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孤立性儿童生长激素缺乏症:30 年最终身高的经验及新的预测模型。

Isolated childhood growth hormone deficiency: a 30-year experience on final height and a new prediction model.

机构信息

Giovanni XXIII Children's Hospital, AOU Policlinico di Bari, piazza G.Cesare 11, Bari, Italy.

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

出版信息

J Endocrinol Invest. 2022 Sep;45(9):1709-1717. doi: 10.1007/s40618-022-01808-4. Epub 2022 May 14.

Abstract

PURPOSE

We aimed to evaluate the near-final height (nFHt) in a large cohort of pediatricpatients with growth hormone deficiency (GHD) and to elaborate a new predictive method of nFHt.

METHODS

We recruited GHD patients diagnosed between 1987 and 2014 and followed-up until nFHt. To predict the values of nFHt, each predictor was run in a univariable spline.

RESULTS

We enrolled 1051 patients. Pre-treatment height was -2.43 SDS, lower than parental height (THt) (-1.09 SDS, p < 0.001). The dose of recombinant human GH (rhGH) was 0.21mg/kg/week at start of treatment. nFHt was -1.08 SDS (height gain 1.27 SDS), higher than pre-treatment height (p < 0.001) and comparable to THt. 1.6% of the patients were shorter than -2 SDS from THt. The rhGH dose at nFHt was 0.19 mg/kg/week, lower than at the start (p < 0.001). The polynomial regression showed that nFHt was affected by gender, THt, age at puberty, height at puberty, age at the end of treatment (F = 325.37, p < 0.0001, R 87.2%).

CONCLUSION

This large national study shows that GHD children can reach their THt. The rhGH/kg/day dose significantly decreased from the start to the end of the treatment. Our model suggests the importance of a timely diagnosis, possibly before puberty, the beneficial effect of long-term treatment with rhGH, and the key-role of THt. Our prediction model has a very acceptable error compared to the majority of other published studies.

摘要

目的

我们旨在评估大量生长激素缺乏症(GHD)儿科患者的最终身高(nFHt),并提出一种新的 nFHt 预测方法。

方法

我们招募了 1987 年至 2014 年间诊断为 GHD 并随访至 nFHt 的患者。为了预测 nFHt 的值,每个预测因子在单变量样条中运行。

结果

我们纳入了 1051 名患者。治疗前身高为-2.43 SDS,低于父母身高(THt)(-1.09 SDS,p <0.001)。治疗开始时,重组人生长激素(rhGH)的剂量为 0.21mg/kg/周。nFHt 为-1.08 SDS(身高增长 1.27 SDS),高于治疗前身高(p <0.001),与 THt 相当。有 1.6%的患者身高比 THt 低-2 SDS。nFHt 时 rhGH 剂量为 0.19 mg/kg/周,低于治疗开始时(p <0.001)。多项式回归显示,nFHt 受性别、THt、青春期年龄、青春期身高、治疗结束年龄的影响(F = 325.37,p <0.0001,R 87.2%)。

结论

这项大型全国性研究表明,GHD 儿童可以达到他们的 THt。rhGH/kg/天的剂量从治疗开始到结束显著下降。我们的模型表明,及时诊断(可能在青春期之前)、rhGH 长期治疗的有益作用以及 THt 的关键作用非常重要。与大多数其他已发表的研究相比,我们的预测模型误差非常小。

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