A comparison of propensity score methods: a case‐study estimating the effectiveness of post‐AMI statin use

Austin, Peter C.; Mamdani, Muhammad

doi:10.1002/sim.2328

Cited by 491 publications

(398 citation statements)

References 31 publications

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“…The method then selected the nearest untreated subject for matching with the treated subject within a fixed caliper width of 0.02 [14]. To assess the success of the matching procedure, we measured standardized differences (measured in percentage points) in observed confounders between the matched groups [15].We estimated the logistic regression model using generalized estimating equation (GEE) methods to incorporate the matched-pairs design [16].…”

Section: Discussionmentioning

confidence: 99%

Thrombolytic therapy and outcome of patients with an acute symptomatic pulmonary embolism

Riera-Mestre

Jiménez

Muriel

et al. 2012

Journal of Thrombosis and Haemostasis

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Summary. Background: While the primary therapy for most patients with a pulmonary embolism (PE) consists of anticoagulation, the efficacy of thrombolysis relative to standard therapy remains unclear. Methods: In this retrospective cohort study of 15 944 patients with an objectively confirmed symptomatic acute PE, identified from the multicenter, international, prospective, Registro Informatizado de la Enfermedad TromboEmbo´lica (RIETE registry), we aimed to assess the association between thrombolytic therapy and all-cause mortality during the first 3 months after the diagnosis of a PE. After creating two subgroups, stratified by systolic blood pressure (SBP) (< 100 mm Hg vs. other), we used propensity scorematching for a comparison of patients who received thrombolysis to those who did not in each subgroup. Results: Patients who received thrombolysis were younger, had fewer comorbid diseases and more signs of clinical severity compared with those who did not receive it. In the subgroup with systolic hypotension, analysis of propensity score-matched pairs (n = 94 pairs) showed a non-statistically significant but clinically relevant lower risk of death for thrombolysis compared with no thrombolysis (odds ratio [OR] 0.72; 95% confidence interval [CI], 0.36-1.46; P = 0.37). In the normotensive subgroup, analysis of propensity score-matched pairs (n = 217 pairs) showed a statistically significant and clinically meaningful increased risk of death for thrombolysis compared with no thrombolysis (OR 2.32; 95% CI, 1.15-4.68; P = 0.018). When we imputed data for missing values for echocardiography and troponin tests in the group of normotensive patients, we no longer detected the increased risk of death associated with thrombolytic therapy. Conclusions: In normotensive patients with acute symptomatic PE, thrombolytic therapy is associated with a higher risk of death than no thrombolytic therapy. In hemodynamically unstable patients, thrombolytic therapy is possibly associated with a lower risk of death than no thrombolytic therapy. However, study design limitations do not imply a causal relationship between thrombolytics and outcome.

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Section: Discussionmentioning

confidence: 99%

Thrombolytic therapy and outcome of patients with an acute symptomatic pulmonary embolism

Riera-Mestre

Jiménez

Muriel

et al. 2012

Journal of Thrombosis and Haemostasis

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“…Common diagnostics include t tests of the covariates, Kolmogorov-Smirnov tests, and other comparisons of distributions (e.g., Austin & Mamdani, 2006). Ho et al (2007) provide a summary of numerical and graphical summaries of balance, including empirical quantile-quantile plots to examine the empirical distribution of each covariate in the matched samples.…”

Section: Diagnostics For Matching Methodsmentioning

confidence: 99%

Best Practices in Quasi–Experimental Designs: Matching Methods for Causal Inference

Stuart¹,

Rubin²

2008

Best Practices in Quantitative Methods

244

200

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“…[57][58][59][60][61] These studies have shown matching and IPTW to be more effective than stratification or covariate adjustment. [58][59][60][61] The principal advantage of propensity scoring over other adjustment methods such as regression analysis is that it can be used even with small sample sizes and therefore may be particularly relevant to regenerative medicine. Propensity scoring also has a number of disadvantages.…”

Section: Propensity Scoringmentioning

confidence: 99%

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Hettle

Corbett

Hinde

et al. 2017

Health Technol Assess

101

142

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BackgroundThe National Institute for Health and Care Excellence (NICE) commissioned a ‘mock technology appraisal’ to assess whether changes to its methods and processes are needed. This report presents the findings of independent research commissioned to inform this appraisal and the deliberations of a panel convened by NICE to evaluate the mock appraisal.MethodsOur research included reviews to identify issues, analysis methods and conceptual differences and the relevance of alternative decision frameworks, alongside the development of an exemplar case study of chimeric antigen receptor (CAR) T-cell therapy for treating acute lymphoblastic leukaemia.ResultsAn assessment of previous evaluations of regenerative medicines found that, although there were a number of evidential challenges, none was unique to regenerative medicines or was beyond the scope of existing methods used to conceptualise decision uncertainty. Regarding the clinical evidence for regenerative medicines, the issues were those associated with a limited evidence base but were not unique to regenerative medicines: small non-randomised studies, high variation in response and the intervention subject to continuing development. The relative treatment effects generated from single-arm trials are likely to be optimistic unless it is certain that the historical data have accurately estimated the efficacy of the control agent. Pivotal trials may use surrogate end points, which, on average, overestimate treatment effects. To reduce overall uncertainty, multivariate meta-analysis of all available data should be considered. Incorporating indirectly relevant but more reliable (more mature) data into the analysis can also be considered; such data may become available as a result of the evolving regulatory pathways being developed by the European Medicines Agency. For the exemplar case of CAR T-cell therapy, target product profiles (TPPs) were developed, which considered the ‘curative’ and ‘bridging to stem-cell transplantation’ treatment approaches separately. Within each TPP, three ‘hypothetical’ evidence sets (minimum, intermediate and mature) were generated to simulate the impact of alternative levels of precision and maturity in the clinical evidence. Subsequent assessments of cost-effectiveness were undertaken, employing the existing NICE reference case alongside additional analyses suggested within alternative frameworks. The additional exploratory analyses were undertaken to demonstrate how assessments of cost-effectiveness and uncertainty could be impacted by alternative managed entry agreements (MEAs), including price discounts, performance-related schemes and technology leasing. The panel deliberated on the range of TPPs, evidence sets and MEAs, commenting on the likely recommendations for each scenario. The panel discussed the challenges associated with the exemplar and regenerative medicines more broadly, focusing on the need for a robust quantification of the level of uncertainty in the cost-effective estimates and the potential value of MEAs in limiting the exposure of the NHS to high upfront costs and loss associated with a wrong decision.ConclusionsIt is to be expected that there will be a significant level of uncertainty in determining the clinical effectiveness of regenerative medicines and their long-term costs and benefits, but the existing methods available to estimate the implications of this uncertainty are sufficient. The use of risk sharing and MEAs between the NHS and manufacturers of regenerative medicines should be investigated further.FundingThe National Institute for Health Research Health Technology Assessment programme.

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A comparison of propensity score methods: a case‐study estimating the effectiveness of post‐AMI statin use

Cited by 491 publications

References 31 publications

Thrombolytic therapy and outcome of patients with an acute symptomatic pulmonary embolism

Thrombolytic therapy and outcome of patients with an acute symptomatic pulmonary embolism

Best Practices in Quasi–Experimental Designs: Matching Methods for Causal Inference

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

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