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中国儿童永久性与暂时性先天性甲状腺功能减退症:体格生长与预测列线图。

Permanent vs Transient Congenital Hypothyroidism in Chinese Children: Physical Growth and Predictive Nomogram.

机构信息

Changzhou Medical Center, Changzhou Maternal and Child Health Care Hospital, Nanjing Medical University, Changzhou 213000, China.

出版信息

J Clin Endocrinol Metab. 2024 Jul 12;109(8):e1616-e1622. doi: 10.1210/clinem/dgad739.

Abstract

CONTEXT

Few reliable markers are available to distinguish transient congenital hypothyroidism (TCH) and permanent congenital hypothyroidism (PCH). Additionally, the differences in growth between TCH and PCH remain unclear.

OBJECTIVE

To investigate the growth of children with TCH and PCH and develop a nomogram for early differentiation of these forms.

METHODS

This retrospective study included children with TCH or PCH. The predictive efficacy of the prognostic predictors was analyzed using receiver operating characteristic analysis. Multivariate prediction models were developed. Measurements of growth were compared between groups.

RESULTS

Patients with TCH had lower initial thyroid-stimulating hormone (TSH) than those with PCH at newborn screening (NBS). The supplementary dose of levothyroxine (L-T4) gradually decreased with age in TCH but not in PCH. The area under the curve (AUC) values of the initial TSH, L-T4 dose at 1 year of age, and L-T4 dose at 2 years of age for distinguishing TCH from PCH were 0.698, 0.71, and 0.879, respectively. The predictive efficacy of the multivariate models at 1 and 2 years of age improved, with AUC values of 0.752 and 0.922, respectively. A nomogram was built based on the multivariate model at 1 year of age. The growth did not differ between children with TCH and those with PCH. However, at 1 year of age, girls with CH exhibited higher z-scores in terms of height and weight than boys with CH.

CONCLUSION

TSH at NBS and L-T4 doses during treatment can be used to distinguish between PCH and TCH early in life, and the predictive efficacy can be improved using multivariable models with a visualized nomogram. At 3 years of age, patients with TCH and PCH showed similar growth.

摘要

背景

目前,用于鉴别暂时性先天性甲状腺功能减退症(TCH)和永久性先天性甲状腺功能减退症(PCH)的可靠标志物较少。此外,TCH 和 PCH 之间的生长差异仍不清楚。

目的

研究 TCH 和 PCH 患儿的生长情况,并制定用于早期鉴别这两种疾病的列线图。

方法

本回顾性研究纳入了 TCH 或 PCH 患儿。采用受试者工作特征分析(ROC 分析)评估预后预测因子的预测效能,建立多变量预测模型。比较各组间的生长指标。

结果

新生儿筛查(NBS)时,TCH 患儿的初始促甲状腺激素(TSH)水平低于 PCH 患儿。TCH 患儿的左甲状腺素(L-T4)补充剂量随年龄逐渐减少,但 PCH 患儿的 L-T4 剂量无此变化。鉴别 TCH 和 PCH 时,初始 TSH、1 岁时 L-T4 剂量和 2 岁时 L-T4 剂量的曲线下面积(AUC)值分别为 0.698、0.71 和 0.879。1 岁和 2 岁时多变量模型的预测效能提高,AUC 值分别为 0.752 和 0.922。基于 1 岁时的多变量模型构建了列线图。TCH 患儿与 PCH 患儿的生长情况无差异。但在 1 岁时,患有 CH 的女孩身高和体重的 z 评分均高于患有 CH 的男孩。

结论

NBS 时的 TSH 和治疗期间的 L-T4 剂量可用于早期鉴别 PCH 和 TCH,多变量模型和可视化列线图可提高预测效能。3 岁时,TCH 和 PCH 患儿的生长情况相似。

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