Oversampling and replacement strategies in propensity score matching: a critical review focused on small sample size in clinical settings.

Monte Carlo simulations Oversampling Propensity score matching Replacement Small samples

Journal

BMC medical research methodology
ISSN: 1471-2288
Titre abrégé: BMC Med Res Methodol
Pays: England
ID NLM: 100968545

Informations de publication

Date de publication:
22 11 2021
Historique:
received: 14 05 2021
accepted: 26 10 2021
entrez: 23 11 2021
pubmed: 24 11 2021
medline: 27 1 2022
Statut: epublish

Résumé

Propensity score matching is a statistical method that is often used to make inferences on the treatment effects in observational studies. In recent years, there has been widespread use of the technique in the cardiothoracic surgery literature to evaluate to potential benefits of new surgical therapies or procedures. However, the small sample size and the strong dependence of the treatment assignment on the baseline covariates that often characterize these studies make such an evaluation challenging from a statistical point of view. In such settings, the use of propensity score matching in combination with oversampling and replacement may provide a solution to these issues by increasing the initial sample size of the study and thus improving the statistical power that is needed to detect the effect of interest. In this study, we review the use of propensity score matching in combination with oversampling and replacement in small sample size settings. We performed a series of Monte Carlo simulations to evaluate how the sample size, the proportion of treated, and the assignment mechanism affect the performances of the proposed approaches. We assessed the performances with overall balance, relative bias, root mean squared error and nominal coverage. Moreover, we illustrate the methods using a real case study from the cardiac surgery literature. Matching without replacement produced estimates with lower bias and better nominal coverage than matching with replacement when 1:1 matching was considered. In contrast to that, matching with replacement showed better balance, relative bias, and root mean squared error than matching without replacement for increasing levels of oversampling. The best nominal coverage was obtained by using the estimator that accounts for uncertainty in the matching procedure on sets of units obtained after matching with replacement. The use of replacement provides the most reliable treatment effect estimates and that no more than 1 or 2 units from the control group should be matched to each treated observation. Moreover, the variance estimator that accounts for the uncertainty in the matching procedure should be used to estimate the treatment effect.

Sections du résumé

BACKGROUND
Propensity score matching is a statistical method that is often used to make inferences on the treatment effects in observational studies. In recent years, there has been widespread use of the technique in the cardiothoracic surgery literature to evaluate to potential benefits of new surgical therapies or procedures. However, the small sample size and the strong dependence of the treatment assignment on the baseline covariates that often characterize these studies make such an evaluation challenging from a statistical point of view. In such settings, the use of propensity score matching in combination with oversampling and replacement may provide a solution to these issues by increasing the initial sample size of the study and thus improving the statistical power that is needed to detect the effect of interest. In this study, we review the use of propensity score matching in combination with oversampling and replacement in small sample size settings.
METHODS
We performed a series of Monte Carlo simulations to evaluate how the sample size, the proportion of treated, and the assignment mechanism affect the performances of the proposed approaches. We assessed the performances with overall balance, relative bias, root mean squared error and nominal coverage. Moreover, we illustrate the methods using a real case study from the cardiac surgery literature.
RESULTS
Matching without replacement produced estimates with lower bias and better nominal coverage than matching with replacement when 1:1 matching was considered. In contrast to that, matching with replacement showed better balance, relative bias, and root mean squared error than matching without replacement for increasing levels of oversampling. The best nominal coverage was obtained by using the estimator that accounts for uncertainty in the matching procedure on sets of units obtained after matching with replacement.
CONCLUSIONS
The use of replacement provides the most reliable treatment effect estimates and that no more than 1 or 2 units from the control group should be matched to each treated observation. Moreover, the variance estimator that accounts for the uncertainty in the matching procedure should be used to estimate the treatment effect.

Identifiants

pubmed: 34809559
doi: 10.1186/s12874-021-01454-z
pii: 10.1186/s12874-021-01454-z
pmc: PMC8609749
doi:

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

256

Informations de copyright

© 2021. The Author(s).

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Auteurs

Daniele Bottigliengo (D)

Unit of Biostatistics, Epidemiology and Public Health, Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padova, Via Loredan 18, 35121, Padova, Italy.

Ileana Baldi (I)

Unit of Biostatistics, Epidemiology and Public Health, Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padova, Via Loredan 18, 35121, Padova, Italy.

Corrado Lanera (C)

Unit of Biostatistics, Epidemiology and Public Health, Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padova, Via Loredan 18, 35121, Padova, Italy.

Giulia Lorenzoni (G)

Unit of Biostatistics, Epidemiology and Public Health, Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padova, Via Loredan 18, 35121, Padova, Italy.

Jonida Bejko (J)

Department of Cardiac, Thoracic,Vascular Sciences and Public Health, University of Padova, Padova, Italy.

Tomaso Bottio (T)

Department of Cardiac, Thoracic,Vascular Sciences and Public Health, University of Padova, Padova, Italy.

Vincenzo Tarzia (V)

Department of Cardiac, Thoracic,Vascular Sciences and Public Health, University of Padova, Padova, Italy.

Massimiliano Carrozzini (M)

Department of Cardiac, Thoracic,Vascular Sciences and Public Health, University of Padova, Padova, Italy.

Gino Gerosa (G)

Department of Cardiac, Thoracic,Vascular Sciences and Public Health, University of Padova, Padova, Italy.

Paola Berchialla (P)

Department of Clinical and Biological Sciences, University of Torino, Torino, Italy.

Dario Gregori (D)

Unit of Biostatistics, Epidemiology and Public Health, Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padova, Via Loredan 18, 35121, Padova, Italy. dario.gregori@unipd.it.

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