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先天性甲状腺功能减退症的左甲状腺素需求量:一项为期12年的纵向研究。

Levothyroxine requirement in congenital hypothyroidism: a 12-year longitudinal study.

作者信息

Delvecchio Maurizio, Salerno Mariacarolina, Vigone Maria Cristina, Wasniewska Malgorzata, Popolo Pietro Pio, Lapolla Rosa, Mussa Alessandro, Tronconi Giulia Maria, D'Acunzo Ida, Di Mase Raffaella, Falcone Rosa Maria, Corrias Andrea, De Luca Filippo, Weber Giovanna, Cavallo Luciano, Faienza Maria Felicia

机构信息

Department of Biomedical Sciences and Human Oncology, University of Bari Aldo Moro, Bari, Italy.

Pediatric Section-Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy.

出版信息

Endocrine. 2015 Dec;50(3):674-80. doi: 10.1007/s12020-015-0574-1. Epub 2015 Mar 12.

DOI:10.1007/s12020-015-0574-1
PMID:25762444
Abstract

The aim of the replacement therapy with levothyroxine in congenital hypothyroidism (CH) is to correct hypothyroidism and ensure normal growth and neuropsychological development. Few data are available about the appropriate dose during childhood and early adolescence; therefore, we performed a multicenter observational study in a large population of patients with CH to assess the required levothyroxine dose to obtain euthyroidism. We recruited 216 patients with permanent CH classified into three groups (agenesia, ectopia, and in situ gland) on the basis of the thyroid imaging. The levothyroxine dose was recorded at 6 and 12 months and then yearly until 12 years of age. The daily levothyroxine requirement progressively decreased during the follow-up, irrespective of etiology. It was significantly lower in patients with in situ gland than in patients with athyreosis during the entire study period and with ectopic gland from the age of 1 year. The levothyroxine requirement at 6 months of age was correlated with the requirement at each later time-point. The daily dose was modified less frequently in patients with in situ thyroid (36 %) than in patients with ectopic gland (41.4 %) or with athyreosis (43.6 %). Patients with in situ gland required a lower dose than the other two subgroups. The dose at 6 months seems predictive of the requirement until 12 years of age. Euthyroidism may be achieved in pre-school and in-school patients by 3-4 and 2-3 µg/kg/day (70-90 and 60-80 µg/m(2)/day) of levothyroxine, respectively.

摘要

先天性甲状腺功能减退症(CH)采用左甲状腺素替代治疗的目的是纠正甲状腺功能减退,确保正常生长及神经心理发育。关于儿童期和青春期早期的合适剂量,现有数据较少;因此,我们对一大群CH患者进行了一项多中心观察性研究,以评估实现甲状腺功能正常所需的左甲状腺素剂量。我们招募了216例永久性CH患者,根据甲状腺影像学将其分为三组(甲状腺缺如、异位和原位甲状腺)。记录6个月和12个月时以及之后每年直至12岁时的左甲状腺素剂量。在整个随访期间,无论病因如何,每日左甲状腺素需求量均逐渐下降。在整个研究期间,原位甲状腺患者的需求量显著低于甲状腺缺如患者,1岁以后低于异位甲状腺患者。6个月大时的左甲状腺素需求量与之后各时间点的需求量相关。原位甲状腺患者(36%)每日剂量调整频率低于异位甲状腺患者(41.4%)或甲状腺缺如患者(43.6%)。原位甲状腺患者所需剂量低于其他两个亚组。6个月时的剂量似乎可预测直至12岁时的需求量。学龄前和学龄期患者分别通过每日3 - 4和2 - 3 μg/kg(70 - 90和60 - 80 μg/m²/天)的左甲状腺素可实现甲状腺功能正常。

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Guidelines for Newborn Screening of Congenital Hypothyroidism (2021 Revision).先天性甲状腺功能减退症新生儿筛查指南(2021年修订版)
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