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心力衰竭治疗评估的成本效益建模方法提案:考虑住院对事件发生率的短期和长期影响。

A Proposal of a Cost-Effectiveness Modeling Approach for Heart Failure Treatment Assessment: Considering the Short- and Long-Term Impact of Hospitalization on Event Rates.

机构信息

University of New South Wales, Sydney, NSW, Australia.

The George Institute for Global Health, Level 5, 1 King St, Newtown, NSW, 2042, Australia.

出版信息

Pharmacoeconomics. 2022 Nov;40(11):1095-1105. doi: 10.1007/s40273-022-01174-2. Epub 2022 Aug 12.

DOI:10.1007/s40273-022-01174-2
PMID:35960435
Abstract

BACKGROUND

The rate of events such as recurrent heart failure (HF) hospitalization and death are known to dramatically increase directly after HF hospitalization. Furthermore, the number of HF hospitalizations is associated with irreversible long-term disease progression, which is in turn associated with increased event rates. However, cost-effectiveness models of HF treatments commonly fail to capture both the short- and long-term association between HF hospitalization and events.

OBJECTIVE

The aim of this study was to provide a decision-analytic model that reflects the short- and long-term association between HF hospitalization and event rates. Furthermore, we assess the impact of omitting these associations.

METHODS

We developed a life-time Markov cohort model to evaluate HF treatments, and modeled the short-term impact of HF hospitalization on event rates via a sequence of tunnel states, with transition probabilities following a parametric survival curve. The corresponding long-term impact was modeled via hazard ratios per HF hospitalization. We obtained baseline event rates and utilities from published literature. Subsequently, we assessed, for a hypothetical HF treatment, how omitting the modeled associations (through a simple two-state model) affects incremental quality-adjusted life-years (QALYs).

RESULTS

We developed a model that incorporates both short- and long-term impacts of HF hospitalizations. Based on an assumed treatment effect of a 20% risk reduction for HF hospitalization (and associated reductions in all-cause mortality of 15%), omitting the short-term, the long-term, or both associations resulted in a 5%, 1%, and 22% decrease in QALYs gained, respectively.

CONCLUSION

For both modeling components, i.e., the short- and long-term implications of HF hospitalization, the impact on incremental outcomes associated with treatment was substantial. Considering these aspects as proposed within this modeling approach better reflects the natural course of this progressive condition and will enhance the evaluation of future HF treatments.

摘要

背景

众所周知,心力衰竭(HF)住院后,诸如心力衰竭复发(HF)住院和死亡等事件的发生率会急剧增加。此外,HF 住院的次数与不可逆转的长期疾病进展相关,而后者又与事件发生率的增加相关。然而,HF 治疗的成本效益模型通常无法同时捕捉 HF 住院与事件之间的短期和长期关联。

目的

本研究旨在提供一种决策分析模型,反映 HF 住院与事件发生率之间的短期和长期关联。此外,我们评估了省略这些关联的影响。

方法

我们开发了一个终生马尔可夫队列模型来评估 HF 治疗,并通过一系列隧道状态来模拟 HF 住院对事件发生率的短期影响,其转移概率遵循参数生存曲线。相应的长期影响通过每例 HF 住院的风险比来建模。我们从已发表的文献中获得了基线事件率和效用值。随后,我们评估了对于一种假设的 HF 治疗,通过一个简单的两状态模型省略建模关联如何影响增量质量调整生命年(QALYs)。

结果

我们开发了一个模型,该模型既包含 HF 住院的短期影响,也包含其长期影响。基于 HF 住院风险降低 20%(并相应降低全因死亡率 15%)的假设治疗效果,省略短期、长期或两者关联会导致 QALYs 分别减少 5%、1%和 22%。

结论

对于 HF 住院的短期和长期影响这两个建模部分,对与治疗相关的增量结果的影响是巨大的。考虑到这种建模方法中的这些方面,可以更好地反映这种进行性疾病的自然病程,并增强对未来 HF 治疗的评估。

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本文引用的文献

1
Health State Utilities of Patients with Heart Failure: A Systematic Literature Review.心力衰竭患者的健康状态效用值:系统文献回顾。
Pharmacoeconomics. 2021 Feb;39(2):211-229. doi: 10.1007/s40273-020-00984-6. Epub 2020 Nov 30.
2
Cardiac Myosin Activation with Omecamtiv Mecarbil in Systolic Heart Failure.肌球蛋白激活治疗收缩性心力衰竭的奥马曲珠单抗
N Engl J Med. 2021 Jan 14;384(2):105-116. doi: 10.1056/NEJMoa2025797. Epub 2020 Nov 13.
3
Cost-effectiveness of Sacubitril-Valsartan in Hospitalized Patients Who Have Heart Failure With Reduced Ejection Fraction.
沙库巴曲缬沙坦治疗射血分数降低的心力衰竭住院患者的成本效果分析。
JAMA Cardiol. 2020 Nov 1;5(11):1236-1244. doi: 10.1001/jamacardio.2020.2822.
4
Developing Markov Models From Real-World Data: A Case Study of Heart Failure Modeling Using Administrative Data.从真实世界数据中开发马尔可夫模型:使用行政数据进行心力衰竭建模的案例研究。
Value Health. 2020 Jun;23(6):743-750. doi: 10.1016/j.jval.2020.02.012.
5
The epidemiology of heart failure in the general Australian community - study of heart failure in the Australian primary carE setting (SHAPE): methods.澳大利亚普通社区心力衰竭的流行病学 - 澳大利亚初级保健环境中心力衰竭研究 (SHAPE):方法。
BMC Public Health. 2020 May 11;20(1):648. doi: 10.1186/s12889-020-08781-8.
6
Readmission and death in patients admitted with new-onset versus worsening of chronic heart failure: insights from a nationwide cohort.新诊断与慢性心力衰竭恶化患者入院后的再入院和死亡:一项全国性队列研究的结果。
Eur J Heart Fail. 2020 Oct;22(10):1777-1785. doi: 10.1002/ejhf.1800. Epub 2020 Mar 30.
7
Omecamtiv Mecarbil in Chronic Heart Failure With Reduced Ejection Fraction: Rationale and Design of GALACTIC-HF.奥马曲班在射血分数降低的慢性心力衰竭中的应用:GALACTIC-HF 的原理和设计。
JACC Heart Fail. 2020 Apr;8(4):329-340. doi: 10.1016/j.jchf.2019.12.001. Epub 2020 Feb 6.
8
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J Am Heart Assoc. 2019 Dec 3;8(23):e014240. doi: 10.1161/JAHA.119.014240. Epub 2019 Nov 27.
9
Prevalence of heart failure and left ventricular dysfunction in China: the China Hypertension Survey, 2012-2015.中国高血压调查 2012-2015 年:心力衰竭和左心室功能障碍的患病率。
Eur J Heart Fail. 2019 Nov;21(11):1329-1337. doi: 10.1002/ejhf.1629.
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J Am Coll Cardiol. 2020 Jan 28;75(3):245-254. doi: 10.1016/j.jacc.2019.11.003. Epub 2019 Nov 11.