Modeling strategies to improve parameter estimates in prognostic factors analyses with patient-reported outcomes in oncology.

Purpose: The inclusion of patient-reported outcome (PRO) questionnaires in prognostic factor analyses in oncology has substantially increased in recent years. We performed a simulation study to compare the performances of four different modeling strategies in estimating the prognostic impact of mult...

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Published in:Quality of Life Research Vol. 28; no. 5; pp. 1315 - 1326
Main Authors: Cottone, Francesco, Deliu, Nina, Collins, Gary S., Anota, Amelie, Bonnetain, Franck, Van Steen, Kristel, Cella, David, Efficace, Fabio
Format: Journal Article
Published: Springer Nature May2019
Online Access:View this record in EBSCOhost
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      dt: May2019
      vid: 28
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      pub: Springer Nature
      place: New York, New York
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        10.1007/s11136-018-02097-2
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        atl: Modeling strategies to improve parameter estimates in prognostic factors analyses with patient-reported outcomes in oncology.
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          Cottone, Francesco
          Deliu, Nina
          Collins, Gary S.
          Anota, Amelie
          Bonnetain, Franck
          Van Steen, Kristel
          Cella, David
          Efficace, Fabio
        affil: Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy
      sug:
        subj:
          Quality of Life Psychosocial Factors
          Neoplasms Psychosocial Factors
          Neoplasms Therapy
          Young Adult
          Middle Age
          Aged, 80 and Over
          Male
          Adult
          Factor Analysis
          Prognosis
          Cox Proportional Hazards Model
          Female
          Aged
          Sample Size
          Impact of Events Scale
          Middle Aged: 45-64 years
          Aged, 80 & over
          Adult: 19-44 years
          Aged: 65+ years
          Male
          Female
      ab: Purpose: The inclusion of patient-reported outcome (PRO) questionnaires in prognostic factor analyses in oncology has substantially increased in recent years. We performed a simulation study to compare the performances of four different modeling strategies in estimating the prognostic impact of multiple collinear scales from PRO questionnaires.Methods: We generated multiple scenarios describing survival data with different sample sizes, event rates and degrees of multicollinearity among five PRO scales. We used the Cox proportional hazards (PH) model to estimate the hazard ratios (HR) using automatic selection procedures, which were based on either the likelihood ratio-test (Cox-PV) or the Akaike Information Criterion (Cox-AIC). We also used Cox PH models which included all variables and were either penalized using the Ridge regression (Cox-R) or were estimated as usual (Cox-Full). For each scenario, we simulated 1000 independent datasets and compared the average outcomes of all methods.Results: The Cox-R showed similar or better performances with respect to the other methods, particularly in scenarios with medium-high multicollinearity (ρ = 0.4 to ρ = 0.8) and small sample sizes (n = 100). Overall, the Cox-PV and Cox-AIC performed worse, for example they did not select one or more prognostic collinear PRO scales in some scenarios. Compared with the Cox-Full, the Cox-R provided HR estimates with similar bias patterns but smaller root-mean-squared errors, particularly in higher multicollinearity scenarios.Conclusions: Our findings suggest that the Cox-R is the best approach when performing prognostic factor analyses with multiple and collinear PRO scales, particularly in situations of high multicollinearity, small sample sizes and low event rates.
      pubtype: Academic Journal
      doctype: Journal Article
      ougenre: Article
    language: English
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