Individualized treatment to optimize eventual cognitive outcome in congenital hypothyroidism

作者：Bongers Schokking Jacoba J^*; Resing Wilma C M; Oostdijk Wilma; de Rijke Yolanda B; Keizer Schrama Sabine M P F de Muinck

来源：Pediatric Research, 2016, 80(6): 816-823.

DOI：10.1038/pr.2016.159

BACKGROUND: In congenital hypothyroidism (CH), age-specific reference ranges (asRR) for fT(4) and thyrotropine (TSH) are usually used to signal over/under-treatment. We compared the consequences of individual fT(4) steady-state concentrations (SSC's) and asRR regarding over-treatment signaling and intelligence quotient at 11 y (IQ(11)) and the effect of early over -treatment with high L-T-4 dosages on IQ(11).
METHODS: Sixty-one patients (27 severe, 34 mild CH) were psychologically tested at 1.8, 6, and 11 y. Development scores were related to over-treatment in the period 0-24 mo, relative to either individual fT(4)SSC's or asRR. Three groups were formed, based on severity of over/under-treatment 0-5 mo (severe, mild, and no over/under-treatment).
RESULTS: FT4 and TSH asRR missed 41-50% of the over treatment episodes and consequently 22% of the over-treated patients, classified as such by fT(4)SSC's. Severe over -treatment 0-5 mo led to lowered and to a 5.5-fold higher risk of IQ(11) < 85 than other treatment regimes. Under-treatment had no effect on development scores. Initial L-T-4 dosages >10 mu g/kg resulted in a 3.7-fold higher risk of over-treatment than lower dosages.
CONCLUSIONS: Data suggest that asRR, compared to fT(4)SSC's, signal over-treatment insufficiently. Using fT(4)SSC's and avoiding over-treatment may optimize cognitive outcome. Lowered are usually a late complication of severe early over-treatment.

• 出版日期2016-12