在三名新生儿中使用mTOR抑制剂依维莫司治疗与结节性硬化症相关的肿瘤。
Use of mTOR inhibitor everolimus in three neonates for treatment of tumors associated with tuberous sclerosis complex.
作者信息
Goyer Isabelle, Dahdah Nagib, Major Philippe
机构信息
Department of Pharmacy, CHU Sainte-Justine, University of Montreal, Montreal, Quebec, Canada.
Division of Pediatric Cardiology, CHU Sainte-Justine, University of Montreal, Montreal, Quebec, Canada.
出版信息
Pediatr Neurol. 2015 Apr;52(4):450-3. doi: 10.1016/j.pediatrneurol.2015.01.004. Epub 2015 Jan 14.
BACKGROUND
Tuberous sclerosis complex is characterized by the growth of benign tumors in multiple organs, caused by the disinhibition of the mammalian target of rapamycin (mTOR) protein. mTOR inhibitors, such as everolimus, are used in patients with tuberous sclerosis complex, mainly to reduce the size of renal angiomyolipomas and subependymal giant cell astrocytomas. There are minimal data available regarding its use during the neonatal period.
METHODS
We report clinical and pharmacological data of three neonates treated with the mTOR inhibitor everolimus (two hemodynamically significant cardiac rhabdomyomas and one voluminous subependymal giant cell astrocytoma).
RESULTS
Beneficial clinical responses were observed in all three patients and the medication was generally well-tolerated. Optimal dose was 0.1 mg orally once daily and was confirmed with therapeutic drug monitoring.
CONCLUSION
Everolimus is a promising pharmacological approach to treat clinically significant inoperable cardiac rhabdomyomas or subependymal giant cell astrocytoma associated with tuberous sclerosis complex during the neonatal period.
背景
结节性硬化症的特征是多个器官出现良性肿瘤，这是由雷帕霉素哺乳动物靶点（mTOR）蛋白的抑制解除所引起。mTOR抑制剂，如依维莫司，用于结节性硬化症患者，主要是为了缩小肾血管平滑肌脂肪瘤和室管膜下巨细胞星形细胞瘤的大小。关于其在新生儿期使用的数据极少。
方法
我们报告了3例接受mTOR抑制剂依维莫司治疗的新生儿的临床和药理学数据（2例有血流动力学意义的心脏横纹肌瘤和1例巨大室管膜下巨细胞星形细胞瘤）。
结果
所有3例患者均观察到有益的临床反应，且药物耐受性总体良好。最佳剂量为口服0.1mg，每日1次，并通过治疗药物监测得以确认。
结论
依维莫司是治疗新生儿期与结节性硬化症相关的临床上无法手术的有显著意义的心脏横纹肌瘤或室管膜下巨细胞星形细胞瘤的一种有前景的药理学方法。
Zotero 插件