Minturn Jane E, Mochizuki Aaron Y, Partap Sonia, Belasco Jean B, Lange Beverly J, Li Yimei, Phillips Peter C, Gibbs Iris C, Fisher Paul G, Fisher Michael J, Janss Anna J
Department of Pediatrics, Division of Oncology, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.
Department of Pediatrics, Division of Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States.
Front Oncol. 2021 Sep 2;11:744739. doi: 10.3389/fonc.2021.744739. eCollection 2021.
Medulloblastoma is one of the most common malignant brain tumors in children. To date, the treatment of average-risk (non-metastatic, completely resected) medulloblastoma includes craniospinal radiation therapy and adjuvant chemotherapy. Modern treatment modalities and now risk stratification of subgroups have extended the survival of these patients, exposing the long-term morbidities associated with radiation therapy. Prior to advances in molecular subgrouping, we sought to reduce the late effects of radiation in patients with average-risk medulloblastoma.
We performed a single-arm, multi-institution study, reducing the dose of craniospinal irradiation by 25% to 18 Gray (Gy) with the goal of maintaining the therapeutic efficacy as described in CCG 9892 with maintenance chemotherapy.
Twenty-eight (28) patients aged 3-30 years were enrolled across three institutions between April 2001 and December 2010. Median age at enrollment was 9 years with a median follow-up time of 11.7 years. The 3-year relapse-free (RFS) and overall survival (OS) were 79% (95% confidence interval [CI] 58% to 90%) and 93% (95% CI 74% to 98%), respectively. The 5-year RFS and OS were 71% (95% CI 50% to 85%) and 86% (95% CI 66% to 94%), respectively. Toxicities were similar to those seen in other studies; there were no grade 5 toxicities.
Given the known neurocognitive adverse effects associated with cranial radiation therapy, studies to evaluate the feasibility of dose reduction are needed. In this study, we demonstrate that select patients with average-risk medulloblastoma may benefit from a reduced craniospinal radiation dose of 18 Gy without impacting relapse-free or overall survival.
ClinicalTrials.gov identifier: NCT00031590.
髓母细胞瘤是儿童最常见的恶性脑肿瘤之一。迄今为止，平均风险（非转移性、完全切除）髓母细胞瘤的治疗包括颅脊髓放射治疗和辅助化疗。现代治疗方式以及目前亚组的风险分层延长了这些患者的生存期，同时也暴露了与放射治疗相关的长期并发症。在分子亚组分类取得进展之前，我们试图降低平均风险髓母细胞瘤患者放射治疗的晚期效应。
我们进行了一项单臂、多机构研究，将颅脊髓照射剂量降低25%至18格雷（Gy），目标是在维持化疗的情况下保持CCG 9892中所述的治疗效果。
2001年4月至2010年12月期间，三个机构共纳入了28例年龄在3至30岁之间的患者。入组时的中位年龄为9岁，中位随访时间为11.7年。3年无复发生存率（RFS）和总生存率（OS）分别为79%（95%置信区间[CI]58%至90%）和93%（95%CI 74%至98%）。5年RFS和OS分别为71%（95%CI 50%至85%）和86%（95%CI 66%至94%）。毒性与其他研究中观察到的相似；无5级毒性。
鉴于已知与颅脑放射治疗相关的神经认知不良反应，需要开展研究评估降低剂量的可行性。在本研究中，我们证明部分平均风险髓母细胞瘤患者可能受益于18 Gy的降低颅脊髓放射剂量，且不影响无复发生存率或总生存率。
ClinicalTrials.gov标识符：NCT00031590。
