Modeling severe uncontrolled asthma: Transitioning away from health states

IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL
Tereza Lanitis , Asif H. Khan , Irina Proskorovsky , Ivan Houisse , Andreas Kuznik , Siddhesh Kamat , Conrado Franco-Villalobos , Florence Joulain
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引用次数: 0

Abstract

Background

Models developed to date to simulate long-term outcomes of asthma have been criticized for lacking granularity and ignoring disease heterogeneity.

Objective

To propose an alternative approach to modeling asthma and apply it to model long-term outcomes in a population with moderate-to-severe type 2 asthma (patients with raised fractional exhaled nitric oxide or eosinophils) and treated with conventional therapy.

Methods

A discretely integrated condition event (DICE) approach was adopted, simulating individual profiles with asthma over patients’ lifetime in terms of exacerbations, asthma-related death, and death unrelated to asthma. The timing of these events is dependent on profile characteristics including lung function, asthma control, exacerbation history, and other baseline characteristics or contextual factors. Predictive equations were derived from a clinical trial to model time to exacerbation, change in asthma control, lung function, and utility. Real-world studies were used to supplement data gaps. Outcomes evaluated included life expectancy, quality-adjusted life-years (QALY), number of exacerbations, and lung function over time.

Results

Average annual rates of severe and moderate exacerbations were 1.82 and 3.08 respectively, with rates increasing over time. Lung function declined at a higher rate compared with the general population. Average life expectancy was 75.2 years, compared with 82.4 years in a matched general population. The majority of life-years were spent with uncontrolled asthma and impaired lung function.

Conclusion

Patients with moderate-to-severe type 2 asthma and a history of exacerbations suffer from frequent exacerbations and reduced lung function and life expectancy. Capturing multiple conditions to simulate long-term outcomes in patients with asthma may provide more realistic projections of exacerbation rates.
模拟严重失控的哮喘:从健康状态过渡
背景迄今为止,为模拟哮喘的长期预后而开发的模型因缺乏精细度和忽视疾病的异质性而饱受诟病。方法 采用离散综合条件事件(DICE)方法,模拟哮喘患者一生中哮喘加重、哮喘相关死亡和与哮喘无关死亡的个体特征。这些事件发生的时间取决于个人特征,包括肺功能、哮喘控制情况、哮喘加重史以及其他基线特征或背景因素。通过临床试验得出了预测方程,以模拟哮喘恶化的时间、哮喘控制的变化、肺功能和效用。真实世界研究用于补充数据缺口。评估的结果包括预期寿命、质量调整生命年(QALY)、哮喘加重次数和肺功能随时间的变化。与普通人群相比,肺功能的下降率更高。平均预期寿命为 75.2 岁,而相匹配的普通人群为 82.4 岁。结论患有中重度 2 型哮喘并有病情加重病史的患者病情会频繁加重,肺功能和预期寿命都会缩短。捕捉多种情况来模拟哮喘患者的长期预后,可以更真实地预测病情恶化率。
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来源期刊
Contemporary Clinical Trials Communications
Contemporary Clinical Trials Communications Pharmacology, Toxicology and Pharmaceutics-Pharmacology
CiteScore
2.70
自引率
6.70%
发文量
146
审稿时长
20 weeks
期刊介绍: Contemporary Clinical Trials Communications is an international peer reviewed open access journal that publishes articles pertaining to all aspects of clinical trials, including, but not limited to, design, conduct, analysis, regulation and ethics. Manuscripts submitted should appeal to a readership drawn from a wide range of disciplines including medicine, life science, pharmaceutical science, biostatistics, epidemiology, computer science, management science, behavioral science, and bioethics. Contemporary Clinical Trials Communications is unique in that it is outside the confines of disease specifications, and it strives to increase the transparency of medical research and reduce publication bias by publishing scientifically valid original research findings irrespective of their perceived importance, significance or impact. Both randomized and non-randomized trials are within the scope of the Journal. Some common topics include trial design rationale and methods, operational methodologies and challenges, and positive and negative trial results. In addition to original research, the Journal also welcomes other types of communications including, but are not limited to, methodology reviews, perspectives and discussions. Through timely dissemination of advances in clinical trials, the goal of Contemporary Clinical Trials Communications is to serve as a platform to enhance the communication and collaboration within the global clinical trials community that ultimately advances this field of research for the benefit of patients.
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