New QSAR Models to Predict Human Transthyretin Disruption by Per- and Polyfluoroalkyl Substances (PFAS): Development and Application

Per- and polyfluoroalkyl substances (PFAS) are of concern because of their potential thyroid hormone system disruption by binding to human transthyretin (hTTR). However, the amount of experimental data is scarce. In this work, new classification and regression QSARs were developed to predict the hTT...

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Main Authors: Marco Evangelista, Nicola Chirico, Ester Papa
Format: Article
Language:English
Published: MDPI AG 2025-07-01
Series:Toxics
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Online Access:https://www.mdpi.com/2305-6304/13/7/590
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author Marco Evangelista
Nicola Chirico
Ester Papa
author_facet Marco Evangelista
Nicola Chirico
Ester Papa
author_sort Marco Evangelista
collection DOAJ
description Per- and polyfluoroalkyl substances (PFAS) are of concern because of their potential thyroid hormone system disruption by binding to human transthyretin (hTTR). However, the amount of experimental data is scarce. In this work, new classification and regression QSARs were developed to predict the hTTR disruption based on experimental data measured for 134 PFAS. Bootstrapping, randomization procedures, and external validation were used to check for overfitting, to avoid random correlations, and to evaluate the predictivity of the QSARs, respectively. The best QSARs were characterized by good performances (e.g., training and test accuracies in classification of 0.89 and 0.85, respectively; R<sup>2</sup>, Q<sup>2</sup><sub>loo</sub>, and Q<sup>2</sup><sub>F3</sub> in regression of 0.81, 0.77, and 0.82, respectively) and significantly broader domains compared to the few existing similar models. The application of QSARs application to the OECD List of PFAS allowed for the identification of structural categories of major concern, such as per- and polyfluoroalkyl ether-based, perfluoroalkyl carbonyl, and perfluoroalkane sulfonyl compounds. Forty-nine PFAS showed a stronger binding affinity to hTTR than the natural ligand T4. Uncertainty quantification for each model and prediction further enhanced the reliability assessment of predictions. The implementation of the new QSARs in non-commercial software facilitates their application to support future research efforts and regulatory actions.
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spelling doaj-art-b50ff68c98764b72b2732063edb8b7a32025-08-20T02:47:07ZengMDPI AGToxics2305-63042025-07-0113759010.3390/toxics13070590New QSAR Models to Predict Human Transthyretin Disruption by Per- and Polyfluoroalkyl Substances (PFAS): Development and ApplicationMarco Evangelista0Nicola Chirico1Ester Papa2QSAR Research Unit in Environmental Chemistry and Ecotoxicology, Department of Theoretical and Applied Sciences, University of Insubria, via J.H. Dunant 3, 21100 Varese, ItalyQSAR Research Unit in Environmental Chemistry and Ecotoxicology, Department of Theoretical and Applied Sciences, University of Insubria, via J.H. Dunant 3, 21100 Varese, ItalyQSAR Research Unit in Environmental Chemistry and Ecotoxicology, Department of Theoretical and Applied Sciences, University of Insubria, via J.H. Dunant 3, 21100 Varese, ItalyPer- and polyfluoroalkyl substances (PFAS) are of concern because of their potential thyroid hormone system disruption by binding to human transthyretin (hTTR). However, the amount of experimental data is scarce. In this work, new classification and regression QSARs were developed to predict the hTTR disruption based on experimental data measured for 134 PFAS. Bootstrapping, randomization procedures, and external validation were used to check for overfitting, to avoid random correlations, and to evaluate the predictivity of the QSARs, respectively. The best QSARs were characterized by good performances (e.g., training and test accuracies in classification of 0.89 and 0.85, respectively; R<sup>2</sup>, Q<sup>2</sup><sub>loo</sub>, and Q<sup>2</sup><sub>F3</sub> in regression of 0.81, 0.77, and 0.82, respectively) and significantly broader domains compared to the few existing similar models. The application of QSARs application to the OECD List of PFAS allowed for the identification of structural categories of major concern, such as per- and polyfluoroalkyl ether-based, perfluoroalkyl carbonyl, and perfluoroalkane sulfonyl compounds. Forty-nine PFAS showed a stronger binding affinity to hTTR than the natural ligand T4. Uncertainty quantification for each model and prediction further enhanced the reliability assessment of predictions. The implementation of the new QSARs in non-commercial software facilitates their application to support future research efforts and regulatory actions.https://www.mdpi.com/2305-6304/13/7/590endocrine disruptionhuman transthyretin disruptionnew approach methodologiesPFASQSAR
spellingShingle Marco Evangelista
Nicola Chirico
Ester Papa
New QSAR Models to Predict Human Transthyretin Disruption by Per- and Polyfluoroalkyl Substances (PFAS): Development and Application
Toxics
endocrine disruption
human transthyretin disruption
new approach methodologies
PFAS
QSAR
title New QSAR Models to Predict Human Transthyretin Disruption by Per- and Polyfluoroalkyl Substances (PFAS): Development and Application
title_full New QSAR Models to Predict Human Transthyretin Disruption by Per- and Polyfluoroalkyl Substances (PFAS): Development and Application
title_fullStr New QSAR Models to Predict Human Transthyretin Disruption by Per- and Polyfluoroalkyl Substances (PFAS): Development and Application
title_full_unstemmed New QSAR Models to Predict Human Transthyretin Disruption by Per- and Polyfluoroalkyl Substances (PFAS): Development and Application
title_short New QSAR Models to Predict Human Transthyretin Disruption by Per- and Polyfluoroalkyl Substances (PFAS): Development and Application
title_sort new qsar models to predict human transthyretin disruption by per and polyfluoroalkyl substances pfas development and application
topic endocrine disruption
human transthyretin disruption
new approach methodologies
PFAS
QSAR
url https://www.mdpi.com/2305-6304/13/7/590
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AT nicolachirico newqsarmodelstopredicthumantransthyretindisruptionbyperandpolyfluoroalkylsubstancespfasdevelopmentandapplication
AT esterpapa newqsarmodelstopredicthumantransthyretindisruptionbyperandpolyfluoroalkylsubstancespfasdevelopmentandapplication