Estimating the quality of optimal treatment regimes.

When multiple treatment alternatives are available for a disease, an obvious question is which alternative is most effective for which patient. One may address this question by searching for optimal treatment regimes that specify for each individual the preferable treatment alternative based on that...

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Publicado en:Statistics in Medicine Vol. 38; no. 25; pp. 4925 - 4939
Autores principales: Sies, Aniek, Van Mechelen, Iven
Formato: research Journal Article
Publicado: Wiley-Blackwell 11/10/2019
Acceso en línea:Ver este registro en EBSCOhost
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      dt: 11/10/2019
      vid: 38
      iid: 25
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      pub: Wiley-Blackwell
      place: Malden, Massachusetts
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        139135391
        10.1002/sim.8342
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        atl: Estimating the quality of optimal treatment regimes.
      aug:
        au:
          Sies, Aniek
          Van Mechelen, Iven
        affil: Faculty of Psychology and Educational Sciences, KU Leuven, Leuven Belgium
      sug:
        subj:
          Models, Statistical
          Therapeutics Statistics and Numerical Data
          Decision Making
          Depression Drug Therapy
          Study Design
          Computer Simulation
          Antidepressive Agents Administration and Dosage
          Drug Therapy, Combination
          Human
          Clinical Trials
          Validation Studies
          Comparative Studies
          Evaluation Research
          Multicenter Studies
          Ferrans and Powers Quality of Life Index
      ab: When multiple treatment alternatives are available for a disease, an obvious question is which alternative is most effective for which patient. One may address this question by searching for optimal treatment regimes that specify for each individual the preferable treatment alternative based on that individual's baseline characteristics. When such a regime has been estimated, its quality (in terms of the expected outcome if it was used for treatment assignment of all patients in the population under study) is of obvious interest. Obtaining a good and reliable estimate of this quantity is a key challenge for which so far no satisfactory solution is available. In this paper, we consider for this purpose several estimators of the expected outcome in conjunction with several resampling methods. The latter have been evaluated before within the context of statistical learning to estimate the prediction error of estimated prediction rules. Yet, the results of these evaluations were equivocal, with different best performing methods in different studies, and with near-zero and even negative correlations between true and estimated prediction errors. Moreover, for different reasons, it is not straightforward to extrapolate the findings of these studies to the context of optimal treatment regimes. To address these issues, we set up a new and comprehensive simulation study. In this study, combinations of different estimators with .632+ and out-of-bag bootstrap resampling methods performed best. In addition, the study shed a surprising new light on the previously reported problematic correlations between true and estimated prediction errors in the area of statistical learning.
      pubtype: Academic Journal
      doctype:
        research
        Journal Article
      ougenre: Article
    language: English
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