H49亨廷顿舞蹈病药物治疗的有效性

K. Doleckova, P. Dušek, R. Konvalinková, J. Roth, J. Klempír
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摘要

亨廷顿病(HD)运动障碍对症治疗的潜在益处仍在讨论中。本研究的目的是了解不同阶段药物治疗的效果,在我们的Registry数据库中包含309例检查患者(年龄50.3±13.4岁,病程9.13±3.51年,CAG 40 ~ 70次重复),分析不同药物的使用情况并进行比较。为了客观评价患者的表现,采用统一亨廷顿病评定量表-总运动评分、运动量表分(自主、眼动、舞蹈病、肌张力障碍、强直)、总功能容量和功能评估。利培酮是使用最多的药物,有154项评估接受了这种治疗,155项评估没有接受这种治疗。其他具有最强代表性的制剂有金刚烷胺(97:212)、氯硝西泮(56:253)、噻必利(49:260)、四苯那嗪(13:296)和氟哌啶醇(11:298)。统计学上(Mann-Whitney u检验)比较了接受和未接受特殊药物治疗的患者,只有氯硝西泮治疗的患者有显著的结果,他们只有明显更高的自主运动亚评分(p<0.001)。此外,我们使用普通最小二乘计算进行线性模型,以计算疾病进展的影响(年龄、疾病持续时间、CAG重复次数)。自主运动评分的差异与疾病进展无关。虽然应该进行纵向研究来证明氯硝西泮的因果关系,但在给HD患者开氯硝西泮处方时应谨慎。
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H49 Effectivness of pharmacotherapy in huntington’s disease
The potentional benefit of symptomatic treatment of motor impairment in Huntington´s disease (HD) is still discussed. The aim of this study was to find out the effect of medication at different stages of this disease, In our Registry database containing 309 examinations of patients (aged 50.3±13.4 years, disease duration 9.13±3.51 years, CAG ranging from 40 to 70 repeats) was analysed usage of different drugs in comparison. To objectify performance of patients, Unified Huntington´s Disease Rating Scale – total motor score, motor scale subscores (voluntary, occulomotor, chorea, dystonia, rigidity), Total Functional Capacity and Functional Assessment were used. Risperidone was the most used medication with 154 assessements with this treatment and 155 without. Other preparates with the strongest representation were amantadine (97:212 events), clonazepam (56:253), tiapride (49:260), tetrabenazine (13:296) and haloperidol (11:298). Statistically (Mann-Whitney U-test) were compared patients with and without particular medical treatement, with only significant results in patients with clonazepam therapy, who had only significantly higher voluntary motor subscore (p<0.001). Additionally, we performed a linear model using ordinary least squares calculation to count for the effect of disease progression (age, disease duration, CAG repeats). The difference in voluntary motor subscore was independent on the disease progression. Although a longitudinal study should be performed to prove causality of clonazepam, caution should be used when prescribing clonazepam in HD.
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