影响先天性甲减左甲状腺素治疗疗效的多因素分析及个体化给药策略探索

Multifactorial Analysis Affecting the Efficacy of Levothyroxine Treatment in Congenital Hypothyroidism and Exploration of Individualized Dosing Strategies

摘要: 目的：探讨影响先天性甲减患儿左甲状腺素治疗疗效的相关因素，并基于上述因素提出左甲状腺素的个体化给药策略。方法：2021年1月至2024年12月期间在我院儿科内分泌门诊确诊并接受规范L-T4治疗的先天性甲减患儿109例作为研究对象，详细收集患儿的临床资料及辅助检查资料。按照疗效将其分为疗效良好组和疗效不佳组，对两组患儿的基线资料进行统计分析，找出危险因素。结果：疗效不佳组患儿出生体重更低、开始用药时月龄更大、初始治疗剂量更低、确诊时FT3、FT4水平更低、TSH水平更高、HGB水平更低、伴有甲状腺形态异常的比例更高，合并缺铁性贫血和维生素D缺乏的比例更高(P均<0.05)。进一步的多因素回归分析显示，开始用药时月龄大、初始治疗剂量低、确诊时FT4水平低、伴有甲状腺形态异常，合并缺铁性贫血和维生素D缺乏等均为影响L-T4治疗效果的独立危险因素(P均<0.05)。结论：开始用药时月龄、初始治疗剂量、确诊时FT4水平、伴有甲状腺形态异常，合并缺铁性贫血和维生素D缺乏等均为影响L-T4治疗效果的影响因素，临床实践中需通过尽早启动L-T4治疗、设定充足的初始剂量并根据患儿情况动态调整剂量、纠正缺铁和维生素D缺乏等措施，以进一步提升CH患儿的治疗效果。

Abstract: Objective: To investigate the factors influencing the efficacy of levothyroxine (L-T4) treatment in children with congenital hypothyroidism (CH) and to propose individualized L-T4 dosing strategies based on these factors. Methods: A total of 109 children with CH diagnosed and receiving standardized L-T4 treatment in the pediatric endocrinology outpatient department of our hospital from January 2021 to December 2024 were selected as the study subjects. Clinical data and auxiliary examination results were collected in detail. The children were divided into a good efficacy group and a poor efficacy group based on treatment outcomes. The baseline data of the two groups were statistically analyzed to identify risk factors. Results: The poor efficacy group had significantly lower birth weight, older age at medication initiation, lower initial treatment dose, lower FT3 and FT4 levels at diagnosis, higher TSH levels at diagnosis, lower HGB levels, a higher proportion with thyroid morphological abnormalities, and a higher proportion comorbid with iron deficiency anemia and vitamin D deficiency (all P < 0.05). Further multivariate regression analysis showed that older age at medication initiation, low initial treatment dose, low FT4 level at diagnosis, presence of thyroid morphological abnormalities, and comorbidity with iron deficiency anemia and vitamin D deficiency were all independent risk factors affecting the efficacy of L-T4 treatment (all P < 0.05). Conclusion: Age at medication initiation, initial treatment dose, FT4 level at diagnosis, presence of thyroid morphological abnormalities, and comorbidity with iron deficiency anemia and vitamin D deficiency are all factors influencing the efficacy of L-T4 treatment. In clinical practice, it is necessary to further improve the treatment efficacy for children with CH by initiating L-T4 treatment as early as possible, setting adequate initial doses and dynamically adjusting the dose based on the child’s condition, and correcting iron deficiency and vitamin D deficiency.