Medical Decision Making最新文献

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Unclear Trajectory and Uncertain Benefit: Creating a Lexicon for Clinical Uncertainty in Patients with Critical or Advanced Illness Using a Delphi Consensus Process. 不明确的轨迹和不确定的益处:利用德尔菲共识过程创建危重或晚期患者临床不确定性词典。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-11-19 DOI: 10.1177/0272989X241293446
Samuel K McGowan, Maria-Jose Corrales-Martinez, Teva Brender, Alexander K Smith, Shannen Kim, Krista L Harrison, Hunter Mills, Albert Lee, David Bamman, Julien Cobert
{"title":"Unclear Trajectory and Uncertain Benefit: Creating a Lexicon for Clinical Uncertainty in Patients with Critical or Advanced Illness Using a Delphi Consensus Process.","authors":"Samuel K McGowan, Maria-Jose Corrales-Martinez, Teva Brender, Alexander K Smith, Shannen Kim, Krista L Harrison, Hunter Mills, Albert Lee, David Bamman, Julien Cobert","doi":"10.1177/0272989X241293446","DOIUrl":"https://doi.org/10.1177/0272989X241293446","url":null,"abstract":"<p><strong>Background: </strong>Clinical uncertainty is associated with increased resource utilization, worsened health-related quality of life for patients, and provider burnout, particularly during critical illness. Existing data are limited, because determining uncertainty from notes typically requires manual, qualitative review. We sought to develop a consensus list of descriptors of clinical uncertainty and then, using a thematic analysis approach, describe how respondents consider their use in intensive care unit (ICU) notes, such that future work can extract uncertainty data at scale.</p><p><strong>Design: </strong>We conducted a Delphi consensus study with physicians across multiple institutions nationally who care for critically ill patients or patients with advanced illnesses. Participants were given a definition for clinical uncertainty and collaborated through multiple rounds to determine which words represent uncertainty in clinician notes. We also administered surveys that included open-ended questions to participants about clinical uncertainty. Following derivation of a consensus list, we analyzed participant responses using thematic analysis to understand the role of uncertainty in clinical documentation.</p><p><strong>Results: </strong>Nineteen physicians participated in at least 2 of the Delphi rounds. Consensus was achieved for 44 words or phrases over 5 rounds of the Delphi process. Clinicians described comfort with using uncertainty terms and used them in a variety of ways: documenting and processing the diagnostic thinking process, enlisting help, identifying incomplete information, and practicing transparency to reflect uncertainty that was present.</p><p><strong>Conclusions: </strong>Using a consensus process, we created an uncertainty lexicon that can be used for uncertainty data extraction from the medical record. We demonstrate that physicians, particularly in the ICU, are comfortable with uncertainty and document uncertainty terms frequently to convey the complexity and ambiguity that is pervasive in critical illness.</p><p><strong>Highlights: </strong>Question: What words do physicians caring for critically ill patients use to document clinical uncertainty, and why?Findings: A consensus list of 44 words or phrases was identified by a group of experts. Physicians expressed comfort with using these words in the electronic health record.Meaning: Physicians are comfortable with uncertainty words and document them frequently to convey the complexity and ambiguity that is pervasive in critical illness.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X241293446"},"PeriodicalIF":3.1,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142669758","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Multi-indication Evidence Synthesis in Oncology Health Technology Assessment: Meta-analysis Methods and Their Application to a Case Study of Bevacizumab. 肿瘤健康技术评估中的多适应症证据综合:Meta 分析方法及其在贝伐珠单抗案例研究中的应用。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-11-18 DOI: 10.1177/0272989X241295665
Janharpreet Singh, Sumayya Anwer, Stephen Palmer, Pedro Saramago, Anne Thomas, Sofia Dias, Marta O Soares, Sylwia Bujkiewicz
{"title":"Multi-indication Evidence Synthesis in Oncology Health Technology Assessment: Meta-analysis Methods and Their Application to a Case Study of Bevacizumab.","authors":"Janharpreet Singh, Sumayya Anwer, Stephen Palmer, Pedro Saramago, Anne Thomas, Sofia Dias, Marta O Soares, Sylwia Bujkiewicz","doi":"10.1177/0272989X241295665","DOIUrl":"https://doi.org/10.1177/0272989X241295665","url":null,"abstract":"<p><strong>Background: </strong>Multi-indication cancer drugs receive licensing extensions to include additional indications, as trial evidence on treatment effectiveness accumulates. We investigate how sharing information across indications can strengthen the inferences supporting health technology assessment (HTA).</p><p><strong>Methods: </strong>We applied meta-analytic methods to randomized trial data on bevacizumab, to share information across oncology indications on the treatment effect on overall survival (OS) or progression-free survival (PFS) and on the surrogate relationship between effects on PFS and OS. Common or random indication-level parameters were used to facilitate information sharing, and the further flexibility of mixture models was also explored.</p><p><strong>Results: </strong>Treatment effects on OS lacked precision when pooling data available at present day within each indication separately, particularly for indications with few trials. There was no suggestion of heterogeneity across indications. Sharing information across indications provided more precise estimates of treatment effects and surrogacy parameters, with the strength of sharing depending on the model. When a surrogate relationship was used to predict treatment effects on OS, uncertainty was reduced only when sharing effects on PFS in addition to surrogacy parameters. Corresponding analyses using the earlier, sparser (within and across indications) evidence available for particular HTAs showed that sharing on both surrogacy and PFS effects did not notably reduce uncertainty in OS predictions. Little heterogeneity across indications meant limited added value of the mixture models.</p><p><strong>Conclusions: </strong>Meta-analysis methods can be usefully applied to share information on treatment effectiveness across indications in an HTA context, to increase the precision of target indication estimates. Sharing on surrogate relationships requires caution, as meaningful precision gains in predictions will likely require a substantial evidence base and clear support for surrogacy from other indications.</p><p><strong>Highlights: </strong>We investigated how sharing information across indications can strengthen inferences on the effectiveness of multi-indication treatments in the context of health technology assessment (HTA).Multi-indication meta-analysis methods can provide more precise estimates of an effect on a final outcome or of the parameters describing the relationship between effects on a surrogate endpoint and a final outcome.Precision of the predicted effect on the final outcome based on an effect on the surrogate endpoint will depend on the precision of the effect on the surrogate endpoint and the strength of evidence of a surrogate relationship across indications.Multi-indication meta-analysis methods can be usefully applied to predict an effect on the final outcome, particularly where there is limited evidence in the indication of interest.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X241295665"},"PeriodicalIF":3.1,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142649525","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Use of Adaptive Conjoint Analysis-Based Values Clarification in a Patient Decision Aid Is Not Associated with Better Perceived Values Clarity or Reduced Decisional Conflict but Enhances Values Congruence. 在患者决策辅助工具中使用基于自适应联合分析的价值观澄清与更好地感知价值观清晰度或减少决策冲突无关,但能增强价值观一致性。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-11-18 DOI: 10.1177/0272989X241298630
Nida Gizem Yılmaz, Arwen H Pieterse, Danielle R M Timmermans, Annemarie Becker, Birgit Witte-Lissenberg, Olga C Damman
{"title":"Use of Adaptive Conjoint Analysis-Based Values Clarification in a Patient Decision Aid Is Not Associated with Better Perceived Values Clarity or Reduced Decisional Conflict but Enhances Values Congruence.","authors":"Nida Gizem Yılmaz, Arwen H Pieterse, Danielle R M Timmermans, Annemarie Becker, Birgit Witte-Lissenberg, Olga C Damman","doi":"10.1177/0272989X241298630","DOIUrl":"https://doi.org/10.1177/0272989X241298630","url":null,"abstract":"<p><strong>Background: </strong>Evidence is lacking on the most effective values clarification methods (VCMs) in patient decision aids (PtDAs). We tested the effects of an adaptive conjoint analysis (ACA)-based VCM compared with a ranking-based VCM and no VCM on several decision-related outcomes, with the decisional conflict and its subscale \"perceived values clarity\" as primary outcomes.</p><p><strong>Design: </strong>Online experimental study with 3 conditions: no VCM versus ranking-based VCM versus <i>ACA</i>-based VCM (<i>N</i> = 282; <i>M<sub>age</sub></i> = 63.11 y, <i>s</i> = 12.12), with the latter 2 conditions including attributes important for a lung cancer treatment decision. We assessed 1) decisional conflict, 2) perceived values clarity (decisional conflict subscale), 3) perceived cognitive load, 4) anticipated regret, 5) ambivalence, 6) preparedness for decision making, 7) hypothetical treatment preference, and 8) values congruence (proxy). We performed analysis of covariance and linear regression. Age and level of deliberation were included as potential moderators, and we controlled for subjective numeracy (covariate). We exploratively tested the moderating effects of subjective numeracy and health literacy (without covariates).</p><p><strong>Results: </strong>We found no significant effect of type of VCM on overall decisional conflict or perceived values clarity. Age had a moderating effect: in younger participants, no VCM (v. ranking-based VCM) led to more values clarity, while in older participants, a ranking-based VCM (v. no VCM) led to more values clarity. Completing the ACA-based VCM, compared with no VCM, resulted in more values congruence.</p><p><strong>Limitations: </strong>The hypothetical choice situation might have induced lower levels of cognitive/affective involvement in the decision.</p><p><strong>Conclusions: </strong>This study found mixed effects of an ACA-based VCM. It did not decrease decisional conflict or increase perceived values clarity, yet it did improve values congruence.</p><p><strong>Implications: </strong>Completion of an ACA-based VCM in a PtDA may increase values congruence.</p><p><strong>Highlights: </strong>An adaptive conjoint analysis or a ranking-based values clarification method did not decrease analog patients' decisional conflict nor did it increase their perceived values clarity.In younger participants, no VCM (v. ranking-based VCM) led to more values clarity, while in older participants, a ranking-based VCM (v. no VCM) led to more values clarity.An adaptive conjoint analysis task for values clarification resulted in more values congruence.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X241298630"},"PeriodicalIF":3.1,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142649526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Sequential Calibration Approach to Address Challenges of Repeated Calibration of a COVID-19 Model. 采用顺序校准法应对 COVID-19 模型重复校准的挑战。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-11-15 DOI: 10.1177/0272989X241292012
Eva A Enns, Zongbo Li, Shannon B McKearnan, Szu-Yu Zoe Kao, Erinn C Sanstead, Alisha Baines Simon, Pamela J Mink, Stefan Gildemeister, Karen M Kuntz
{"title":"A Sequential Calibration Approach to Address Challenges of Repeated Calibration of a COVID-19 Model.","authors":"Eva A Enns, Zongbo Li, Shannon B McKearnan, Szu-Yu Zoe Kao, Erinn C Sanstead, Alisha Baines Simon, Pamela J Mink, Stefan Gildemeister, Karen M Kuntz","doi":"10.1177/0272989X241292012","DOIUrl":"10.1177/0272989X241292012","url":null,"abstract":"<p><strong>Background: </strong>Mathematical models served a critical role in COVID-19 decision making throughout the pandemic. Model calibration is an essential, but often computationally burdensome, step in model development that provides estimates for difficult-to-measure parameters and establishes an up-to-date modeling platform for scenario analysis. In the evolving COVID-19 pandemic, frequent recalibration was necessary to provide ongoing support to decision makers. In this study, we address the computational challenges of frequent recalibration with a new calibration approach.</p><p><strong>Methods: </strong>We calibrated and recalibrated an age-stratified dynamic compartmental model of COVID-19 in Minnesota to statewide COVID-19 cumulative mortality and prevalent age-specific hospitalizations from March 22, 2020 through August 20, 2021. This period was divided into 10 calibration periods, reflecting significant changes in policies, messaging, and/or epidemiological conditions in Minnesota. When recalibrating the model from one period to the next, we employed a sequential calibration approach that leveraged calibration results from previous periods and adjusted only parameters most relevant to the calibration target data of the new calibration period to improve computational efficiency. We compared computational burden and performance of the sequential calibration approach to a more traditional calibration method, in which all parameters were readjusted with each recalibration.</p><p><strong>Results: </strong>Both calibration methods identified parameter sets closely reproducing prevalent hospitalizations and cumulative deaths over time. By the last calibration period, both approaches converged to similar parameter values. However, the sequential calibration approach identified parameter sets that more tightly fit calibration targets and required substantially less computation time than traditional calibration.</p><p><strong>Conclusions: </strong>Sequential calibration is an efficient approach to maintaining up-to-date models with evolving, time-varying parameters and potentially identifies better-fitting parameter sets than traditional calibration.</p><p><strong>Highlights: </strong>This study used a sequential calibration approach, which takes advantage of previous calibration results to reduce the number of parameters to be estimated in each round of calibration, improving computational efficiency and algorithm convergence to best-fitting parameter values.Both sequential and traditional calibration approaches were able to identify parameter sets that closely reproduced calibration targets. However, the sequential calibration approach generated parameter sets that yielded tighter fits and was less computationally burdensome.Sequential calibration is an efficient approach to maintaining up-to-date models with evolving, time-varying parameters.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X241292012"},"PeriodicalIF":3.1,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142631439","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Longitudinal Study of the Association of Awareness of Disease Incurability with Patient-Reported Outcomes in Heart Failure. 心力衰竭患者对疾病不可治愈性的认识与患者报告结果之间关系的纵向研究。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-11-15 DOI: 10.1177/0272989X241297694
Jia Jia Lee, Chetna Malhotra, Kheng Leng David Sim, Khung Keong Yeo, Eric Finkelstein, Semra Ozdemir
{"title":"A Longitudinal Study of the Association of Awareness of Disease Incurability with Patient-Reported Outcomes in Heart Failure.","authors":"Jia Jia Lee, Chetna Malhotra, Kheng Leng David Sim, Khung Keong Yeo, Eric Finkelstein, Semra Ozdemir","doi":"10.1177/0272989X241297694","DOIUrl":"https://doi.org/10.1177/0272989X241297694","url":null,"abstract":"<p><strong>Objectives: </strong>To examine awareness of disease incurability among patients with heart failure over 24 mo and its associations with patient characteristics and patient-reported outcomes (distress, emotional, and spiritual well-being).</p><p><strong>Methods: </strong>This study analyzed 24-mo data from a prospective cohort study of 251 patients with heart failure (New York Heart Association class III/IV) recruited from inpatient wards in Singapore General Hospital and National Heart Centre Singapore. Patients were asked to report if their doctor told them they were receiving treatment to cure their condition. \"No\" responses were categorized as being aware of disease incurability, while \"Yes\" and \"Uncertain\" were categorized as being unaware and being uncertain about disease incurability, respectively. We used mixed-effects multinomial logistic regression to investigate the associations between awareness of disease incurability and patient characteristics and mixed-effects linear regressions to investigate associations with patient outcomes.</p><p><strong>Results: </strong>The percentage of patients who were aware of disease incurability increased from 51.6% at baseline to 76.4% at 24-mo follow-up (<i>P</i> < 0.001). Compared with being unaware of disease incurability, being aware was associated with older age (relative risk ratio [RRR] = 1.04; <i>P</i> = 0.005), adequate self-care confidence (RRR = 5.06; <i>P</i> < 0.001), participation in treatment decision making (RRR = 2.13; <i>P</i> = 0.006), higher education (RRR = 2.00; <i>P</i> = 0.033), financial difficulty (RRR = 1.18; <i>P</i> = 0.020), symptom burden (RRR = 1.08; <i>P</i> = 0.001), and ethnicity (<i>P</i> < 0.05). Compared with being unaware of disease incurability, being aware was associated with higher emotional well-being (β = 0.76; <i>P</i> = 0.024), while being uncertain about disease incurability was associated with poorer spiritual well-being (β = -3.16; <i>P</i> = 0.006).</p><p><strong>Conclusions: </strong>Our findings support the importance of being aware of disease incurability, addressing uncertainty around disease incurability among patients with heart failure, and helping patients make informed medical decisions. The findings are important to Asian and other cultures where the prognosis disclosure to terminally ill patients is generally low with an intention to \"protect\" patients.</p><p><strong>Highlights: </strong>Our 24-mo study with heart failure patients showed an increase from 52% to 76% in patients being aware of disease incurability.Compared with being unaware of disease incurability, being aware was associated with higher emotional well-being, while uncertainty about disease incurability was associated with poorer spiritual well-being.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X241297694"},"PeriodicalIF":3.1,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142640086","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Awareness of Disease Incurability Moderates the Association between Patients' Health Status and Their Treatment Preferences. 对疾病不可治愈性的认识可调节患者健康状况与治疗偏好之间的关系。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-11-09 DOI: 10.1177/0272989X241293716
Louisa Camille Poco, Ishwarya Balasubramanian, Isha Chaudhry, Chetna Malhotra
{"title":"Awareness of Disease Incurability Moderates the Association between Patients' Health Status and Their Treatment Preferences.","authors":"Louisa Camille Poco, Ishwarya Balasubramanian, Isha Chaudhry, Chetna Malhotra","doi":"10.1177/0272989X241293716","DOIUrl":"https://doi.org/10.1177/0272989X241293716","url":null,"abstract":"<p><strong>Background: </strong>With advancing illness, some patients with heart failure (HF) opt to receive life-extending treatments despite their high costs, while others choose to forgo these treatments, emphasizing cost containment. We examined the association between patients' health status and their preferences for treatment cost containment versus life extension and whether their patients' awareness of disease incurability moderated this association.</p><p><strong>Methods: </strong>In a prospective cohort of patients (<i>N</i> = 231) with advanced HF in Singapore, we assessed patients' awareness of disease incurability, health status, and treatment preferences every 4 mo for up to 4 y (up to 13 surveys). Using random effects multinomial logistic regression models, we assessed whether patients' awareness of disease incurability moderated the association between their health status and treatment preferences.</p><p><strong>Results: </strong>About half of the patients in our study lacked awareness of HF's incurability. Results from regression analyses showed that patients with better health status, as indicated by lower distress scores (odds ratio [OR] [95% confidence interval {CI}]: 0.862 [0.754, 0.985]) and greater physical well-being (1.12 [1.03, 1.21]); and who lacked awareness of their disease's incurability were more likely to prefer higher cost containment/minimal life extension treatments compared with lower cost containment/maximal life extension.</p><p><strong>Conclusions: </strong>This study underscores the significance of patients' awareness in disease incurability in shaping the relationship between their health status and treatment preferences. Our findings emphasize the need to incorporate illness education during goals-of-care conversations with patients and the importance of revisiting these conversations frequently to accommodate changing treatment preferences.</p><p><strong>Highlights: </strong>The health status of patients with advanced heart failure was associated with their treatment preferences.Patients whose health status improved and who lacked awareness of their disease's incurability were more likely to prefer higher cost containment/minimal life extension treatments.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X241293716"},"PeriodicalIF":3.1,"publicationDate":"2024-11-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142631360","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Assessing Methods for Adjusting Estimates of Treatment Effectiveness for Patient Nonadherence in the Context of Time-to-Event Outcomes and Health Technology Assessment: A Simulation Study. 在事件发生时间结果和健康技术评估的背景下,评估根据患者不依从性调整治疗效果估计值的方法:模拟研究。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-11-08 DOI: 10.1177/0272989X241293414
Abualbishr Alshreef, Nicholas Latimer, Paul Tappenden, Simon Dixon
{"title":"Assessing Methods for Adjusting Estimates of Treatment Effectiveness for Patient Nonadherence in the Context of Time-to-Event Outcomes and Health Technology Assessment: A Simulation Study.","authors":"Abualbishr Alshreef, Nicholas Latimer, Paul Tappenden, Simon Dixon","doi":"10.1177/0272989X241293414","DOIUrl":"https://doi.org/10.1177/0272989X241293414","url":null,"abstract":"<p><strong>Purpose: </strong>We aim to assess the performance of methods for adjusting estimates of treatment effectiveness for patient nonadherence in the context of health technology assessment using simulation methods.</p><p><strong>Methods: </strong>We simulated trial datasets with nonadherence, prognostic characteristics, and a time-to-event outcome. The simulated scenarios were based on a trial investigating immunosuppressive treatments for improving graft survival in patients who had had a kidney transplant. The primary estimand was the difference in restricted mean survival times in all patients had there been no nonadherence. We compared generalized methods (g-methods; marginal structural model with inverse probability of censoring weighting [IPCW], structural nested failure time model [SNFTM] with g-estimation) and simple methods (intention-to-treat [ITT] analysis, per-protocol [PP] analysis) in 90 scenarios each with 1,900 simulations. The methods' performance was primarily assessed according to bias.</p><p><strong>Results: </strong>In implementation nonadherence scenarios, the average percentage bias was 20% (ranging from 7% to 37%) for IPCW, 20% (8%-38%) for SNFTM, 20% (8%-38%) for PP, and 40% (20%-75%) for ITT. In persistence nonadherence scenarios, the average percentage bias was 26% (9%-36%) for IPCW, 26% (14%-39%) for SNFTM, 26% (14%-36%) for PP, and 47% (16%-72%) for ITT. In initiation nonadherence scenarios, the percentage bias ranged from -29% to 110% for IPCW, -34% to 108% for SNFTM, -32% to 102% for PP, and between -18% and 200% for ITT.</p><p><strong>Conclusion: </strong>In this study, g-methods and PP produced more accurate estimates of the treatment effect adjusted for nonadherence than the ITT analysis did. However, considerable bias remained in some scenarios.</p><p><strong>Highlights: </strong>Randomized controlled trials are usually analyzed using the intention-to-treat (ITT) principle, which produces a valid estimate of effectiveness relating to the underlying trial, but when patient adherence to medications in the real world is known to differ from that observed in the trial, such estimates are likely to result in a biased representation of real-world effectiveness and cost-effectiveness.Our simulation study demonstrates that generalized methods (g-methods; IPCW, SNFTM) and per-protocol analysis provide more accurate estimates of the treatment effect than the ITT analysis does, when adjustment for nonadherence is required; however, even with these adjustment methods, considerable bias may remain in some scenarios.When real-world adherence is expected to differ from adherence observed in a trial, adjustment methods should be used to provide estimates of real-world effectiveness.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X241293414"},"PeriodicalIF":3.1,"publicationDate":"2024-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142631358","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
International Systematic Review of Utility Values Associated with Cardiovascular Disease and Reflections on Selecting Evidence for a UK Decision-Analytic Model. 与心血管疾病相关的效用值的国际系统性回顾以及为英国决策分析模型选择证据的思考。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-02-01 Epub Date: 2024-01-04 DOI: 10.1177/0272989X231214782
Rob Hainsworth, Alexander J Thompson, Bruce Guthrie, Katherine Payne, Gabriel Rogers
{"title":"International Systematic Review of Utility Values Associated with Cardiovascular Disease and Reflections on Selecting Evidence for a UK Decision-Analytic Model.","authors":"Rob Hainsworth, Alexander J Thompson, Bruce Guthrie, Katherine Payne, Gabriel Rogers","doi":"10.1177/0272989X231214782","DOIUrl":"10.1177/0272989X231214782","url":null,"abstract":"<p><strong>Purpose: </strong>Evaluating interventions for cardiovascular disease (CVD) requires estimates of its effect on utility. We aimed to 1) systematically review utility estimates for CVDs published since 2013 and 2) critically appraise UK-relevant estimates and calculate corresponding baseline utility multipliers.</p><p><strong>Methods: </strong>We searched MEDLINE and Embase (April 22, 2021) using CVD and utility terms. We screened results for primary studies reporting utility distributions for people with experience of heart failure, myocardial infarction, peripheral arterial disease, stable angina, stroke, transient ischemic attack, or unstable angina. We extracted characteristics from studies included. For UK estimates based on the EuroQoL 5-dimension (EQ-5D) measure, we assessed risk of bias and applicability to a decision-analytic model, pooled arms/time points as appropriate, and estimated baseline utility multipliers using predicted utility for age- and sex- matched populations without CVD. We sought utility sources from directly applicable studies with low risk of bias, prioritizing plausibility of severity ordering in our base-case model and highest population ascertainment in a sensitivity analysis.</p><p><strong>Results: </strong>Most of the 403 studies identified used EQ-5D (<i>n</i> = 217) and most assessed Organisation for Economic Co-operation and Development populations (<i>n</i> = 262), although measures and countries varied widely. UK studies using EQ-5D (<i>n</i> = 29) produced very heterogeneous baseline utility multipliers for each type of CVD, precluding meta-analysis and implying different possible severity orderings. We could find sources that provided a plausible ordering of utilities while adequately representing health states.</p><p><strong>Conclusions: </strong>We cataloged international CVD utility estimates and calculated UK-relevant baseline utility multipliers. Modelers should consider unreported sources of heterogeneity, such as population differences, when selecting utility evidence from reviews.</p><p><strong>Highlights: </strong>Published systematic reviews have summarized estimates of utility associated with cardiovascular disease published up to 2013.We 1) reviewed utility estimates for 7 types of cardiovascular disease published since 2013, 2) critically appraised UK-relevant studies, and 3) estimated the effect of each cardiovascular disease on baseline utility.Our review 1) recommends a consistent and reliable set of baseline utility multipliers for 7 types of cardiovascular disease and 2) provides systematically identified reference information for researchers seeking utility evidence for their own context.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"217-234"},"PeriodicalIF":3.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10865747/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139089195","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Overestimation of Survival Rates of Cardiopulmonary Resuscitation Is Associated with Higher Preferences to Be Resuscitated: Evidence from a National Survey of Older Adults in Switzerland. 高估心肺复苏的存活率与较高的复苏意愿有关:来自瑞士老年人全国调查的证据。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-02-01 Epub Date: 2023-12-29 DOI: 10.1177/0272989X231218691
Clément Meier, Sarah Vilpert, Maud Wieczorek, Gian Domenico Borasio, Ralf J Jox, Jürgen Maurer
{"title":"Overestimation of Survival Rates of Cardiopulmonary Resuscitation Is Associated with Higher Preferences to Be Resuscitated: Evidence from a National Survey of Older Adults in Switzerland.","authors":"Clément Meier, Sarah Vilpert, Maud Wieczorek, Gian Domenico Borasio, Ralf J Jox, Jürgen Maurer","doi":"10.1177/0272989X231218691","DOIUrl":"10.1177/0272989X231218691","url":null,"abstract":"<p><strong>Background: </strong>Many widely used advance directives templates include direct questions on individuals' preferences for cardiopulmonary resuscitation (CPR) in case of decision-making incapacity during medical emergencies. However, as knowledge of the survival rates of CPR is often limited, individuals' advance decisions on CPR may be poorly aligned with their preferences if false beliefs about the survival rates of CPR shape stated preferences for CPR.</p><p><strong>Methods: </strong>We analyzed nationally representative data from 1,469 adults aged 58+ y who responded to wave 8 (2019/2020) of the Swiss version of the Survey on Health, Ageing, and Retirement in Europe (SHARE) to assess the partial association between knowledge of CPR survival rates and stated preferences for CPR using multivariable probit regression models that adjust for social, health, and regional characteristics. Knowledge of CPR survival rates was assessed by asking how likely it is in general in Switzerland for a 70-y-old to survive until hospital discharge from a CPR performed outside of a hospital. Preferences for CPR were measured by asking respondents if they would wish to be resuscitated in case of cardiac arrest.</p><p><strong>Results: </strong>Only 9.3% of respondents correctly assessed the chances for a 70-y-old to survive until hospital discharge from a CPR performed outside of a hospital, while 65.2% indicated a preference to be resuscitated in case of a cardiac arrest. Respondents who correctly assessed CPR survival were significantly more likely to wish not to be resuscitated (average marginal effect: 0.18, <i>P</i> < 0.001).</p><p><strong>Conclusions: </strong>Reducing misconceptions concerning the survival rates of CPR could change older adults' preferences for CPR and make them more likely to forgo such treatments.</p><p><strong>Highlights: </strong>Many older adults in Switzerland overestimate the survival rates of cardiopulmonary resuscitation (CPR).The study reveals that individuals with accurate knowledge of CPR survival rates are more likely to refuse resuscitation in case of cardiac arrest.Overestimation of CPR survival rates may lead to a mismatch between individuals' preferences for CPR and their actual end-of-life care decisions.Improving the general population's knowledge of CPR survival rates is crucial to ensure informed decision making and effective advance care planning.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"129-134"},"PeriodicalIF":3.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10865767/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139075731","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparing Discrete Choice Experiment with Swing Weighting to Estimate Attribute Relative Importance: A Case Study in Lung Cancer Patient Preferences. 比较离散选择实验与摇摆加权法估算属性相对重要性:肺癌患者偏好案例研究》。
IF 3.1 3区 医学
Medical Decision Making Pub Date : 2024-02-01 Epub Date: 2024-01-04 DOI: 10.1177/0272989X231222421
J Veldwijk, I P Smith, S Oliveri, S Petrocchi, M Y Smith, L Lanzoni, R Janssens, I Huys, G A de Wit, C G M Groothuis-Oudshoorn
{"title":"Comparing Discrete Choice Experiment with Swing Weighting to Estimate Attribute Relative Importance: A Case Study in Lung Cancer Patient Preferences.","authors":"J Veldwijk, I P Smith, S Oliveri, S Petrocchi, M Y Smith, L Lanzoni, R Janssens, I Huys, G A de Wit, C G M Groothuis-Oudshoorn","doi":"10.1177/0272989X231222421","DOIUrl":"10.1177/0272989X231222421","url":null,"abstract":"<p><strong>Introduction: </strong>Discrete choice experiments (DCE) are commonly used to elicit patient preferences and to determine the relative importance of attributes but can be complex and costly to administer. Simpler methods that measure relative importance exist, such as swing weighting with direct rating (SW-DR), but there is little empirical evidence comparing the two. This study aimed to directly compare attribute relative importance rankings and weights elicited using a DCE and SW-DR.</p><p><strong>Methods: </strong>A total of 307 patients with non-small-cell lung cancer in Italy and Belgium completed an online survey assessing preferences for cancer treatment using DCE and SW-DR. The relative importance of the attributes was determined using a random parameter logit model for the DCE and rank order centroid method (ROC) for SW-DR. Differences in relative importance ranking and weights between the methods were assessed using Cohen's weighted kappa and Dirichlet regression. Feedback on ease of understanding and answering the 2 tasks was also collected.</p><p><strong>Results: </strong>Most respondents (>65%) found both tasks (very) easy to understand and answer. The same attribute, survival, was ranked most important irrespective of the methods applied. The overall ranking of the attributes on an aggregate level differed significantly between DCE and SW-ROC (<i>P</i> < 0.01). Greater differences in attribute weights between attributes were reported in DCE compared with SW-DR (<i>P</i> < 0.01). Agreement between the individual-level attribute ranking across methods was moderate (weighted Kappa 0.53-0.55).</p><p><strong>Conclusion: </strong>Significant differences in attribute importance between DCE and SW-DR were found. Respondents reported both methods being relatively easy to understand and answer. Further studies confirming these findings are warranted. Such studies will help to provide accurate guidance for methods selection when studying relative attribute importance across a wide array of preference-relevant decisions.</p><p><strong>Highlights: </strong>Both DCEs and SW tasks can be used to determine attribute relative importance rankings and weights; however, little evidence exists empirically comparing these methods in terms of outcomes or respondent usability.Most respondents found the DCE and SW tasks very easy or easy to understand and answer.A direct comparison of DCE and SW found significant differences in attribute importance rankings and weights as well as a greater spread in the DCE-derived attribute relative importance weights.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"203-216"},"PeriodicalIF":3.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10865764/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139099092","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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