Levothyroxine Use in Infants and Children with Congenital or Acquired Hypothyroidism

Contributing Editor: Marcia L. Buck, Pharm.D.; Editorial Board: Kristi N. Hofer, Pharm.D.; Michelle W. McCarthy, Pharm.D.


Pediatr Pharm. 2008;14(10) 

In This Article

Use in Congenital Hypothyroidism

Congenital hypothyroidism occurs in approximately 1:3,000 to 1:4,000 newborns, worldwide. Newborn screening has allowed for earlier diagnosis and institution of treatment, avoiding the impairments in growth and development associated with hypothyroidism.[1,4,5] Several studies have demonstrated the benefit of levothyroxine in these patients. In a 2006 paper published in Hormone Research, Marti and colleagues used auditory brain event-related potentials (ERPs) to evaluate neurologic function in 15 children with congenital hypothyroidism (enrolled at the time of diagnosis) and 33 healthy children at 5 years of age.[1] The authors found that the initiation of high dose levothyroxine (mean dose 11.6 mcg/kg/day) within the first weeks of life resulted in functional anatomical and cognitive organization of the auditory system that did not differ from controls. Based on their findings, the authors concluded that intensive treatment appeared to eliminate abnormal neurologic development in children with congenital hypothyroidism.

Early intervention may also improve cardiac function, another complication of congenital hypothyroidism. In a study of 50 neonates, Mao and colleagues found that levothyroxine significantly improved measurements of left and right ventricular function after just one month of treatment.[6] Dinleyici and colleagues recently described a series of four infants with hypothyroidism associated with Down syndrome who had resolution of their pericardial effusions when levothyroxine was initiated.[7]


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