Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis.
Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis.
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DOI：
10.1056/nejmoa1001671
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发表时间：
2010-11-04
影响因子：
158.5
通讯作者：
Franz, David Neal
中科院分区：
文献类型：
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作者：
Krueger, Darcy A.;Care, Marguerite M.;Franz, David Neal
背景资料：神经外科切除术是结节性硬化症患者室管膜下巨细胞星形细胞瘤的标准治疗方法。另一种可能是使用依维莫司，它抑制哺乳动物的雷帕霉素，一种蛋白质调节的基因产物参与结节性硬化complex.Methods：患者3岁或以上的连续增长的室管膜下巨细胞星形细胞瘤有资格为这个开放标签的研究。主要疗效终点是基线和6个月之间室管膜下巨细胞星形细胞瘤体积的变化。我们口服依维莫司，剂量为3.0毫克每平方米体表面积，达到谷浓度为5至15纳克每毫升。依维莫司治疗与原发性室管膜下巨细胞星形细胞瘤体积有临床意义的减小相关，这是独立的中心审查评估的结果（P
Background: Neurosurgical resection is the standard treatment for subependymal giant-cell astrocytomas in patients with the tuberous sclerosis complex. An alternative may be the use of everolimus, which inhibits the mammalian target of rapamycin, a protein regulated by gene products involved in the tuberous sclerosis complex.Methods: Patients 3 years of age or older with serial growth of subependymal giant-cell astrocytomas were eligible for this open-label study. The primary efficacy end point was the change in volume of subependymal giant-cell astrocytomas between baseline and 6 months. We gave everolimus orally, at a dose of 3.0 mg per square meter of body-surface area, to achieve a trough concentration of 5 to 15 ng per milliliter.Results: We enrolled 28 patients. Everolimus therapy was associated with a clinically meaningful reduction in volume of the primary subependymal giant-cell astrocytoma, as assessed on independent central review (P