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用于治疗与亨廷顿舞蹈病相关的舞蹈症的氘代丁苯那嗪。

Valbenazine for the treatment of chorea associated with Huntington's disease.

作者信息

Patino Jorge, Furr Stimming Erin, Testa Claudia M, Mehanna Raja

机构信息

Department of Neurology, UTHealth Houston McGovern Medical School, Houston, TX, USA.

Department of Neurology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

出版信息

Expert Opin Pharmacother. 2025 Feb;26(2):127-132. doi: 10.1080/14656566.2024.2445728. Epub 2024 Dec 30.

Abstract

INTRODUCTION

Chorea is a motor manifestation of Huntington's disease (HD), which can lead to decreased functional independence and falls. Even though multiple classes of medications have been used to treat this symptom, only the vesicular monoamine transporter 2 (VMAT2) inhibitors tetrabenazine, deutetrabenazine, and valbenazine have been approved by the FDA for this indication.

AREAS COVERED

This article reviews the pharmacological properties, clinical efficacy, safety, and tolerability of valbenazine in the treatment of chorea in HD. Key considerations in the concomitant use of VMAT2 inhibitors with other medications are discussed, particularly considerations specific to valbenazine use plus medications often used to treat neuropsychiatric symptoms in HD.

EXPERT OPINION

Valbenazine effectively addresses chorea. Its selectivity avoids VMAT1-related side effects and tardive dyskinesia and may result in less off-target side effects such as parkinsonism, behavioral changes, and akathisia. The cost of this medication could be a barrier to access, even for those with insurance, due to high co-pay fees. Head-to-head clinical trials are needed to compare valbenazine's efficacy with the other approved drugs for chorea in HD.

摘要

引言

舞蹈症是亨廷顿舞蹈病(HD)的一种运动表现,可导致功能独立性下降和跌倒。尽管已使用多种药物治疗该症状,但只有囊泡单胺转运体2(VMAT2)抑制剂丁苯那嗪、氘代丁苯那嗪和缬苯那嗪已获美国食品药品监督管理局(FDA)批准用于这一适应症。

涵盖领域

本文综述了缬苯那嗪治疗HD舞蹈症的药理特性、临床疗效、安全性和耐受性。讨论了VMAT2抑制剂与其他药物联合使用时的关键注意事项,特别是缬苯那嗪与常用于治疗HD神经精神症状的药物联合使用时的特殊注意事项。

专家观点

缬苯那嗪能有效治疗舞蹈症。其选择性避免了与VMAT1相关的副作用和迟发性运动障碍,可能会减少诸如帕金森症、行为改变和静坐不能等非靶向副作用。由于高额的自付费用,即使对于有保险的人来说,这种药物的成本也可能成为获取障碍。需要进行直接比较的临床试验,以比较缬苯那嗪与其他已批准用于治疗HD舞蹈症的药物的疗效。

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