Department of Pediatrics, Morgan Adams Foundation Pediatric Brain Tumor Research Program, Children's Hospital Colorado, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.
Center for Cancer and Blood Disorders, Morgan Adams Foundation Pediatric Brain Tumor Research Program, University of Colorado School of Medicine, 13123 East 16th Avenue, Box B115, Aurora, CO, 80045, USA.
J Neurooncol. 2020 Jul;148(3):569-575. doi: 10.1007/s11060-020-03549-x. Epub 2020 Jun 6.
Low-grade glioma (LGG) represent the most common pediatric central nervous system tumor. When total surgical resection is not feasible, chemotherapy is first-line therapy in children. Multiple pediatric LGG chemotherapy regimens have been investigated with variable 2-year event free survival (EFS) rates of 39-69%. To date, treatment of pediatric LGG with a carboplatin and vinblastine (C/VBL) chemotherapy regimen has only been evaluated in a phase 1 dose-finding study.
A retrospective review of pediatric patients with LGG who were treated with C/VBL at Children's Hospital of Colorado or Akron Children's Hospital from 2011 to 2017 was conducted. Data collected included patient demographics, tumor location, disease response, neurofibromatosis 1 (NF1) status, therapy duration and toxicities. Response to therapy was determined by objective findings on imaging and treating physicians' evaluation.
Forty-six patients were identified for analysis, all of whom were chemotherapy-naive. Only five patients treated in this cohort had NF1. BRAF fusion was identified in 65% (22/34) of tested tumors. Best therapy response was partial response in nine patients and stable disease in twenty-five patients. Twelve patients had progressive disease. One-year, 3-year, and 5-year EFS probabilities for all patients were 69.6%, 39.4%, and 34.5%, respectively. Nine patients had admissions for febrile neutropenia and seven patients experienced one delay in chemotherapy due to neutropenia. Only two patients had to discontinue this chemotherapy regimen because of treatment-related toxicities [carboplatin allergy (n = 1) and vinblastine neuropathy (n = 1)].
C/VBL achieves similar EFS rates to other single-agent and combination cytotoxic chemotherapy regimens for pediatric LGG with manageable toxicities.
低级别胶质瘤（LGG）是最常见的儿童中枢神经系统肿瘤。当无法进行全切除手术时，化疗是儿童的一线治疗方法。已经研究了多种儿科 LGG 化疗方案，其 2 年无事件生存率（EFS）为 39-69%。迄今为止，卡铂和长春碱（C/VBL）化疗方案仅在一项 1 期剂量发现研究中评估了治疗儿科 LGG。
对 2011 年至 2017 年在科罗拉多儿童医院或阿克伦儿童医院接受 C/VBL 治疗的 LGG 儿科患者进行了回顾性审查。收集的数据包括患者人口统计学、肿瘤位置、疾病反应、神经纤维瘤病 1 型（NF1）状态、治疗持续时间和毒性。治疗反应通过影像学的客观发现和治疗医生的评估来确定。
确定了 46 名患者进行分析，所有患者均为化疗初治。在此队列中接受治疗的只有 5 名患者患有 NF1。在 65%（22/34）的测试肿瘤中发现了 BRAF 融合。9 名患者的最佳治疗反应为部分缓解，25 名患者为稳定疾病。12 名患者病情进展。所有患者的 1 年、3 年和 5 年 EFS 概率分别为 69.6%、39.4%和 34.5%。9 名患者因发热性中性粒细胞减少症入院，7 名患者因中性粒细胞减少症而延迟化疗一次。只有两名患者因治疗相关毒性而不得不停止这种化疗方案[卡铂过敏（n=1）和长春碱神经病变（n=1）]。
C/VBL 治疗儿科 LGG 的 EFS 率与其他单药和联合细胞毒性化疗方案相似，毒性可管理。
