficits can be spared. Patients should have a staging work-up to assess the extent of disease. This includes postoperative magnetic resonance imaging (MRI) of the brain, MRI of the entire spine and lumbar cerebrospinal fluid (CSF) sampling for cytological examination, if deemed safe. Radiation therapy to the entire CNS axis is required, with a greater dose (boost) given to the region of the primary site or any bulky residual disease for older children. Adjuvant chemotherapy must be given even if no evidence of disease after radiation therapy exists, as the risk of relapse is substantial after radiation alone. Subsets of younger children with medulloblastoma, arbitrarily defined as those younger than 3 years of age in some studies and 4 or even 5 years in other studies, can be effectively treated with chemotherapy alone. Recent genomic studies have revealed further subtypes of disease than previously recognized. Clinical trials to exploit these biologic differences are required to assess potential efficacy of targeted agents. The treatment of medulloblastoma and CNS PNET can cause significant impairment in neurologic function. Evaluations by physical therapy, occupational therapy, speech therapy and neurocognitive assessments should be obtained, as needed. After therapy is completed, survivors need follow-up of endocrine function, surveillance scans and psychosocial support.
髓母细胞瘤和中枢神经系统（CNS）原始神经外胚层肿瘤（PNET）是小儿原发性脑肿瘤，需要多学科治疗。尽管在临床试验中经常进行类似的治疗，但它们在生物学上是不同的疾病。即使在髓母细胞瘤和 CNS PNET 中，也存在具有不同表现和预后的分子上明显不同的亚组。总的来说，髓母细胞瘤的预后较好。这些类型癌症的具体治疗方法在不断发展，以最大限度地提高生存率并最大限度地减少治疗的长期后遗症。如果符合条件，患者应在临床试验中接受治疗，因为他们可能会从最低风险中获益，超过当前的标准治疗。手术后残留疾病的数量与髓母细胞瘤的生存相关性优于 CNS PNET。只有在可以避免额外的永久性神经功能缺损的情况下，才应进行肿瘤的最大程度手术切除。患者应进行分期检查以评估疾病的范围。这包括脑术后磁共振成像（MRI）、整个脊柱 MRI 和腰椎脑脊液（CSF）取样进行细胞学检查，如果认为安全的话。需要对整个 CNS 轴进行放射治疗，对于较大的儿童，在原发部位或任何大块残留肿瘤区域给予更高剂量（增强）。即使在放射治疗后没有疾病证据，也必须给予辅助化疗，因为单独放射治疗后复发的风险很高。根据一些研究定义为 3 岁以下，而根据其他研究定义为 4 岁甚至 5 岁以下的年轻儿童的髓母细胞瘤亚组，可以单独用化疗有效治疗。最近的基因组研究揭示了比以前认识到的更多的疾病亚型。需要进行临床试验来利用这些生物学差异，以评估靶向药物的潜在疗效。髓母细胞瘤和 CNS PNET 的治疗会导致神经功能严重受损。应根据需要获得物理治疗、职业治疗、言语治疗和神经认知评估。治疗完成后，幸存者需要内分泌功能、监测扫描和社会心理支持的随访。
