Killoran Annie, Biglan Kevin M
Experimental Therapeutics in Movement Disorders, University of Rochester School of Medicine and Dentistry, 1351 Mt. Hope Avenue, Suite 223, Rochester, NY, 14620, USA,
Curr Treat Options Neurol. 2012 Feb 8. doi: 10.1007/s11940-012-0165-x.
There is no specific treatment for Huntington's disease (HD). Its many symptoms of motor, psychiatric, and cognitive deterioration are managed with symptomatic relief, rehabilitation, and support. The only drug approved by the US Food and Drug Administration (FDA) for the treatment of HD is an antichoreic agent, tetrabenazine, but this drug is used sparingly because of uneasiness regarding its propensity to cause depression and suicidality in this population, which is already at risk for these complications. Neuroleptics are still first-line treatments for chorea accompanied by comorbid depression and/or behavioral or psychotic symptoms, as is often the case. Psychiatric features, which have a significant impact on a patient's professional and personal life, often become the major focus of management. In addition to neuroleptics, commonly used medications include antidepressants, mood stabilizers, anxiolytics, and psychostimulants. In contrast, few treatment options are available for cognitive impairment in HD; this remains an important and largely unmet therapeutic need. HD patients typically lack insight into their disease manifestations, failing to recognize their need for treatment, and possibly even arguing against it. Multipurpose medications are employed advantageously to simplify the medication regimen, so as to facilitate compliance and not overwhelm the patient. For example, haloperidol can be prescribed for a patient with chorea, agitation, and anorexia, rather than targeting each symptom with a different drug. This approach also limits the potential for adverse effects, which can be difficult to distinguish from the features of the disease itself. With HD's complexity, it is best managed with a multidisciplinary approach that includes a movement disorders specialist, a genetic counselor, a mental health professional, a physical therapist, and a social worker for support and coordination of services. As the disease progresses, there may be need for other specialists, such as a speech and occupational therapist, a nutritionist for weight loss, and ultimately, a palliative care specialist.
亨廷顿舞蹈症(HD)尚无特效治疗方法。其运动、精神和认知功能衰退的诸多症状通过对症缓解、康复治疗及支持措施来进行管理。美国食品药品监督管理局(FDA)批准用于治疗HD的唯一药物是抗舞蹈症药物丁苯那嗪,但由于担心该药会使该人群(本身就有这些并发症风险)出现抑郁和自杀倾向,所以使用较少。抗精神病药物仍是伴有共病抑郁和/或行为或精神症状的舞蹈症的一线治疗药物,情况通常如此。对患者职业和个人生活有重大影响的精神症状往往成为治疗的主要重点。除抗精神病药物外,常用药物还包括抗抑郁药、心境稳定剂、抗焦虑药和精神振奋药。相比之下,HD认知障碍的治疗选择很少;这仍然是一个重要且基本未得到满足的治疗需求。HD患者通常对自身疾病表现缺乏洞察力,意识不到自己需要治疗,甚至可能对此表示反对。使用多功能药物有助于简化用药方案,以促进患者依从性且不让患者负担过重。例如,对于患有舞蹈症、激越和厌食症的患者,可以开具氟哌啶醇,而不是针对每种症状使用不同药物。这种方法还能限制不良反应的可能性,因为不良反应可能难以与疾病本身的特征区分开来。鉴于HD的复杂性,最好采用多学科方法进行管理,该方法包括运动障碍专家、遗传咨询师心理健康专业人员、物理治疗师以及提供支持和服务协调的社会工作者。随着疾病进展,可能需要其他专家,如言语和职业治疗师、负责体重减轻的营养师,最终还需要姑息治疗专家。