Tetrabenazine as anti-chorea therapy in Huntington disease: an open-label continuation study. Huntington Study Group/TETRA-HD Investigators.
Tetrabenazine as anti-chorea therapy in Huntington disease: an open-label continuation study. Huntington Study Group/TETRA-HD Investigators.
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DOI：
10.1186/1471-2377-9-62
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发表时间：
2009-12-18
期刊：
影响因子：
2.6
通讯作者：
Frank S
中科院分区：
文献类型：
--
作者：
Frank S
Tetrabenazine (TBZ) 通过与 2 型囊泡单胺转运蛋白可逆结合来选择性地消耗中心单胺。先前针对亨廷顿病 (HD) 的一项双盲研究表明，TBZ 可以有效抑制舞蹈病，并具有良好的短期安全性 (Neurology 2006;66:366-372)。本研究的目的是评估 TBZ 治疗 HD 舞蹈病的长期安全性和有效性。完成 13 周双盲方案的受试者被邀请参加这项长达 80 周的开放标签扩展研究。受试者逐渐调整至最佳个体剂量或最大 200 毫克/天。使用统一亨廷顿病评定量表中的总最大舞蹈病（TMC）评分来评估舞蹈症。在 75 名参与者中，45 名受试者完成了 80 周。三名参与者因不良事件 (AE) 终止，包括抑郁、与先前自杀行为相关的妄想和声音抽动。一名受试者因乳腺癌死亡。其他 26 名受试者由于各种原因选择不继续进行随后的每次延期。当排除轻度和不相关的 AE 时，最常报告的 AE（受试者数量）是镇静/嗜睡 (18)、情绪抑郁 (17)、焦虑 (13)、失眠 (10) 和静坐不能 (9)。与基线相比，第 80 周时帕金森症和吞咽困难评分显着增加。第 80 周时，舞蹈病较基线显着改善，TMC 评分平均降低 4.6 (SD 5.5) 个单位。第 80 周时的平均剂量为 63.4 mg（范围 12.5-175 mg）。 TBZ 可有效抑制 HD 相关舞蹈症长达 80 周。长期接受 TBZ 治疗的患者应监测帕金森症、吞咽困难和其他副作用，包括睡眠障碍、抑郁、焦虑和静坐不能。 ClinicalTrials.gov 注册号（初步研究）：NCT00219804
Tetrabenazine (TBZ) selectively depletes central monoamines by reversibly binding to the type-2 vesicular monoamine transporter. A previous double blind study in Huntington disease (HD) demonstrated that TBZ effectively suppressed chorea, with a favorable short-term safety profile (Neurology 2006;66:366-372). The objective of this study was to assess the long-term safety and effectiveness of TBZ for chorea in HD. Subjects who completed the 13-week, double blind protocol were invited to participate in this open label extension study for up to 80 weeks. Subjects were titrated to the best individual dose or a maximum of 200 mg/day. Chorea was assessed using the Total Maximal Chorea (TMC) score from the Unified Huntington Disease Rating Scale. Of the 75 participants, 45 subjects completed 80 weeks. Three participants terminated due to adverse events (AEs) including depression, delusions with associated previous suicidal behavior, and vocal tics. One subject died due to breast cancer. The other 26 subjects chose not to continue on with each ensuing extension for various reasons. When mild and unrelated AEs were excluded, the most commonly reported AEs (number of subjects) were sedation/somnolence (18), depressed mood (17), anxiety (13), insomnia (10), and akathisia (9). Parkinsonism and dysphagia scores were significantly increased at week 80 compared to baseline. At week 80, chorea had significantly improved from baseline with a mean reduction in the TMC score of 4.6 (SD 5.5) units. The mean dosage at week 80 was 63.4 mg (range 12.5-175 mg). TBZ effectively suppresses HD-related chorea for up to 80 weeks. Patients treated chronically with TBZ should be monitored for parkinsonism, dysphagia and other side effects including sleep disturbance, depression, anxiety, and akathisia. Clinicaltrials.gov registration number (initial study): NCT00219804
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