Department of Surgery, McMaster University, Hamilton, Ontario, Canada.
Stem Cell and Cancer Research Institute, McMaster University, Hamilton, Ontario, Canada.
Can J Neurol Sci. 2019 Jul;46(4):403-414. doi: 10.1017/cjn.2019.39.
Children diagnosed with medulloblastoma (MB) who are refractory to upfront therapy or experience recurrence have very poor prognoses. Although phase I and phase II trials exist, these treatments bear significant treatment-related morbidity and mortality.
A retrospective review of children diagnosed with a recurrence of MB from 2002 to 2015 at McMaster University was undertaken.
Recurrent disease in 10 patients involved leptomeningeal dissemination, with 3 experiencing local recurrence. In three recurrent patients the disease significantly progressed, and the children were palliated. The remaining 10 children underwent some form of salvage therapy, including surgical re-resection, radiation, and chemotherapy, either in isolation or in varying combinations. Of the 13 children experiencing treatment-refractory or recurrent disease, 4 are currently alive with a median follow-up of 38.5 months (75.5 months). Of the eight patients with molecular subgrouping data, none of the Wnt MB experienced recurrence.
Recurrent MB carried a poor prognosis with a 5-year overall survival (OS) of 18.2% despite the administration of salvage therapy. The upfront therapy received, available treatment, and tolerability of the proposed salvage therapy resulted in significant heterogeneity in the treatment of our recurrent cohort.
对初始治疗耐药或复发的儿童患有髓母细胞瘤(MB)预后非常差。虽然存在 I 期和 II 期试验,但这些治疗方法具有显著的治疗相关发病率和死亡率。
对 2002 年至 2015 年在麦克马斯特大学被诊断为 MB 复发的儿童进行回顾性研究。
10 名患者的复发性疾病涉及软脑膜播散,其中 3 名患者出现局部复发。在 3 名复发性患者中,疾病显著进展,患儿接受姑息治疗。其余 10 名儿童接受了某种形式的挽救性治疗,包括单独或不同组合的手术再切除、放疗和化疗。在 13 名经历治疗耐药或复发疾病的儿童中,4 名目前存活,中位随访时间为 38.5 个月(75.5 个月)。在有分子亚群分组数据的 8 名患者中,没有 Wnt MB 出现复发。
尽管进行了挽救性治疗,复发性 MB 的预后仍较差,5 年总生存率(OS)为 18.2%。初始治疗、可获得的治疗方法以及对拟议挽救性治疗的耐受性导致我们的复发性患者队列的治疗方法存在显著的异质性。