Radiation Oncology, Hospital General Universitario Gregorio Marañón, Madrid, Spain.
Computing Services, Research Support, Complutense University of Madrid, Madrid, Spain.
Eur J Pediatr. 2021 Jan;180(1):127-136. doi: 10.1007/s00431-020-03722-4. Epub 2020 Jun 21.
Medulloblastoma (MB) is a malignant embryonal tumor that develops especially in childhood, with overall survival (OS) at 5 years of up to 70%. The objective of this study is to analyze treatment delivery variables in a retrospective cohort and evaluate the impact of these treatment quality parameters on survival. From 2000 to 2018, 40 pediatric patients with medulloblastoma, treated according to current international protocols, were retrospectively analyzed. Treatment delivery quality indicators were analyzed including the extent of surgery, radiotherapy (RT) parameters, and chemotherapy variables, related with time and dose-intensity deviations. With a median follow-up of 74 months (range, 6-195), OS at 5 years was 74 ± 7%, 81 ± 8% for standard-risk, and 55 ± 16% for high-risk patients (p = 0.090). Disease-free survival at 5 years was not significantly affected by extent of surgery (p = 0.428) and RT-related variables such as surgery-RT interval (p = 0.776) neither RT duration (p = 0.172) or maintenance chemotherapy compliance (p = 0.634). Multivariate analysis identified risk groups predictive of worse DFS (p = 0.032) and leptomeningeal dissemination associated with inferior OS (p = 0.029).Conclusion: Treatment delivery optimization has improved survival rates of patients with MB. Despite this, in our study, we have not established a clear influence of the considered radiotherapy and chemotherapy treatment quality parameters on outcomes. What is Known: • Improvement in treatment modalities during the last decades has reached a 5-year OS of up to 70% in these patients. • Extent of resection and radiotherapy parameters such as interval between surgery-radiotherapy and radiotherapy duration has been described as probable survival prognostic factors. What is New: • Differences in medulloblastoma survival rates between prospective studies and retrospective series. • The impact on survival of the three main treatment variables, surgery, radiotherapy and chemotherapy, susceptible to improvement.
髓母细胞瘤（MB）是一种恶性胚胎肿瘤，尤其在儿童中发育，5 年总生存率（OS）高达 70%。本研究的目的是分析回顾性队列中的治疗提供变量，并评估这些治疗质量参数对生存的影响。2000 年至 2018 年间，对 40 名按照现行国际方案治疗的髓母细胞瘤患儿进行回顾性分析。分析了治疗提供质量指标，包括手术范围、放疗（RT）参数和化疗变量，与时间和剂量强度偏差有关。中位随访时间为 74 个月（范围 6-195），5 年 OS 为 74±7%，标准风险为 81±8%，高危为 55±16%（p=0.090）。5 年无病生存率不受手术范围（p=0.428）和 RT 相关变量（如手术-RT 间隔，p=0.776）的显著影响，也不受 RT 持续时间（p=0.172）或维持化疗依从性（p=0.634）的影响。多变量分析确定了DFS 较差的风险组（p=0.032）和与 OS 较差相关的软脑膜播散（p=0.029）。结论：治疗提供的优化提高了 MB 患者的生存率。尽管如此，在我们的研究中，我们没有确定所考虑的放疗和化疗治疗质量参数对结果的明确影响。已知的是：• 过去几十年治疗方式的改进使这些患者的 5 年 OS 达到 70%。• 切除范围和放疗参数，如手术-放疗间隔和放疗持续时间，被描述为可能的生存预后因素。新的是：• 前瞻性研究和回顾性系列之间髓母细胞瘤生存率的差异。• 手术、放疗和化疗这三种主要治疗变量对生存的影响，是可以改善的。
