Metabolic profiles and prediction of failure to thrive of citrin deficiency with normal liver function based on metabolomics and machine learning.

Purpose: This study aimed to explore metabolite pathways and identify residual metabolites during the post-neonatal intrahepatic cholestasis caused by citrin deficiency (post-NICCD) phase, while developing a predictive model for failure to thrive (FTT) using selected metabolites. Method: A case-cont...

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Publicado en:Nutrition & Metabolism Vol. 22; no. 1; pp. 1 - 13
Autores principales: Wang, Peiyao, Zhou, Duo, Hu, Lingwei, Ge, Pingping, Cen, Ziyan, Hu, Zhenzhen, He, Qimin, Zhou, Kejun, Wu, Benqing, Huang, Xinwen
Formato: research tables/charts Journal Article
Publicado: BioMed Central 5/12/2025
Acceso en línea:Ver este registro en EBSCOhost
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      dt: 5/12/2025
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      pub: BioMed Central
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        10.1186/s12986-025-00928-x
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        atl: Metabolic profiles and prediction of failure to thrive of citrin deficiency with normal liver function based on metabolomics and machine learning.
      aug:
        au:
          Wang, Peiyao
          Zhou, Duo
          Hu, Lingwei
          Ge, Pingping
          Cen, Ziyan
          Hu, Zhenzhen
          He, Qimin
          Zhou, Kejun
          Wu, Benqing
          Huang, Xinwen
        affil: https://ror.org/025fyfd20 Department of Genetics and Metabolism, Children's Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, No. 3333 Binsheng Road, Binjiang District, 310052, Hangzhou City, Zhejiang Province, China
      sug:
        subj:
          Failure to Thrive Diagnosis
          Metabolomics
          Machine Learning
          Mutation
          Liver Physiology
          Prediction Models
          Cholestasis, Intrahepatic Diagnosis
          Postnatal Period
          Cholestasis, Intrahepatic Therapy
          Treatment Outcomes
          Human
          Case Control Studies
          Male
          Female
          Child, Preschool
          Child
          Random Forest
          Regression
          Biological Markers Blood
          ROC Curve
          Chromatography, Liquid
          Mass Spectrometry
          Amino Acids Blood
          Arginine Blood
          Alanine Blood
          Aspartic Acid Blood
          Descriptive Statistics
          Funding Source
          Child, Preschool: 2-5 years
          Child: 6-12 years
          Male
          Female
      ab: Purpose: This study aimed to explore metabolite pathways and identify residual metabolites during the post-neonatal intrahepatic cholestasis caused by citrin deficiency (post-NICCD) phase, while developing a predictive model for failure to thrive (FTT) using selected metabolites. Method: A case-control study was conducted from October 2020 to July 2024, including 16 NICCD patients, 31 NICCD-matched controls, 34 post-NICCD patients, and 70 post-NICCD-matched controls. Post-NICCD patients were further stratified into two groups based on growth outcomes. Biomarkers for FTT were identified using Lasso regression and random forest analysis. A non-invasive predictive model was developed, visualized as a nomogram, and internally validated using the enhanced bootstrap method. The model's performance was evaluated with receiver operating characteristic curves and calibration curves. Metabolite concentrations (amino acids, acylcarnitines, organic acids, and free fatty acids) were measured using liquid chromatography or ultra-performance liquid chromatography-tandem mass spectrometry. Results: The biosynthesis of unsaturated fatty acids was identified as the most significantly altered pathway in post-NICCD patients. Twelve residual metabolites altered during both NICCD and post-NICCD phases were identified, including: 2-hydroxyisovaleric acid, alpha-ketoisovaleric acid, C5:1, 3-methyl-2-oxovaleric acid, C18:1OH, C20:4, myristic acid, eicosapentaenoic acid, carnosine, hydroxylysine, phenylpyruvic acid, and 2-methylcitric acid. Lasso regression and random forest analysis identified kynurenine, arginine, alanine, and aspartate as the optimal biomarkers for predicting FTT in post-NICCD patients. The predictive model constructed with these four biomarkers demonstrated an AUC of 0.947. Conclusion: While post-NICCD patients recover clinically and biochemically, their metabolic profiles remain incompletely restored. The predictive model based on kynurenine, arginine, alanine, and aspartate provides robust diagnostic performance for detecting FTT in post-NICCD patients.
      pubtype: Academic Journal
      doctype:
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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