Primary congenital hypothyroidism: a clinical review

Congenital hypothyroidism (CH) is the most common neonatal endocrine disorder. It is one of the clinical conditions that has benefited most from the introduction of newborn screening 50 years ago, as clinical management has changed and long-term consequences have been significantly reduced. In areas...

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Main Authors: Paolo Cavarzere, Valentina Mancioppi, Riccardo Battiston, Valentina Lupieri, Anita Morandi, Claudio Maffeis
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-08-01
Series:Frontiers in Endocrinology
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Online Access:https://www.frontiersin.org/articles/10.3389/fendo.2025.1592655/full
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author Paolo Cavarzere
Valentina Mancioppi
Riccardo Battiston
Valentina Lupieri
Anita Morandi
Anita Morandi
Claudio Maffeis
Claudio Maffeis
author_facet Paolo Cavarzere
Valentina Mancioppi
Riccardo Battiston
Valentina Lupieri
Anita Morandi
Anita Morandi
Claudio Maffeis
Claudio Maffeis
author_sort Paolo Cavarzere
collection DOAJ
description Congenital hypothyroidism (CH) is the most common neonatal endocrine disorder. It is one of the clinical conditions that has benefited most from the introduction of newborn screening 50 years ago, as clinical management has changed and long-term consequences have been significantly reduced. In areas where neonatal screening is active, most affected patients show a clinically normal phenotype and/or only mild symptoms. At the same time, thanks to a progressive reduction in the TSH level used as cut-off for neonatal screening, the number of cases of CH with gland in situ is increasing, while the number of patients with abnormal thyroid development has remained essentially unchanged over time. Furthermore, important changes are observed in managing patients with CH and gland in situ. On the one hand, they are subjected to genetic investigations to understand the underlying molecular mechanism; on the other hand, a reassessment of thyroid function is suggested starting from the sixth month of life if their L-thyroxine requirement is low. This review aims to describe the clinical approach to CH and to optimize the management and treatment of this disease.
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publishDate 2025-08-01
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series Frontiers in Endocrinology
spelling doaj-art-b2eba59a0f664a978cad9d221a0d919f2025-08-20T03:59:36ZengFrontiers Media S.A.Frontiers in Endocrinology1664-23922025-08-011610.3389/fendo.2025.15926551592655Primary congenital hypothyroidism: a clinical reviewPaolo Cavarzere0Valentina Mancioppi1Riccardo Battiston2Valentina Lupieri3Anita Morandi4Anita Morandi5Claudio Maffeis6Claudio Maffeis7Department of Mother and Child, Pediatric Unit B, University Hospital of Verona, Verona, ItalyDepartment of Mother and Child, Pediatric Unit B, University Hospital of Verona, Verona, ItalyDepartment of Mother and Child, Pediatric Unit B, University Hospital of Verona, Verona, ItalyDepartment of Mother and Child, Pediatric Unit B, University Hospital of Verona, Verona, ItalyDepartment of Mother and Child, Pediatric Unit B, University Hospital of Verona, Verona, ItalyDepartment of Surgery, Dentistry, Gynecology and Pediatrics, Section of Pediatric Diabetes and Metabolism, University of Verona, Verona, ItalyDepartment of Mother and Child, Pediatric Unit B, University Hospital of Verona, Verona, ItalyDepartment of Surgery, Dentistry, Gynecology and Pediatrics, Section of Pediatric Diabetes and Metabolism, University of Verona, Verona, ItalyCongenital hypothyroidism (CH) is the most common neonatal endocrine disorder. It is one of the clinical conditions that has benefited most from the introduction of newborn screening 50 years ago, as clinical management has changed and long-term consequences have been significantly reduced. In areas where neonatal screening is active, most affected patients show a clinically normal phenotype and/or only mild symptoms. At the same time, thanks to a progressive reduction in the TSH level used as cut-off for neonatal screening, the number of cases of CH with gland in situ is increasing, while the number of patients with abnormal thyroid development has remained essentially unchanged over time. Furthermore, important changes are observed in managing patients with CH and gland in situ. On the one hand, they are subjected to genetic investigations to understand the underlying molecular mechanism; on the other hand, a reassessment of thyroid function is suggested starting from the sixth month of life if their L-thyroxine requirement is low. This review aims to describe the clinical approach to CH and to optimize the management and treatment of this disease.https://www.frontiersin.org/articles/10.3389/fendo.2025.1592655/fullcongenital hypothyroidismthyroid glandnewborn screening for congenital hypothyroidismdyshormonogenesisdysgenesis
spellingShingle Paolo Cavarzere
Valentina Mancioppi
Riccardo Battiston
Valentina Lupieri
Anita Morandi
Anita Morandi
Claudio Maffeis
Claudio Maffeis
Primary congenital hypothyroidism: a clinical review
Frontiers in Endocrinology
congenital hypothyroidism
thyroid gland
newborn screening for congenital hypothyroidism
dyshormonogenesis
dysgenesis
title Primary congenital hypothyroidism: a clinical review
title_full Primary congenital hypothyroidism: a clinical review
title_fullStr Primary congenital hypothyroidism: a clinical review
title_full_unstemmed Primary congenital hypothyroidism: a clinical review
title_short Primary congenital hypothyroidism: a clinical review
title_sort primary congenital hypothyroidism a clinical review
topic congenital hypothyroidism
thyroid gland
newborn screening for congenital hypothyroidism
dyshormonogenesis
dysgenesis
url https://www.frontiersin.org/articles/10.3389/fendo.2025.1592655/full
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