在哮喘吸入皮质类固醇的比较有效性研究中应用的精确匹配和倾向评分方法的评价。

IF 2.3 Q2 MEDICINE, GENERAL & INTERNAL Pragmatic and Observational Research Pub Date : 2017-03-22 eCollection Date: 2017-01-01 DOI:10.2147/POR.S122563
Anne Burden, Nicolas Roche, Cristiana Miglio, Elizabeth V Hillyer, Dirkje S Postma, Ron Mc Herings, Jetty A Overbeek, Javaria Mona Khalid, Daniela van Eickels, David B Price
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引用次数: 48

摘要

背景:观察性研究在估计治疗效果时使用队列匹配和回归模型来控制混杂因素。我们的目的是评估精确匹配和倾向评分方法,将它们应用于1年的前后历史数据库研究,以调查哮喘治疗相关的结果。方法:我们利用PHARMO数据库中哮喘患者的纵向病历数据进行比较(环来奈德和细颗粒吸入皮质类固醇[ICS])。我们评估的倾向评分方法包括使用两种不同算法的倾向评分匹配(PSM)、处理加权逆概率(IPTW)、使用倾向评分的协变量调整和倾向评分分层。我们使用标准化差异来定义平衡,作为结果的差异:在4064名符合条件的患者中,1382名(34%)患者使用环奈德,2682名(66%)患者使用细颗粒ICS。IPTW和基于倾向评分的方法比精确匹配(90%)保留了更多的患者(96%-100%);精确匹配选择较轻的患者。结论:我们发现每种方法都有其独特的优势,我们建议每个匹配的队列研究至少应用两种方法来评估研究结果的稳健性。平衡诊断应应用于所有方法,以检查治疗队列之间混杂因素的平衡。如果使用精确匹配,倾向评分的计算可能有助于识别需要平衡的变量,从而告知匹配标准的选择以及临床考虑。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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An evaluation of exact matching and propensity score methods as applied in a comparative effectiveness study of inhaled corticosteroids in asthma.

Background: Cohort matching and regression modeling are used in observational studies to control for confounding factors when estimating treatment effects. Our objective was to evaluate exact matching and propensity score methods by applying them in a 1-year pre-post historical database study to investigate asthma-related outcomes by treatment.

Methods: We drew on longitudinal medical record data in the PHARMO database for asthma patients prescribed the treatments to be compared (ciclesonide and fine-particle inhaled corticosteroid [ICS]). Propensity score methods that we evaluated were propensity score matching (PSM) using two different algorithms, the inverse probability of treatment weighting (IPTW), covariate adjustment using the propensity score, and propensity score stratification. We defined balance, using standardized differences, as differences of <10% between cohorts.

Results: Of 4064 eligible patients, 1382 (34%) were prescribed ciclesonide and 2682 (66%) fine-particle ICS. The IPTW and propensity score-based methods retained more patients (96%-100%) than exact matching (90%); exact matching selected less severe patients. Standardized differences were >10% for four variables in the exact-matched dataset and <10% for both PSM algorithms and the weighted pseudo-dataset used in the IPTW method. With all methods, ciclesonide was associated with better 1-year asthma-related outcomes, at one-third the prescribed dose, than fine-particle ICS; results varied slightly by method, but direction and statistical significance remained the same.

Conclusion: We found that each method has its particular strengths, and we recommend at least two methods be applied for each matched cohort study to evaluate the robustness of the findings. Balance diagnostics should be applied with all methods to check the balance of confounders between treatment cohorts. If exact matching is used, the calculation of a propensity score could be useful to identify variables that require balancing, thereby informing the choice of matching criteria together with clinical considerations.

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来源期刊
Pragmatic and Observational Research
Pragmatic and Observational Research MEDICINE, GENERAL & INTERNAL-
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发文量
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期刊介绍: Pragmatic and Observational Research is an international, peer-reviewed, open-access journal that publishes data from studies designed to closely reflect medical interventions in real-world clinical practice, providing insights beyond classical randomized controlled trials (RCTs). While RCTs maximize internal validity for cause-and-effect relationships, they often represent only specific patient groups. This journal aims to complement such studies by providing data that better mirrors real-world patients and the usage of medicines, thus informing guidelines and enhancing the applicability of research findings across diverse patient populations encountered in everyday clinical practice.
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