Feleus Stephanie, Skotnicki Lara E M, Roos Raymund A C, de Bot Susanne T
Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands.
Mov Disord Clin Pract. 2024 Dec;11(12):1530-1541. doi: 10.1002/mdc3.14230. Epub 2024 Oct 21.
Huntington's Disease is a rare neurodegenerative disorder in which appropriate medication management is essential. While many medications are prescribed based on expert knowledge, overviews of actual medication use in HD are sparse.
We provide a detailed overview of medication use and associated indications across HD disease stages, considering sex and regional differences.
Data from the largest observational HD study, ENROLL-HD, were used. We created HD-related medication and indication classes to identify medication trends in manifest, premanifest and control subjects. We studied medication use in adult, childhood- and adolescent-onset HD, incorporating disease stage (including phenoconverters), sex and regional differences.
In 8546 manifest HD patients, 84.6% used medication (any type), with the average number of medications per user rising from 2.5 in premanifest HD to 5.2 in end stage disease. Antipsychotics (29.2%), SSRIs (27.5%) and painkillers (21.8%) were most often used. Medication use varied with disease progression. Several differences were observed between the sexes, and notably between Europe and Northern America as well. Medication use increased after phenoconversion (from 64.8% to 70.6%, P < 0.05), with the largest difference in antipsychotic use (4.4%-7.8%, P < 0.05). Medication patterns were different in childhood-onset HD, with no use of painkillers, less use of anti-chorea and antidepressant drugs, and more for aggression and irritability.
Medication use in HD increases with disease progression, with varying types of medications prescribed based on disease stage, sex, and region of living. Recognizing these medication trends is vital for further personalized HD management.
亨廷顿舞蹈症是一种罕见的神经退行性疾病，恰当的药物管理至关重要。虽然许多药物是根据专业知识开处方的，但关于亨廷顿舞蹈症实际用药情况的综述却很少。
我们提供了一份关于亨廷顿舞蹈症疾病各阶段用药情况及相关适应症的详细综述，同时考虑了性别和地区差异。
使用了来自最大规模的亨廷顿舞蹈症观察性研究ENROLL-HD的数据。我们创建了与亨廷顿舞蹈症相关的药物和适应症类别，以确定显性、前驱期和对照受试者的用药趋势。我们研究了成年、儿童期和青少年期发病的亨廷顿舞蹈症患者的用药情况，纳入了疾病阶段（包括表型转换者）、性别和地区差异。
在8546例显性亨廷顿舞蹈症患者中，84.6%使用了药物（任何类型），每位使用者的平均用药数量从前驱期亨廷顿舞蹈症的2.5种增加到疾病终末期的5.2种。最常使用的药物是抗精神病药物（29.2%）、选择性5-羟色胺再摄取抑制剂（27.5%）和止痛药（21.8%）。用药情况随疾病进展而变化。观察到了性别之间的一些差异，欧洲和北美之间也存在显著差异。表型转换后用药量增加（从64.8%增至70.6%，P<0.05），抗精神病药物使用差异最大（4.4%-7.8%，P<0.05）。儿童期发病的亨廷顿舞蹈症用药模式不同，不使用止痛药，抗舞蹈症和抗抑郁药物使用较少，更多用于攻击行为和易怒。
亨廷顿舞蹈症的用药量随疾病进展而增加，根据疾病阶段、性别和居住地区会开出不同类型的药物。认识这些用药趋势对于进一步的亨廷顿舞蹈症个性化管理至关重要。
