Patil Divya, Gorman Emily F, Mattingly T Joseph
University of Maryland School of Pharmacy, Baltimore, MD, USA.
Health Sciences and Human Services Library, University of Maryland, Baltimore, MD, USA.
Pharmacoecon Open. 2025 Jan;9(1):5-13. doi: 10.1007/s41669-024-00531-5. Epub 2024 Oct 15.
Huntington's disease (HD) is associated with significant financial burden for patients and payers. The objective was to identify and quantify costs of HD by stage of disease progression.
A systematic search of Medline, Embase, Scopus, and Cochrane Library was used to identify types of costs that are frequently reported for individuals diagnosed with HD and to quantify those costs across early, middle, and late stages of HD. Full-text, original research articles were included if they reported costs specific to HD burden. To standardize stage-level costs, Shoulson Fahn and Barthel Index scores were combined to estimate the cost for early, middle-, and late-stage HD.
A total of 692 abstracts were identifie,d and following abstract screening, a total of 80 full-text articles were reviewed for inclusion. Only five studies were included for extraction and synthesis including three from the USA, one from the United Kingdom (UK), and one from Peru. Annual inpatient, outpatient, drug costs, and caregiving costs all increased substantially as disease progressed. Outpatient costs were approximately 2.5 times greater than inpatient costs for early and middle stages of HD. Among all the costs associated with HD, annual caregiver cost emerges as the most significant costs in the economic burden of HD, ranging from a minimum of $6041 for early stage to a maximum of $133,200 for late-stage HD. Significant variation was observed across studies, especially comparing costs observed in Peru with the USA and UK.
Outpatient costs exceed inpatient costs, especially in early and middle stages, underscoring the importance of outpatient care. All costs seem to rise rapidly, in a nonlinear fashion, as patients advance to later stages. While only two studies reported caregiver burden, these costs were significanly higher in the most severe stage, where patients were completely dependent on a caregiver. This review highlights the complexity of cost assessment in HD and underscores the need for consistent methods and further research to guide effective policy actions.
亨廷顿舞蹈症（HD）给患者和支付方带来了巨大的经济负担。目的是确定并量化HD在疾病进展各阶段的成本。
系统检索Medline、Embase、Scopus和Cochrane图书馆，以确定HD患者经常报告的成本类型，并量化HD早期、中期和晚期的这些成本。如果全文原始研究文章报告了HD负担的特定成本，则纳入其中。为了标准化各阶段成本，将Shoulson Fahn和巴氏指数评分相结合，以估计HD早期、中期和晚期的成本。
共识别出692篇摘要，经过摘要筛选后，共审查了80篇全文文章以确定是否纳入。仅纳入了五项研究进行提取和综合分析，其中三项来自美国，一项来自英国，一项来自秘鲁。随着疾病进展，年度住院、门诊、药物成本和护理成本均大幅增加。HD早期和中期的门诊成本约为住院成本的2.5倍。在与HD相关的所有成本中，年度护理成本是HD经济负担中最显著的成本，从早期的最低6041美元到晚期的最高133200美元不等。各研究之间观察到显著差异，特别是将秘鲁观察到的成本与美国和英国进行比较时。
门诊成本超过住院成本，尤其是在早期和中期，这凸显了门诊护理的重要性。随着患者进入后期阶段，所有成本似乎都以非线性方式迅速上升。虽然只有两项研究报告了护理负担，但这些成本在最严重阶段显著更高，此时患者完全依赖护理人员。本综述强调了HD成本评估的复杂性，并强调需要一致的方法和进一步研究以指导有效的政策行动。
