文摘
摘要目的:描述的有效性和安全性水泡2单胺转运蛋白抑制剂deutetrabenazine (DTBZ)结合抗精神病药物(APs)亨廷顿病(HD) -相关舞蹈病患者在现实的设置。
背景:DTBZ HD-related舞蹈病的有效性和安全性已被记录在临床试验;然而,病人接受APs并不包括在内。安全性和有效性数据DTBZ + AP需要通知实际练习。
设计/方法:数据收集从图表HD-associated舞蹈病患者接受DTBZ + AP范德比尔特大学医学中心从4月3日,2017年,直到数据收集(May-November 2021)。效果评估总最大舞蹈病(TMC)的分数。安全评估通过药品不良反应(adr)基于预定义的已知DTBZ adr的列表。
结果:DTBZ + AP患者57,13日开始APs第一,TMC分数在1个月之前和之后的任何时间DTBZ,和8 TMC得分前1月内美联社开始可用。后启动APs在添加DTBZ之前,TMC分数提高了2/8(25%)患者和恶化6 (75%)。添加DTBZ之后,TMC成绩进一步提高8/13(62%)的病人,并没有改变1例(8%),4(31%)恶化。四苯喹嗪史患者使用,TMC成绩提高后增加DTBZ 4/8(50%)和4/5(80%)的病人没有历史。在所有患者57 DTBZ + AP, 25例(44%)患者持续≥1新/ ADR,搅拌,静坐不能(n = 7;12%)和嗜睡(n = 4;7%)最常见;2例(4%)患者停止DTBZ因为adr /耐受性问题而接受组合治疗。
结论:在这回顾图表回顾,DTBZ APs改善电动机迹象,有利其概要文件。然而,前瞻性对照试验研究的有效性和安全性DTBZ + AP HD-related舞蹈病患者是必要的。
披露:Claassen博士已经收到个人薪酬在5000 - 9999美元的范围作为顾问Teva神经科学。Claassen博士已经收到个人薪酬在500 - 4999美元的范围作为顾问火花。Claassen博士的机构已收到个人薪酬在50000 - 99999美元的范围为短语担任顾问。Claassen博士已经收到个人薪酬在5000 - 9999美元的范围为在科学咨询服务或数据安全监测委员会Teva神经科学。Claassen博士已经收到个人薪酬在500 - 4999美元的范围作为一个编辑,副主编,或编辑顾问委员会成员对HD的见解。Claassen博士的机构收到NIH的研究支持。Claassen博士的机构已经接到CHDI研究支持。Claassen博士的机构已经接到HDSA研究支持。Claassen博士的机构已经收到了来自国防部的研究支持。Claassen博士的机构已收到研究格里芬家庭基金会的支持。 The institution of Dr. Claassen has received research support from Neurocrine. The institution of Dr. Claassen has received research support from Vaccinex. The institution of Dr. Claassen has received research support from AbbVie. The institution of Dr. Claassen has received research support from CHDI. The institution of Dr. Claassen has received research support from Genentech/ Roche. The institution of Dr. Claassen has received research support from Prilenia. The institution of Dr. Claassen has received research support from Neurocrine/ HSG. Dr. Romdhani has nothing to disclose. Rajeev Ayyagari, PhD has received personal compensation for serving as an employee of Analysis Group, Inc. . Debbie Goldschmidt has received personal compensation for serving as an employee of Analysis Group. Ms. Moroz has nothing to disclose. Ms. Hernandez has nothing to disclose. Nayla Chaijale has received personal compensation for serving as an employee of Teva Pharmaceuticals. Dr. Leo has received personal compensation for serving as an employee of Teva Pharmaceutical Industries.
