Gerasimos Dumi, Dara O'Neill, Christina Daskalopoulou, Tom Keeley, Stephanie Rhoten, Dharmraj Sauriyal, Piper Fromy
{"title":"日记测量的探索性和确认性因素分析中不同数据处理策略的影响:使用模拟和真实世界的哮喘夜间症状日记数据进行评估。","authors":"Gerasimos Dumi, Dara O'Neill, Christina Daskalopoulou, Tom Keeley, Stephanie Rhoten, Dharmraj Sauriyal, Piper Fromy","doi":"10.1080/10543406.2024.2310312","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Daily diaries are an important modality for patient-reported outcome assessment. They typically comprise multiple questions, so understanding their underlying structure is key to appropriate analysis and interpretation. Structural evaluation of such measures poses challenges due to the high volume of repeated measurements. Potential strategies include selecting a single day, averaging item-level observations over time, or using all data while accounting for its multilevel structure.</p><p><strong>Method: </strong>The above strategies were evaluated in a simulated dataset via exploratory and confirmatory factor modelling by comparing their impact on various estimates (i.e., inter-item correlations, factor loadings, model fit). Each strategy was additionally explored using real-world data from an observational study (the Asthma Nighttime Symptoms Diary).</p><p><strong>Results: </strong>Both single day and item average strategies resulted in biased factor loadings. The former displayed lower overall bias (single day: 0.064; item average: 0.121) and mean square error (single day: 0.007; item average: 0.016) but greater frequency of incorrect factor number identification compared with the latter (single day: 46.4%; item average: 0%). Increased estimated inter-item correlations were apparent in the item-average method. Non-trivial between- and within-person variance highlighted the utility of a multilevel approach. However, convergence issues and Heywood cases were more common under the multilevel approach (90.2% and 100.0%, respectively).</p><p><strong>Conclusions: </strong>Our findings suggest that a multilevel approach can enhance our insight when evaluating the structural properties of daily diary data; however, implementation challenges still remain. Our work offers guidance on the impact of data handling decisions in diary assessment.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"944-968"},"PeriodicalIF":1.2000,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"The impact of different data handling strategies in exploratory and confirmatory factor analysis of diary measures: an evaluation using simulated and real-world asthma nighttime symptoms diary data.\",\"authors\":\"Gerasimos Dumi, Dara O'Neill, Christina Daskalopoulou, Tom Keeley, Stephanie Rhoten, Dharmraj Sauriyal, Piper Fromy\",\"doi\":\"10.1080/10543406.2024.2310312\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Daily diaries are an important modality for patient-reported outcome assessment. They typically comprise multiple questions, so understanding their underlying structure is key to appropriate analysis and interpretation. Structural evaluation of such measures poses challenges due to the high volume of repeated measurements. Potential strategies include selecting a single day, averaging item-level observations over time, or using all data while accounting for its multilevel structure.</p><p><strong>Method: </strong>The above strategies were evaluated in a simulated dataset via exploratory and confirmatory factor modelling by comparing their impact on various estimates (i.e., inter-item correlations, factor loadings, model fit). Each strategy was additionally explored using real-world data from an observational study (the Asthma Nighttime Symptoms Diary).</p><p><strong>Results: </strong>Both single day and item average strategies resulted in biased factor loadings. The former displayed lower overall bias (single day: 0.064; item average: 0.121) and mean square error (single day: 0.007; item average: 0.016) but greater frequency of incorrect factor number identification compared with the latter (single day: 46.4%; item average: 0%). Increased estimated inter-item correlations were apparent in the item-average method. Non-trivial between- and within-person variance highlighted the utility of a multilevel approach. However, convergence issues and Heywood cases were more common under the multilevel approach (90.2% and 100.0%, respectively).</p><p><strong>Conclusions: </strong>Our findings suggest that a multilevel approach can enhance our insight when evaluating the structural properties of daily diary data; however, implementation challenges still remain. Our work offers guidance on the impact of data handling decisions in diary assessment.</p>\",\"PeriodicalId\":54870,\"journal\":{\"name\":\"Journal of Biopharmaceutical Statistics\",\"volume\":\" \",\"pages\":\"944-968\"},\"PeriodicalIF\":1.2000,\"publicationDate\":\"2025-08-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Biopharmaceutical Statistics\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1080/10543406.2024.2310312\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/2/14 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q4\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Biopharmaceutical Statistics","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/10543406.2024.2310312","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/2/14 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
The impact of different data handling strategies in exploratory and confirmatory factor analysis of diary measures: an evaluation using simulated and real-world asthma nighttime symptoms diary data.
Background: Daily diaries are an important modality for patient-reported outcome assessment. They typically comprise multiple questions, so understanding their underlying structure is key to appropriate analysis and interpretation. Structural evaluation of such measures poses challenges due to the high volume of repeated measurements. Potential strategies include selecting a single day, averaging item-level observations over time, or using all data while accounting for its multilevel structure.
Method: The above strategies were evaluated in a simulated dataset via exploratory and confirmatory factor modelling by comparing their impact on various estimates (i.e., inter-item correlations, factor loadings, model fit). Each strategy was additionally explored using real-world data from an observational study (the Asthma Nighttime Symptoms Diary).
Results: Both single day and item average strategies resulted in biased factor loadings. The former displayed lower overall bias (single day: 0.064; item average: 0.121) and mean square error (single day: 0.007; item average: 0.016) but greater frequency of incorrect factor number identification compared with the latter (single day: 46.4%; item average: 0%). Increased estimated inter-item correlations were apparent in the item-average method. Non-trivial between- and within-person variance highlighted the utility of a multilevel approach. However, convergence issues and Heywood cases were more common under the multilevel approach (90.2% and 100.0%, respectively).
Conclusions: Our findings suggest that a multilevel approach can enhance our insight when evaluating the structural properties of daily diary data; however, implementation challenges still remain. Our work offers guidance on the impact of data handling decisions in diary assessment.
期刊介绍:
The Journal of Biopharmaceutical Statistics, a rapid publication journal, discusses quality applications of statistics in biopharmaceutical research and development. Now publishing six times per year, it includes expositions of statistical methodology with immediate applicability to biopharmaceutical research in the form of full-length and short manuscripts, review articles, selected/invited conference papers, short articles, and letters to the editor. Addressing timely and provocative topics important to the biostatistical profession, the journal covers:
Drug, device, and biological research and development;
Drug screening and drug design;
Assessment of pharmacological activity;
Pharmaceutical formulation and scale-up;
Preclinical safety assessment;
Bioavailability, bioequivalence, and pharmacokinetics;
Phase, I, II, and III clinical development including complex innovative designs;
Premarket approval assessment of clinical safety;
Postmarketing surveillance;
Big data and artificial intelligence and applications.