作者
Nicole J Ullrich, Sanjay P Prabhu, Alyssa T Reddy, Michael J Fisher, Roger Packer, Stewart Goldman, Nathan J Robison, David H Gutmann, David H Viskochil, Jeffrey C Allen, Bruce Korf, Alan Cantor, Gary Cutter, Coretta Thomas, John P Perentesis, Tomoyuki Mizuno, Alexander A Vinks, Peter E Manley, Susan N Chi, Mark W Kieran
发表日期
2020/10
期刊
Neuro-oncology
卷号
22
期号
10
页码范围
1527-1535
出版商
Oxford University Press
简介
Background
Activation of the mammalian target of rapamycin (mTOR) pathway is observed in neurofibromatosis type 1 (NF1) associated low-grade gliomas (LGGs), but agents that inhibit this pathway, including mTOR inhibitors, have not been studied in this population. We evaluate the efficacy of the orally administered mTOR inhibitor everolimus for radiographically progressive NF1-associated pediatric LGGs.
Methods
Children with radiologic-progressive, NF1-associated LGG and prior treatment with a carboplatin-containing chemotherapy were prospectively enrolled on this phase II clinical trial to receive daily everolimus. Whole blood was analyzed for everolimus and markers of phosphatidylinositol-3 kinase (PI3K)/mTOR pathway inhibition. Serial MRIs were obtained during treatment. The primary endpoint was progression-free survival at 48 weeks.
Results …
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NJ Ullrich, SP Prabhu, AT Reddy, MJ Fisher, R Packer… - Neuro-oncology, 2020