Phase II Weekly Vinblastine for Chemotherapy-Naive Children With Progressive Low-Grade Glioma: A Canadian Pediatric Brain Tumor Consortium Study
Phase II Weekly Vinblastine for Chemotherapy-Naive Children With Progressive Low-Grade Glioma: A Canadian Pediatric Brain Tumor Consortium Study
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DOI：
10.1200/jco.2016.68.1585
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发表时间：
2016-10-10
影响因子：
45.3
通讯作者：
Bouffet, Eric
中科院分区：
文献类型：
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作者：
Lassaletta, Alvaro;Scheinemann, Katrin;Bouffet, Eric
目的长春碱单药治疗儿童低级别胶质瘤（PLGG）患者在化疗和/或放疗后治疗失败后显示出良好的活性和低毒性。本研究的目的是评估长春碱在治疗初治children.Patients and MethodsPatients < 18岁的不可切除和/或进行性治疗初治PLGG的活动是合格的。长春碱每周静脉注射一次，剂量为6 mg/m2，持续70周。视力，生活质量，1型神经纤维瘤病（NF 1）的状态，BRAF突变/融合状态也被确定，并与outcome.ResultsFifty-four患者参加了这项研究，中位年龄为8岁（范围，0.7至17.2年）。大多数患者患有视交叉/下丘脑肿瘤（55.5%），13例患者（24.1%）患有NF 1。最常见的组织学类型为毛细胞型星形细胞瘤（46.3%）。17例患者仅使用放射学标准进行诊断。集中审查了化疗的最佳缓解，缓解率（完全、部分或轻微缓解）为25.9%。47例患者（87.0%）实现了疾病稳定（完全、部分或轻微缓解或疾病稳定）。在20%的视路胶质瘤患者中观察到视力改善。整个队列的5年总生存率和无进展生存率（PFS）分别为94.4%（95% CI，88.5%-100%）和53.2%（95% CI，41.3%-68.5%）。NF 1患者的PFS（85.1%; 95% CI，68.0%-100%）显著优于无NF 1患者（42.0%; 95% CI，29.1%-60.7%; P = 0.012）。年龄< 3 years or >10岁与不良结局无关。治疗耐受性良好，治疗期间生活质量未受影响。在这项试验中，有BRAF的改变和outcome.ConclusionVinblastine每周一次给药的儿童与治疗初治PLGG的耐受性良好。总生存期和PFS与当前治疗相当，具有良好的毒性特征和维持的生活质量。
PurposeVinblastine monotherapy has shown promising activity and a low-toxicity profile in patients with pediatric low-grade glioma (PLGG) who experienced treatment failure after initial treatment with chemotherapy and/or radiation. The aim of this study was to assess the activity of vinblastine in therapy-naive children.Patients and MethodsPatients < 18 years old with unresectable and/or progressive therapy-naive PLGG were eligible. Vinblastine was administered once per week at a dose of 6 mg/m(2) intravenously over a period of 70 weeks. Vision, quality of life, neurofibromatosis type 1 (NF1) status, and BRAF mutation/fusion status were also determined and correlated with outcome.ResultsFifty-four patients were enrolled onto the study, with a median age of 8 years (range, 0.7 to 17.2 years). Most patients had chiasmatic/hypothalamic tumors (55.5%), and 13 patients (24.1%) had NF1. The most common histology was pilocytic astrocytoma (46.3%). Seventeen patients were diagnosed using radiologic criteria alone. Best response to chemotherapy was centrally reviewed with a response rate (complete, partial, or minor response) of 25.9%. Disease stabilization (complete, partial, or minor response or stable disease) was achieved in 47 patients (87.0%). Visual improvement was observed in 20% of patients with optic pathway glioma. Five-year overall survival and progression-free survival (PFS) rates were 94.4% (95% CI, 88.5% to 100%) and 53.2% (95% CI, 41.3% to 68.5%), respectively, for the entire cohort. Patients with NF1 had a significantly better PFS (85.1%; 95% CI, 68.0% to 100%) when compared with patients without NF1 (42.0%; 95% CI, 29.1% to 60.7%; P = .012). Age< 3 years or > 10 years was not associated with poor outcome. Treatment was well tolerated, and quality of life was not affected during treatment. In this trial, there was no correlation between BRAF alterations and outcome.ConclusionVinblastine administered once per week is well tolerated in children with treatment naive PLGG. Overall survival and PFS are comparable to current therapies, with a favorable toxicity profile and a maintained quality of life.