目的 观察不同初始治疗剂量和治疗时机对先天性甲状腺功能减低症（CH）的疗效影响。方法 以云南省4个地区新生儿筛查提示TSH异常、最终确诊并治疗随访的98例CH患儿为研究对象，予左旋甲状腺素钠治疗，按初始治疗剂量分为标准剂量组（每日10~15 μg/kg）和小剂量组（每日 < 10 μg/kg），按照治疗起始时间又分为2月龄内治疗组和2月龄后治疗组。监测治疗前及治疗后多时点的甲状腺功能以及体格和神经发育情况。结果 治疗后2周，标准剂量组的TSH低于小剂量组，FT4高于小剂量组，差异有统计学意义（P < 0.05），治疗后其他时间点的TSH、FT4差异均无统计学意义（P > 0.05）；两组在治疗前以及治疗后各个时间点体格和神经发育的差异无统计学意义（P > 0.05）。不同治疗时机组，治疗后各个不同时间点的TSH和FT4及体格发育的差异均无统计学意义（P > 0.05）；2月龄内开始治疗的患儿在6月龄时适应性行为、大运动、精细动作能区评分，12月龄时大运动能区评分，18月龄时精细动作能区评分，以及24月龄时语言能区评分均较高，差异具有统计学意义（P < 0.05）。结论 标准剂量组疗效优于小剂量组，但需注意治疗后甲亢表现；治疗时机对CH患儿神经发育影响大，一经确诊，应尽早开始治疗。
Abstract
Objective To observe the effects of initial doses and treatment timing of levothyroxine (L-T4) on the clinical effcacy in children with congenital hypothyroidism (CH). Methods This study included 98 children who had an abnormal level of thyroid stimulating hormone (TSH) in neonatal screening in four regions of Yunnan Province and who fnally had a confrmed diagnosis of CH. They received treatment with L-T4 and were divided into standard dose group (10-15 μg/kg per day) and low dose group ( < 10 μg/kg per day) by the therapeutic dose of L-T4. Meanwhile, these patients were also classifed into two treatment groups based on the starting time of L-T4 treatment, namely under 2 months old group and more than 2 months old group. The thyroid function and physical and neural development were examined before and after treatment. Results Compared with the low dose group, the standard dose group had a signifcantly lower TSH level and a signifcantly higher free thyroxine (FT4) level at 2 weeks after treatment (P < 0.05). There were no signifcant differences in TSH and FT4 levels at other time points after treatment between the standard and low dose groups (P > 0.05). The physical and neural development were not signifcantly different between the two dose groups before and at all time points after treatment (P > 0.05). At all time points after treatment, the levels of TSH and FT4 and physical development were not signifcantly different between the different starting time groups (P > 0.05). However, the Gesell score was signifcantly higher in the under 2 months old group than in the more than 2 months old group at all time points after treatment (P < 0.05). Conclusions The standard dose group has a better treatment outcome than the low dose group, whereas the symptoms of hyperthyroidism deserve close attention. The treatment timing is vital to the neurodevelopment of children with CH. Once diagnosed, the patients should receive treatments immediately.
关键词
先天性甲状腺功能减低症 /
初始治疗剂量 /
治疗时机 /
儿童
Key words
Congenital hypothyroidism /
Initial dose /
Treatment timing /
Child
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参考文献
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基金
云南省卫生科技计划项目（2011WS004）。
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