Bongers-Schokking J J, Koot H M, Wiersma D, Verkerk P H, de Muinck Keizer-Schrama S M
Department of Endocrinology, the Department of Child and Adolescent Psychiatry, the Academic Hospital Sophia Children's Hospital, Rotterdam, The Netherlands.
J Pediatr. 2000 Mar;136(3):292-7. doi: 10.1067/mpd.2000.103351.
To test whether early treatment with a high initial dose of levothyroxine can prevent suboptimal mental development in all neonates with congenital hypothyroidism (CH).
Sixty-one patients, 27 with severe CH and 34 with mild CH, were treated either early (<13 days) or late (> or =13 days) with either a high initial dose of levothyroxine (> or =9.5 microg/kg/d) or a low initial dose (<9.5 microg/kg/d). With these criteria, 4 treatment groups were formed. The results of the Bayley test, performed at the age of 10 to 30 months and expressed as mental developmental index (MDI) and psychomotor developmental index (PDI), were related to socioeconomic status, treatment group, initial free thyroxine (FT(4)) concentration, and mean FT(4) concentration during the first 3 months of treatment (FT(4)-A) and the ensuing 9 months (FT(4)-B).
Mean (+/- SD) MDI was 113 +/- 14, and mean PDI was 114 +/- 12. In the severe CH group, only the patients treated early with a high initial dose had normal MDI scores (124 +/- 16), whereas the scores of the other groups ranged from 97 to 103. In contrast, all patients in the mild CH group had normal scores (range, 122-125), except those in the group treated late with a low initial dose, whose score was 110 +/- 10. Forty-three percent of the variance in MDI and PDI scores was explained by treatment factors, such as the treatment group, initial FT(4) concentration, FT(4)-A, and FT(4)-B.
Our data suggest that optimal treatment includes achievement of euthyroidism before the third week of life by initiation of therapy before 13 days with a levothyroxine dose above 9.5 microg/kg/d and maintenance of FT(4) concentrations in the upper normal range during the first year. Thus treated, patients with CH can achieve normal psychomotor development at 10 to 30 months, irrespective of the severity of the disease.
检测先天性甲状腺功能减退症（CH）患儿早期使用高初始剂量左甲状腺素治疗是否能预防其智力发育欠佳。
61例患者，27例为重度CH，34例为轻度CH，分别于早期（<13天）或晚期（≥13天）接受高初始剂量（≥9.5μg/kg/d）或低初始剂量（<9.5μg/kg/d）的左甲状腺素治疗。依此标准，形成4个治疗组。在10至30个月龄时进行贝利测试，结果以智力发育指数（MDI）和精神运动发育指数（PDI）表示，并与社会经济地位、治疗组、初始游离甲状腺素（FT4）浓度以及治疗前3个月（FT4 - A）和随后9个月（FT4 - B）的平均FT4浓度相关。
平均（±标准差）MDI为113±14，平均PDI为114±12。在重度CH组中，仅早期接受高初始剂量治疗的患者MDI评分正常（124±16），而其他组的评分在97至103之间。相比之下，轻度CH组中所有患者评分均正常（范围为122 - 125），但晚期接受低初始剂量治疗组的患者评分为110±10。MDI和PDI评分中43%的变异可由治疗因素解释，如治疗组、初始FT4浓度、FT4 - A和FT4 - B。
我们的数据表明，最佳治疗包括在出生后第3周前实现甲状腺功能正常，即在13天前开始使用剂量高于9.5μg/kg/d的左甲状腺素进行治疗，并在第一年将FT4浓度维持在正常范围上限。经如此治疗，CH患儿在10至30个月时可实现正常的精神运动发育，而不论疾病严重程度如何。
