STEPS: an efficient prospective likelihood approach to genetic association analyses of secondary traits in extreme phenotype sequencing.

It has been well acknowledged that methods for secondary trait (ST) association analyses under a case-control design (ST$_{\text{CC}}$) should carefully consider the sampling process to avoid biased risk estimates. A similar situation also exists in the extreme phenotype sequencing (EPS) designs, wh...

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Publicado en:Biostatistics Vol. 21; no. 1; pp. 33 - 50
Autores principales: Bi, Wenjian, Li, Yun, Smeltzer, Matthew P, Gao, Guimin, Zhao, Shengli, Kang, Guolian
Formato: research Journal Article
Publicado: Oxford University Press / USA Jan2020
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Jan2020
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      pub: Oxford University Press / USA
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        10.1093/biostatistics/kxy030
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        atl: STEPS: an efficient prospective likelihood approach to genetic association analyses of secondary traits in extreme phenotype sequencing.
      aug:
        au:
          Bi, Wenjian
          Li, Yun
          Smeltzer, Matthew P
          Gao, Guimin
          Zhao, Shengli
          Kang, Guolian
        affil: Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA
      sug:
        subj:
          Models, Theoretical
          Genetic Techniques Methods
          Human
          Computer Simulation
          Probability
          Phenotype
          Validation Studies
          Comparative Studies
          Evaluation Research
          Multicenter Studies
          Funding Source
      ab: It has been well acknowledged that methods for secondary trait (ST) association analyses under a case-control design (ST$_{\text{CC}}$) should carefully consider the sampling process to avoid biased risk estimates. A similar situation also exists in the extreme phenotype sequencing (EPS) designs, which is to select subjects with extreme values of continuous primary phenotype for sequencing. EPS designs are commonly used in modern epidemiological and clinical studies such as the well-known National Heart, Lung, and Blood Institute Exome Sequencing Project. Although naïve generalized regression or ST$_{\text{CC}}$ method could be applied, their validity is questionable due to difference in statistical designs. Herein, we propose a general prospective likelihood framework to perform association testing for binary and continuous STs under EPS designs (STEPS), which can also incorporate covariates and interaction terms. We provide a computationally efficient and robust algorithm to obtain the maximum likelihood estimates. We also present two empirical mathematical formulas for power/sample size calculations to facilitate planning of binary/continuous STs association analyses under EPS designs. Extensive simulations and application to a genome-wide association study of benign ethnic neutropenia under an EPS design demonstrate the superiority of STEPS over all its alternatives above.
      pubtype: Academic Journal
      doctype:
        research
        Journal Article
      ougenre: Article
    language: English
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