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丁苯那嗪用于治疗与亨廷顿舞蹈病相关的舞蹈症。

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

作者信息

Gupta Harshit, Perkins Wil, Stark Cain, Kikkeri Sathya, Kakazu Juyeon, Kaye Adam, Kaye Alan D

机构信息

School of Medicine David Geffen School of Medicine.

School of Medicine Louisiana State University Shreveport School of Medicine.

出版信息

Health Psychol Res. 2022 Jun 28;10(5):36040. doi: 10.52965/001c.36040. eCollection 2022.

Abstract

This is a comprehensive review of the literature regarding the use of Deutetrabenazine in treating chorea associated with Huntington's disease. Unfortunately, treatment has been limited for many aspects of this neurodegenerative disease. The present investigation presents the background, evidence, and indications for the use Deutetrabenazine in the setting of Huntington's disease. Huntington's disease is characterized by a variety of motor, psychiatric, and cognitive symptoms with chorea being one of the more notable ones. Chorea is a movement disorder present in multiple neurologic diseases that causes involuntary and irregular muscle movements theorized to be stemming from high dopamine levels. Deutetrabenazine is thought to function as an inhibitor of the VMAT2 vesicular monoamine transporter resulting in decreased monoamine release, including dopamine, in the synaptic cleft which has a therapeutic effect in management of chorea. This drug was approved by the FDA in 2017 with a specific indication for tardive dyskinesia and choreiform movement in Huntington's disease. Currently, there is no definitive treatment for Huntington's disease. Thus, management is primarily focused on symptom management with the use of a variety of pharmaceutical agents. Chorea is one of the many manifestations that significantly alter the quality of life of many patients. Deutetrabenazine is a promising new option for the treatment of chorea in the setting of Huntington's disease. Although studies so far have displayed mixed results, further research, including head-to-head studies, is necessary to elucidate the true potential of this drug.

摘要

这是一篇关于使用氘代丁苯那嗪治疗亨廷顿舞蹈病相关舞蹈症的文献综述。遗憾的是,针对这种神经退行性疾病的诸多方面,治疗手段一直有限。本研究介绍了在亨廷顿舞蹈病背景下使用氘代丁苯那嗪的背景、证据及适应证。亨廷顿舞蹈病的特征是出现多种运动、精神和认知症状,舞蹈症是其中较为显著的症状之一。舞蹈症是一种存在于多种神经系统疾病中的运动障碍,会导致不自主且不规则的肌肉运动,据推测是由多巴胺水平过高引起的。氘代丁苯那嗪被认为是囊泡单胺转运体2(VMAT2)的抑制剂,可减少突触间隙中包括多巴胺在内的单胺释放,这对舞蹈症的治疗具有疗效。该药物于2017年获得美国食品药品监督管理局(FDA)批准,有治疗亨廷顿舞蹈病迟发性运动障碍和舞蹈样动作的特定适应证。目前,尚无针对亨廷顿舞蹈病的确切治疗方法。因此,治疗主要集中在使用多种药物进行症状管理。舞蹈症是显著影响众多患者生活质量的诸多表现之一。氘代丁苯那嗪是治疗亨廷顿舞蹈病相关舞蹈症的一个有前景的新选择。尽管目前的研究结果不一,但仍需要进一步研究,包括头对头研究,以阐明这种药物的真正潜力。

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