Treatment of congenital hypothyroidism: comparison between L-thyroxine oral solution and tablet formulations up to 3 years of age

in European Journal of Endocrinology
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  • 1 Department of Pediatrics, Endocrine Unit, Vita-Salute San Raffaele University, IRCSS San Raffaele, Milan, Italy
  • | 2 Sant’Orsola-Malpighi University Hospital, Pediatric Unit, Program of Pediatric Endocrinology, Bologna, Italy
  • | 3 Pediatric Department, Universitary Hospital of Verona, Verona, Italy
  • | 4 Department of Public Health and Pediatrics, University of Torino, Torino, Italy
  • | 5 Giannina Gaslini Children’s Hospital, Genoa, Italy
  • | 6 Department of Pediatrics, AOU Federico II, Naples, Italy
  • | 7 AUSL-IRCCS Reggio Emilia, Department of Pediatrics, Reggio Emilia, Italy
  • | 8 Department of Pediatrics, University of Messina, Messina, Italy

Correspondence should be addressed to M C Vigone Email vigone.mariacristina@hsr.it
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Objective

Oral solution and tablet formulations of levothyroxine (L-T4) are both used in the treatment of congenital hypothyroidism (CH). However, few studies and with a limited follow-up period have been published comparing these two formulations in children.

Design

The aim of this multicenter study was to compare the effectiveness of L-T4 oral solution (with ethanol as excipient) and tablet formulation in children with CH up to 3 years of age.

Methods

Children diagnosed with CH between 2006 and 2015 were enrolled and divided into two groups according to the L-T4 formulation used: solution in drops (group D) or tablets (group T). Auxological parameters, thyroid-stimulating hormone (TSH) and free thyroxine (FT4) values and L-T4 dose were collected at diagnosis and at 15 days, 1, 3, 6, 12, 24 and 36 months of treatment. The developmental quotient (DQ) at 1 and 3 years of age was evaluated using Griffiths’ Scale.

Results

In this study, 254 children were enrolled among which 117 were treated with solution and 137 with tablets. Auxological parameters, dose and thyroid function values at diagnosis, 3, 6, 12, 24, 36 months were not significantly different. TSH at 15 days (P = 0.002) and 1 month (P = 0.009) was significantly reduced in group D. At 2-year follow-up, median TSH was significantly lower in group T (P = 0.03). No statistical difference was detected between the median DQ; however, group D showed lower values in the language subscale at 12 months and in eye–hand coordination at 36 months.

Conclusions

Both therapeutic strategies are effective in the treatment of CH. A higher risk of overtreatment in the first months of therapy seems to be associated with oral solution L-T4; therefore, a different strategy should be considered when starting and adjusting the dose. No negative effects on cognitive development were observed. The data obtained are encouraging but long-term follow-up is needed.

 

     European Society of Endocrinology

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