Brain Tumor Center Amsterdam, Department of Neurology, Amsterdam UMC, Amsterdam, The Netherlands.
Brain Tumor Center, Department of Neurology, Erasmus MC Cancer Institute, Rotterdam, The Netherlands.
J Neurooncol. 2023 Mar;162(1):225-235. doi: 10.1007/s11060-023-04285-8. Epub 2023 Mar 15.
Medulloblastoma is a rare tumor in adults. The objective of this nationwide, multicenter study was to evaluate the toxicity and efficacy of the Dutch treatment protocol for adult medulloblastoma patients.
Adult medulloblastoma patients diagnosed between 2010 and 2018 were identified in the Dutch rare tumors registry or nationwide pathology database. Patients with intention to treat according to the national treatment protocol were included. Risk stratification was performed based on residual disease, histological subtype and extent of disease. All patients received postoperative radiotherapy [craniospinal axis 36 Gy/fossa posterior boost 19.8 Gy (14.4 Gy in case of metastases)]. High-risk patients received additional neoadjuvant (carboplatin-etoposide), concomitant (vincristine) and adjuvant chemotherapy (carboplatin-vincristine-cyclophosphamide) as far as feasible by toxicity. Methylation profiling, and additional next-generation sequencing in case of SHH-activated medulloblastomas, were performed.
Forty-seven medulloblastoma patients were identified, of whom 32 were treated according to the protocol. Clinical information and tumor material was available for 28 and 20 patients, respectively. The histological variants were mainly classic (43%) and desmoplastic medulloblastoma (36%). Sixteen patients (57%) were considered standard-risk and 60% were SHH-activated medulloblastomas. Considerable treatment reductions and delays in treatment occurred due to especially hematological and neurotoxicity. Only one high-risk patient could complete all chemotherapy courses. 5-years progression-free survival (PFS) and overall survival (OS) for standard-risk patients appeared worse than for high-risk patients (PFS 69% vs. 90%, OS 81% vs. 90% respectively), although this wasn't statistically significant.
Combined chemo-radiotherapy is a toxic regimen for adult medulloblastoma patients that may result in improved survival.
成神经管细胞瘤在成人中较为罕见。本项全国性多中心研究旨在评估荷兰成神经管细胞瘤治疗方案对成人患者的毒性和疗效。
在荷兰罕见肿瘤登记处或全国病理数据库中确定 2010 年至 2018 年期间诊断为成神经管细胞瘤的成年患者。根据国家治疗方案进行意向治疗的患者纳入研究。根据残留疾病、组织学亚型和疾病范围进行风险分层。所有患者均接受术后放疗[颅脊髓轴 36 Gy/后颅窝加量 19.8 Gy（有转移时为 14.4 Gy）]。高危患者尽可能接受新辅助（卡铂-依托泊苷）、同期（长春新碱）和辅助化疗（卡铂-长春新碱-环磷酰胺），具体取决于毒性。进行甲基化分析，以及在 SHH 激活型成神经管细胞瘤中进行额外的下一代测序。
共确定 47 例成神经管细胞瘤患者，其中 32 例按方案治疗。分别有 28 例和 20 例患者可获得临床信息和肿瘤材料。组织学变异型主要为经典型（43%）和促结缔组织增生型成神经管细胞瘤（36%）。16 例（57%）患者被认为是标准风险，60%为 SHH 激活型成神经管细胞瘤。由于血液学和神经毒性，治疗剂量显著减少且延迟。只有 1 例高危患者能够完成所有化疗疗程。标准风险患者的 5 年无进展生存率（PFS）和总生存率（OS）似乎低于高危患者（PFS 分别为 69%和 90%，OS 分别为 81%和 90%），尽管无统计学意义。
联合放化疗对成神经管细胞瘤患者毒性较大，可能提高生存率。
