Depth of Public Health and Pediatric Sciences, University of Turin, Turin, Italy.
Depth of Neurosciences, University of Turin, Turin, Italy.
J Endocrinol Invest. 2018 Jul;41(7):839-848. doi: 10.1007/s40618-017-0816-6. Epub 2018 Jan 9.
Recombinant GH has been offered to GH-deficient (GHD) subjects for more than 30 years, in order to improve height and growth velocity in children and to enhance metabolic effects in adults.
The aim of our work is to describe the long-term effect of rhGH treatment in GHD pediatric patients, suggesting a growth prediction model.
A homogeneous database is defined for diagnosis and treatment modalities, based on GHD patients afferent to Hospital Regina Margherita in Turin (Italy). In this study, 232 GHD patients are selected (204 idiopathic GHD and 28 organic GHD). Each measure is shown in terms of mean with relative standard deviations (SD) and 95% confidence interval (95% CI). To estimate the final height of each patient on the basis of few measures, a mathematical growth prediction model [based on Gompertzian function and a mixed method based on the radial basis functions (RBFs) and the particle swarm optimization (PSO) models] was performed.
The results seem to highlight the benefits of an early start of treatment, further confirming what is suggested by the literature. Generally, the RBF-PSO method shows a good reliability in the prediction of the final height. Indeed, RMSE is always lower than 4, i.e., in average the forecast will differ at most of 4 cm to the real value.
In conclusion, the large and accurate database of Italian GHD patients allowed us to assess the rhGH treatment efficacy and compare the results with those obtained in other Countries. Moreover, we proposed and validated a new mathematical model forecasting the expected final height after therapy which was validated on our cohort.
重组人生长激素（rhGH）已在生长激素缺乏症（GHD）患者中使用超过 30 年，以改善儿童的身高和生长速度，并增强成人的代谢作用。
我们的工作旨在描述 rhGH 治疗 GHD 儿科患者的长期效果，并提出一个生长预测模型。
基于都灵 Regina Margherita 医院（意大利）收治的 GHD 患者，定义了一个用于诊断和治疗方式的同质数据库。在这项研究中，选择了 232 名 GHD 患者（204 名特发性 GHD 和 28 名器质性 GHD）。每个指标均以平均值表示，并给出了相对标准差（SD）和 95%置信区间（95%CI）。为了根据少数几个指标估计每位患者的最终身高，我们进行了一个数学生长预测模型（基于 Gompertz 函数和一种基于径向基函数（RBF）和粒子群优化（PSO）模型的混合方法）。
结果似乎突出了早期治疗的益处，进一步证实了文献中的建议。通常，RBF-PSO 方法在预测最终身高方面具有良好的可靠性。实际上，RMSE 始终低于 4，即在大多数情况下，预测值与真实值的差异最多为 4 厘米。
总之，意大利 GHD 患者的大型和准确数据库使我们能够评估 rhGH 治疗的疗效，并将结果与其他国家的结果进行比较。此外，我们提出并验证了一种新的数学模型，可以预测治疗后的预期最终身高，并在我们的队列中进行了验证。
