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预测特发性中枢性性早熟女童成年身高模型的建立与验证

Development and validation of a model for predicting the adult height of girls with idiopathic central precocious puberty.

作者信息

Wu Wenyong, Zhu Xiaoyun, Chen Yun, Yang Xiaohong, Zhang Ying, Chen Ruimin

机构信息

Fuzhou Children's Hospital of Fujian Medical University, Fuzhou, Fujian, China.

出版信息

Eur J Pediatr. 2023 Apr;182(4):1627-1635. doi: 10.1007/s00431-023-04842-3. Epub 2023 Jan 28.

Abstract

UNLABELLED

The aim was to develop a model to predict the adult height (AH) of idiopathic central precocious puberty (ICPP) girls who underwent gonadotropin-releasing hormone analog (GnRHa) treatment. Data analysis included 258 girls with ICPP. Among them, 101 girls who reached final AH (FAH) with GnRHa treatment were analyzed to verify three previous prediction models and develop a unique model based on multiple linear regression. The control group consisted of 41 untreated ICPP girls. Moreover, 116 girls treated with GnRHa who almost attained FAH were included for external validation. Based on our cohorts, all of the three previously published models underestimated the FAH with an R of 0.667, 0.793, and 0.664. The AH prediction model was built as follows: Calculated AH (cm) = 1.89630 * Height SDS + 2.29927 * Height SDS for bone age + 0.40776 * Target height + 100.16684 (R = 0.66 and adjusted R = 0.65). Internal validation showed a mean root mean squared error (RMSE) of 2.16 cm and a mean absolute error (MAE) of 1.64 cm. External validation showed that a significant error (> 1 SD) appeared only in 7 of 116 girls (6.0%). The model is displayed on the website: http://cpppredict.shinyapps.io/dynnomapp .

CONCLUSION

A model for predicting the AH of girls with ICPP was developed incorporating the variables of height SDS, height SDS for bone age, and target height. The internal and external validation ensures an appropriate degree of discrimination and calibration of the prediction model.

WHAT IS KNOWN

• Uncertainty prevails as how to predict the adult height of patients with central precocious puberty following gonadotropin-releasing hormone analog treatment. • Previous models for predicting adult height of girls with idiopathic central precocious puberty have not been proven translational to the Chinese population.

WHAT IS NEW

• This study develops a new model for predicting the adult height of idiopathic central precocious puberty girls who underwent gonadotropin-releasing hormone analog treatment. • The internal and external validation assures a good degree of discrimination and calibration of the prediction model in this study.

摘要

未标注

目的是建立一个模型,以预测接受促性腺激素释放激素类似物(GnRHa)治疗的特发性中枢性性早熟(ICPP)女孩的成年身高(AH)。数据分析纳入了258例ICPP女孩。其中,对101例经GnRHa治疗达到最终成年身高(FAH)的女孩进行分析,以验证之前的三个预测模型,并基于多元线性回归建立一个独特的模型。对照组由41例未经治疗的ICPP女孩组成。此外,纳入116例接受GnRHa治疗且几乎达到FAH的女孩进行外部验证。基于我们的队列研究,之前发表的三个模型均低估了FAH,其R值分别为0.667、0.793和0.664。成年身高预测模型构建如下:计算成年身高(厘米)=1.89630×身高标准差(SDS)+2.29927×骨龄身高SDS+0.40776×靶身高+100.16684(R=0.66,调整后R=0.65)。内部验证显示平均均方根误差(RMSE)为2.16厘米,平均绝对误差(MAE)为1.64厘米。外部验证显示,116例女孩中只有7例(6.0%)出现显著误差(>1个标准差)。该模型可在网站查看:http://cpppredict.shinyapps.io/dynnomapp

结论

建立了一个预测ICPP女孩成年身高的模型,纳入了身高SDS、骨龄身高SDS和靶身高变量。内部和外部验证确保了预测模型具有适当程度的区分度和校准度。

已知信息

•对于如何预测促性腺激素释放激素类似物治疗后中枢性性早熟患者的成年身高,目前仍存在不确定性。•之前用于预测特发性中枢性性早熟女孩成年身高的模型尚未被证明适用于中国人群。

新发现

•本研究建立了一个新模型,用于预测接受促性腺激素释放激素类似物治疗的特发性中枢性性早熟女孩的成年身高。•内部和外部验证确保了本研究中预测模型具有良好的区分度和校准度。

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