Background The incidence of congenital hypothyroidism (CH) has been increasing worldwide. This large-scale population study investigated the use and discontinuation of levothyroxine in a nationwide cohort of patients with presumed CH over 10 years. Methods Diagnostic, laboratory, and prescription codes from the National Health Insurance Service (NHIS) were used to identify patients treated for presumed CH in early life between 2008 and 2018. Healthcare utilization data were assessed for levothyroxine treatment duration and characteristics associated with treatment discontinuation. Results The NHIS data identified 10,525 patients with presumed CH, whose prevalence and incidence more than doubled during the study period. The prevalence and incidence were significantly higher in preterm infants. The cumulative rates of levothyroxine discontinuation within 3, 5, and 10 years of treatment initiation were 39%, 67%, and 79%, respectively, with higher discontinuation rates observed among patients born preterm or with low birth weight. Male sex, preterm-birth, low birth weight, and prescriptions from non-metropolitan or non-general hospitals, predicted transient levothyroxine use. Conclusion This study provides national-level estimates based on NHIS data showing an increasing number of infants diagnosed with presumed CH, with a majority eventually discontinuing levothyroxine. Further validation of NHIS data and evaluation of long-term treatment effects may improve epidemiologic research and the clinical management of CH. Impact <UnorderedList Mark="Bullet"> <ItemContent> Nationwide cohort data showed a marked rise in infants treated for presumed congenital hypothyroidism, with most discontinuing levothyroxine within 3–5 years. </ItemContent> <ItemContent> Male sex, preterm birth, low birth weight, and prescriptions from non-metropolitan or non-general hospitals were predictors for transient treatment. </ItemContent> <ItemContent> Earlier and greater discontinuation rates were observed in preterm birth and low birthweight infants. </ItemContent> <ItemContent> This study provides large-scale, real-world evidence on long-term treatment patterns and discontinuation of levothyroxine. The findings highlight the need for validated registry data, standardized national guidelines, and outcome-based research to optimize diagnosis and management. </ItemContent> </UnorderedList>

Levothyroxine use and discontinuation in presumed congenital hypothyroidism: a 10-year nationwide cohort study

期刊论文
发表日期：

2026年6月16日

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Seon Young Kim,
Hyun Ah Woo,
Eun Hye Lee,
You Ree Kim,
Ja Hyang Cho,
Kye Shik Shim,
Hae Woon Jung

摘要

Background

The incidence of congenital hypothyroidism (CH) has been increasing worldwide. This large-scale population study investigated the use and discontinuation of levothyroxine in a nationwide cohort of patients with presumed CH over 10 years.

Methods

Diagnostic, laboratory, and prescription codes from the National Health Insurance Service (NHIS) were used to identify patients treated for presumed CH in early life between 2008 and 2018. Healthcare utilization data were assessed for levothyroxine treatment duration and characteristics associated with treatment discontinuation.

Results

The NHIS data identified 10,525 patients with presumed CH, whose prevalence and incidence more than doubled during the study period. The prevalence and incidence were significantly higher in preterm infants. The cumulative rates of levothyroxine discontinuation within 3, 5, and 10 years of treatment initiation were 39%, 67%, and 79%, respectively, with higher discontinuation rates observed among patients born preterm or with low birth weight. Male sex, preterm-birth, low birth weight, and prescriptions from non-metropolitan or non-general hospitals, predicted transient levothyroxine use.

Conclusion

This study provides national-level estimates based on NHIS data showing an increasing number of infants diagnosed with presumed CH, with a majority eventually discontinuing levothyroxine. Further validation of NHIS data and evaluation of long-term treatment effects may improve epidemiologic research and the clinical management of CH.

Impact

Nationwide cohort data showed a marked rise in infants treated for presumed congenital hypothyroidism, with most discontinuing levothyroxine within 3–5 years.

Male sex, preterm birth, low birth weight, and prescriptions from non-metropolitan or non-general hospitals were predictors for transient treatment.

Earlier and greater discontinuation rates were observed in preterm birth and low birthweight infants.

This study provides large-scale, real-world evidence on long-term treatment patterns and discontinuation of levothyroxine. The findings highlight the need for validated registry data, standardized national guidelines, and outcome-based research to optimize diagnosis and management.