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帕金森病新疗法的经济及与健康相关的生活质量考量

Economic and health-related quality of life considerations of new therapies in Parkinson's disease.

作者信息

Rubenstein L M, DeLeo A, Chrischilles E A

机构信息

Department of Epidemiology, College of Public Health, University of Iowa, Iowa City, 52242, USA.

出版信息

Pharmacoeconomics. 2001;19(7):729-52. doi: 10.2165/00019053-200119070-00003.

Abstract

The progressive disability of Parkinson's disease results in substantial burdens for patients, their families and society in terms of increased health resource use, poorer quality of life, caregiver burden, disrupted family relationships, decreases in social and leisure activities, deteriorating emotional well-being, and direct and indirect costs of illness. Health-related quality of life (HR-QOL) measures have been used successfully in cross-sectional studies to identify and characterise these burdens; however, there is not yet substantial evidence that these instruments will be responsive to changes in patients over time and that the results will provide patients and health professionals with clinically meaningful information useful in making decisions about treatment strategies. The few studies documenting direct and indirect costs indicate increased use of ancillary health and community services, significant adaptations in home and transportation, increased use of mobility and self-care aids, and lack of access to appropriate healthcare providers. Patients with Parkinson's disease incur higher hospital expenses, have increased number of prescriptions, and experience earnings loss; the latter also applies to family caregivers. The choice, intensity and timing of therapy are determined by a variety of factors: presenting symptoms, age, employment status, comorbidity, cognitive impairment and level of functional impairment. Choices must be individually tailored to a patient's physical and personal needs. To be useful for patients with Parkinson's disease in clinical practice, clinicians should be able to use HR-QOL measures to identify appropriate medical interventions or socio-behavioural modifications to modify the HR-QOL deficits. However, while the interplay of interventions and clinical outcomes are often well understood, the effects of interventions on HR-QOL outcomes have not been studied extensively. Little research has been done that explicitly links the signs and symptoms of Parkinson's disease to the HR-QOL outcomes. The only Parkinson's disease cost-effectiveness study as yet performed indicated higher costs for patients receiving pramipexole than for those not taking the drug, but additional quality life-years were gained. Longer term effectiveness of many treatment strategies, and the usefulness of HR-QOL instruments to assess these treatments for individual patients over time, are critical areas for future research.

摘要

帕金森病的进行性残疾给患者、其家庭和社会带来了沉重负担,包括卫生资源使用增加、生活质量下降、照料者负担、家庭关系破裂、社交和休闲活动减少、情绪健康恶化以及疾病的直接和间接成本。与健康相关的生活质量(HR-QOL)测量方法已成功用于横断面研究,以识别和描述这些负担;然而,尚无充分证据表明这些工具能随时间反映患者的变化,其结果能为患者和医疗专业人员提供对制定治疗策略有临床意义的有用信息。少数记录直接和间接成本的研究表明,辅助医疗和社区服务的使用增加、家庭和交通方面的重大调整、移动和自我护理辅助工具的使用增加以及无法获得合适的医疗服务提供者。帕金森病患者的住院费用更高、处方数量增加且收入减少;后者也适用于家庭照料者。治疗的选择、强度和时机由多种因素决定:出现的症状、年龄、就业状况、合并症、认知障碍和功能障碍程度。选择必须根据患者的身体和个人需求进行个性化定制。为了在临床实践中对帕金森病患者有用,临床医生应能够使用HR-QOL测量方法来识别适当的医疗干预或社会行为调整,以改善HR-QOL缺陷。然而,虽然干预措施与临床结果之间的相互作用通常得到很好的理解,但干预措施对HR-QOL结果的影响尚未得到广泛研究。很少有研究明确将帕金森病的体征和症状与HR-QOL结果联系起来。迄今为止唯一进行的帕金森病成本效益研究表明,接受普拉克索治疗的患者比未服用该药物的患者成本更高,但获得了额外的质量调整生命年。许多治疗策略的长期有效性以及HR-QOL工具随时间评估个体患者这些治疗的有用性,是未来研究的关键领域。

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