Model-Informed Dose Optimization of Spironolactone in Neonates and Infants

<b>Background/Objectives</b>: Spironolactone (SP) has been used off-label in pediatrics since its approval, but its use is challenged by limited pharmacokinetic (PK) data in adults and especially in children. <b>Methods</b>: Physiologically based pharmacokinetic (PBPK) models...

Full description

Saved in:
Bibliographic Details
Main Authors: Amira Soliman, Leandro F. Pippa, Jana Lass, Stephanie Leroux, Valvanera Vozmediano, Natalia V. de Moraes
Format: Article
Language:English
Published: MDPI AG 2025-03-01
Series:Pharmaceuticals
Subjects:
Online Access:https://www.mdpi.com/1424-8247/18/3/355
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850207809652654080
author Amira Soliman
Leandro F. Pippa
Jana Lass
Stephanie Leroux
Valvanera Vozmediano
Natalia V. de Moraes
author_facet Amira Soliman
Leandro F. Pippa
Jana Lass
Stephanie Leroux
Valvanera Vozmediano
Natalia V. de Moraes
author_sort Amira Soliman
collection DOAJ
description <b>Background/Objectives</b>: Spironolactone (SP) has been used off-label in pediatrics since its approval, but its use is challenged by limited pharmacokinetic (PK) data in adults and especially in children. <b>Methods</b>: Physiologically based pharmacokinetic (PBPK) models for SP and its active metabolites, canrenone (CAN) and 7α thio-methyl spironolactone (TMS), in adults were developed. These models aim to enhance understanding of SP’s PK and provide a basis for predicting PK and optimizing SP dosing in infants and neonates. Given SP’s complex metabolism, we assumed complete conversion to CAN and TMS by CES1 enzymes, fitting CES1-mediated metabolism to the parent-metabolite model using PK data. We incorporated ontogeny for CES1 and CYP3A4 and other age-related physiological changes into the model to anticipate PK in the pediatric population. <b>Results</b>: The PBPK models for SP, CAN, and TMS accurately captured the observed PK data in healthy adults across various dosing regimens, including the impact of food on drug exposure. The pediatric PBPK model was evaluated using PK data from infants and neonates. Simulations indicate that 2.5 mg/kg in 6-month to 2-year infants and 2 mg/kg in 1–6-months infants matched the total unbound systemic exposure equivalent to the standard recommended daily maintenance dose of 100 mg in adults for treating edema. <b>Conclusions</b>: The developed PBPK model provides valuable insights for dosing decisions and optimizing therapeutic outcomes, especially in populations where clinical studies are challenging.
format Article
id doaj-art-a51b61d8e58a4c87b020dade8bd49113
institution OA Journals
issn 1424-8247
language English
publishDate 2025-03-01
publisher MDPI AG
record_format Article
series Pharmaceuticals
spelling doaj-art-a51b61d8e58a4c87b020dade8bd491132025-08-20T02:10:24ZengMDPI AGPharmaceuticals1424-82472025-03-0118335510.3390/ph18030355Model-Informed Dose Optimization of Spironolactone in Neonates and InfantsAmira Soliman0Leandro F. Pippa1Jana Lass2Stephanie Leroux3Valvanera Vozmediano4Natalia V. de Moraes5Center for Pharmacometrics and Systems Pharmacology, Department of Pharmaceutics, University of Florida, Orlando, FL 32827, USACenter for Pharmacometrics and Systems Pharmacology, Department of Pharmaceutics, University of Florida, Orlando, FL 32827, USAInstitute of Pharmacy, University of Tartu, 50411 Tartu, EstoniaCHU Rennes, University Hospital Rennes, Inserm, EHESP, Irset—UMR_S 1085, F-35000 Rennes, FranceCenter for Pharmacometrics and Systems Pharmacology, Department of Pharmaceutics, University of Florida, Orlando, FL 32827, USACenter for Pharmacometrics and Systems Pharmacology, Department of Pharmaceutics, University of Florida, Orlando, FL 32827, USA<b>Background/Objectives</b>: Spironolactone (SP) has been used off-label in pediatrics since its approval, but its use is challenged by limited pharmacokinetic (PK) data in adults and especially in children. <b>Methods</b>: Physiologically based pharmacokinetic (PBPK) models for SP and its active metabolites, canrenone (CAN) and 7α thio-methyl spironolactone (TMS), in adults were developed. These models aim to enhance understanding of SP’s PK and provide a basis for predicting PK and optimizing SP dosing in infants and neonates. Given SP’s complex metabolism, we assumed complete conversion to CAN and TMS by CES1 enzymes, fitting CES1-mediated metabolism to the parent-metabolite model using PK data. We incorporated ontogeny for CES1 and CYP3A4 and other age-related physiological changes into the model to anticipate PK in the pediatric population. <b>Results</b>: The PBPK models for SP, CAN, and TMS accurately captured the observed PK data in healthy adults across various dosing regimens, including the impact of food on drug exposure. The pediatric PBPK model was evaluated using PK data from infants and neonates. Simulations indicate that 2.5 mg/kg in 6-month to 2-year infants and 2 mg/kg in 1–6-months infants matched the total unbound systemic exposure equivalent to the standard recommended daily maintenance dose of 100 mg in adults for treating edema. <b>Conclusions</b>: The developed PBPK model provides valuable insights for dosing decisions and optimizing therapeutic outcomes, especially in populations where clinical studies are challenging.https://www.mdpi.com/1424-8247/18/3/355PBPKinfantspediatricsspironolactonedose optimization
spellingShingle Amira Soliman
Leandro F. Pippa
Jana Lass
Stephanie Leroux
Valvanera Vozmediano
Natalia V. de Moraes
Model-Informed Dose Optimization of Spironolactone in Neonates and Infants
Pharmaceuticals
PBPK
infants
pediatrics
spironolactone
dose optimization
title Model-Informed Dose Optimization of Spironolactone in Neonates and Infants
title_full Model-Informed Dose Optimization of Spironolactone in Neonates and Infants
title_fullStr Model-Informed Dose Optimization of Spironolactone in Neonates and Infants
title_full_unstemmed Model-Informed Dose Optimization of Spironolactone in Neonates and Infants
title_short Model-Informed Dose Optimization of Spironolactone in Neonates and Infants
title_sort model informed dose optimization of spironolactone in neonates and infants
topic PBPK
infants
pediatrics
spironolactone
dose optimization
url https://www.mdpi.com/1424-8247/18/3/355
work_keys_str_mv AT amirasoliman modelinformeddoseoptimizationofspironolactoneinneonatesandinfants
AT leandrofpippa modelinformeddoseoptimizationofspironolactoneinneonatesandinfants
AT janalass modelinformeddoseoptimizationofspironolactoneinneonatesandinfants
AT stephanieleroux modelinformeddoseoptimizationofspironolactoneinneonatesandinfants
AT valvaneravozmediano modelinformeddoseoptimizationofspironolactoneinneonatesandinfants
AT nataliavdemoraes modelinformeddoseoptimizationofspironolactoneinneonatesandinfants