Newborn screening for central congenital hypothyroidism: past, present and future

Congenital hypothyroidism (CH) is defined as thyroid hormone deficiency at birth and constitutes one of the most common causes of preventable intellectual disability worldwide. Central CH is caused by insufficient pituitary or hypothalamic control of thyroid function, biochemically characterized by...

Full description

Saved in:
Bibliographic Details
Main Authors: Mark R Garrelfs, Christiaan F Mooij, Anita Boelen, A S Paul van Trotsenburg, Nitash Zwaveling-Soonawala
Format: Article
Language:English
Published: Bioscientifica 2025-02-01
Series:European Thyroid Journal
Subjects:
Online Access:https://etj.bioscientifica.com/view/journals/etj/14/1/ETJ-24-0329.xml
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850206844478291968
author Mark R Garrelfs
Christiaan F Mooij
Anita Boelen
A S Paul van Trotsenburg
Nitash Zwaveling-Soonawala
author_facet Mark R Garrelfs
Christiaan F Mooij
Anita Boelen
A S Paul van Trotsenburg
Nitash Zwaveling-Soonawala
author_sort Mark R Garrelfs
collection DOAJ
description Congenital hypothyroidism (CH) is defined as thyroid hormone deficiency at birth and constitutes one of the most common causes of preventable intellectual disability worldwide. Central CH is caused by insufficient pituitary or hypothalamic control of thyroid function, biochemically characterized by a low serum free thyroxine (fT4), in combination with a low, normal or mildly elevated thyroid-stimulating hormone (TSH). Central CH is less common than primary CH and is part of multiple pituitary hormone deficiencies (MPHD) in most of the cases. MPHD at birth, also known as ‘congenital hypopituitarism’, is a potentially life-threatening condition due to the possible co-occurrence of adrenocorticotropin hormone and growth hormone deficiency that can result in severe hypoglycemia and adrenal crisis. To date, central CH is the only pituitary hormone deficiency suitable for newborn screening (NBS), providing an opportunity for early detection of MPHD. Even though the first NBS programs utilized T4-based methods that were able to identify central CH, most countries have since transitioned to TSH-based approaches due to the high rate of false positives associated with T4-based strategies. Now, 50 years after the introduction of NBS for CH, only a few countries around the world have a screening program capable of detecting central CH. In this paper, we review the past, present and future of NBS for central CH. We will outline the importance of early detection of central CH and discuss the challenges and opportunities of screening for this condition.
format Article
id doaj-art-7d4032413424415b9a70a7ecda8b7484
institution OA Journals
issn 2235-0802
language English
publishDate 2025-02-01
publisher Bioscientifica
record_format Article
series European Thyroid Journal
spelling doaj-art-7d4032413424415b9a70a7ecda8b74842025-08-20T02:10:41ZengBioscientificaEuropean Thyroid Journal2235-08022025-02-0114110.1530/ETJ-24-03291Newborn screening for central congenital hypothyroidism: past, present and futureMark R Garrelfs0Christiaan F Mooij1Anita Boelen2A S Paul van Trotsenburg3Nitash Zwaveling-Soonawala4Department of Pediatric Endocrinology, Emma Children’s Hospital, Amsterdam University Medical Centers, University of Amsterdam and Vrije Universiteit, European Reference Network on Rare Endocrine Conditions (Endo-ERN), Amsterdam, The NetherlandsDepartment of Pediatric Endocrinology, Emma Children’s Hospital, Amsterdam University Medical Centers, University of Amsterdam and Vrije Universiteit, European Reference Network on Rare Endocrine Conditions (Endo-ERN), Amsterdam, The NetherlandsAmsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, The NetherlandsDepartment of Pediatric Endocrinology, Emma Children’s Hospital, Amsterdam University Medical Centers, University of Amsterdam and Vrije Universiteit, European Reference Network on Rare Endocrine Conditions (Endo-ERN), Amsterdam, The NetherlandsDepartment of Pediatric Endocrinology, Emma Children’s Hospital, Amsterdam University Medical Centers, University of Amsterdam and Vrije Universiteit, European Reference Network on Rare Endocrine Conditions (Endo-ERN), Amsterdam, The NetherlandsCongenital hypothyroidism (CH) is defined as thyroid hormone deficiency at birth and constitutes one of the most common causes of preventable intellectual disability worldwide. Central CH is caused by insufficient pituitary or hypothalamic control of thyroid function, biochemically characterized by a low serum free thyroxine (fT4), in combination with a low, normal or mildly elevated thyroid-stimulating hormone (TSH). Central CH is less common than primary CH and is part of multiple pituitary hormone deficiencies (MPHD) in most of the cases. MPHD at birth, also known as ‘congenital hypopituitarism’, is a potentially life-threatening condition due to the possible co-occurrence of adrenocorticotropin hormone and growth hormone deficiency that can result in severe hypoglycemia and adrenal crisis. To date, central CH is the only pituitary hormone deficiency suitable for newborn screening (NBS), providing an opportunity for early detection of MPHD. Even though the first NBS programs utilized T4-based methods that were able to identify central CH, most countries have since transitioned to TSH-based approaches due to the high rate of false positives associated with T4-based strategies. Now, 50 years after the introduction of NBS for CH, only a few countries around the world have a screening program capable of detecting central CH. In this paper, we review the past, present and future of NBS for central CH. We will outline the importance of early detection of central CH and discuss the challenges and opportunities of screening for this condition.https://etj.bioscientifica.com/view/journals/etj/14/1/ETJ-24-0329.xmlcentral congenital hypothyroidismnewborn screeningmultiple pituitary hormone deficienciesthyroxinepituitary
spellingShingle Mark R Garrelfs
Christiaan F Mooij
Anita Boelen
A S Paul van Trotsenburg
Nitash Zwaveling-Soonawala
Newborn screening for central congenital hypothyroidism: past, present and future
European Thyroid Journal
central congenital hypothyroidism
newborn screening
multiple pituitary hormone deficiencies
thyroxine
pituitary
title Newborn screening for central congenital hypothyroidism: past, present and future
title_full Newborn screening for central congenital hypothyroidism: past, present and future
title_fullStr Newborn screening for central congenital hypothyroidism: past, present and future
title_full_unstemmed Newborn screening for central congenital hypothyroidism: past, present and future
title_short Newborn screening for central congenital hypothyroidism: past, present and future
title_sort newborn screening for central congenital hypothyroidism past present and future
topic central congenital hypothyroidism
newborn screening
multiple pituitary hormone deficiencies
thyroxine
pituitary
url https://etj.bioscientifica.com/view/journals/etj/14/1/ETJ-24-0329.xml
work_keys_str_mv AT markrgarrelfs newbornscreeningforcentralcongenitalhypothyroidismpastpresentandfuture
AT christiaanfmooij newbornscreeningforcentralcongenitalhypothyroidismpastpresentandfuture
AT anitaboelen newbornscreeningforcentralcongenitalhypothyroidismpastpresentandfuture
AT aspaulvantrotsenburg newbornscreeningforcentralcongenitalhypothyroidismpastpresentandfuture
AT nitashzwavelingsoonawala newbornscreeningforcentralcongenitalhypothyroidismpastpresentandfuture