% 0期刊文章%大卫弗朗茨% Elena Belousova %迈克尔·弗罗斯特% %雷切尔·库珀曼马蒂娜Bebin %一个布鲁斯Korf %史蒂文Sparagana罗伯特·弗拉米尼%迈克尔·Kohrman % %一个吴乔伊斯% Tarek Sahmoud % Gaurav沙% Sergiusz Jozwiak % T Everolimus治疗肾血管肌脂肪瘤在复杂治疗结节性硬化症患者室巨细胞星形细胞瘤(S45.001) % D J神经病学2012% % P S45.001-S45.001 % V 78% N 1补充% X目的:评估Everolimus,口服mTOR抑制剂,用于治疗血管肌脂肪瘤(AML)病变患者室巨细胞星形细胞瘤(世嘉)与结节性硬化症(TSC)复杂有关。首页背景TSC是一种常染色体显性遗传疾病与肿瘤生长相关的多个器官,包括大脑、肾、肺、心。两个最常见的损伤是在大脑和肾脏AML世嘉。设计/方法:EXIST-1 (NCT00789828)是一个前瞻性,双盲,随机,安慰剂对照,第三阶段的研究。串行世嘉增长从prebaseline基线扫描患者被随机分配(2:1)接收everolimus (n = 78)或安慰剂(n = 39)分层利用enzyme-inducing的抗癫痫药物。Everolimus开始剂量为4.5毫克/ m²/天,调整槽的5 - 15 ng / mL基于病人的耐受性。AML响应率的探索性分析,证实AML患者比例的响应(减少目标AML总额从基线≥50%)≥1目标基线AML患者(最长直径≥1.0厘米;n = 44)是由肾脏CT或MRI评估基线,12日,24日,每年和48周,然后。结果:44例(有everolimus n = 30;安慰剂,n = 14)在基线确定会见了AML入选标准。AML everolimus的反应率为53.3%和0%,安慰剂。在12周、24和48,只有everolimus AML患者减少≥50%(56.5%、78.3%、80.0%)与安慰剂的患者相比(0%),分别为。 A higher percentage of everolimus patients had AML reductions ≥30% (82.6%, 100%, 100%) compared with placebo patients (8.3%, 18.2%, 16.7%) at week 12, 24, and 48, respectively.Conclusions: Patients presenting with SEGA associated with TSC and AML experienced reductions in AML lesion volume with everolimus treatment only and not placebo. Everolimus may offer a pharmacological treatment option for patients with AML and SEGA associated with TSC.Supported by: Novartis.Disclosure: Dr. Franz has received personal compensation for activities with Novartis. Dr. Franz's institution has received research support from Novartis Pharmaceuticals. Dr. Belousova has nothing to disclose. Dr. Frost has nothing to disclose. Dr. Kuperman has nothing to disclose. Dr. Bebin has nothing to disclose. Dr. Korf has nothing to disclose. Dr. Flamini has nothing to disclose. Dr. Kohrman has received personal compensation for activities with UCB Pharma, Abbott Laboratories and Novartis. Dr. Kohrman has received research support from UCB Pharma and GlaxoSmithKline, Inc. Dr. Sparagana has received personal compensation for activities with Novartis, Lundbeck, GlaxoSmithKline, UCB, Elisa, KING, and Sunvion. Dr. Wu has received personal compensation for activities with Novartis. Dr. Sahmoud has received personal compensation for activities Novartis as an employee. Dr. Sahmoud holds stock and/or stock options in Novartis. Dr. Shah has received personal compensation for activities with Novartis as an employee. Dr. Jozwiak has received personal compensation for activities with Novartis as a consultant.Thursday, April 26 2012, 13:00 pm-14:45 pm %U