关键词: 生长激素缺乏症, 重组人生长激素, 临床疗效, 列线图

Abstract:

Objective　To investigate the clinical efficacy of recombinant human growth hormone in treating children with growth hormone deficiency (GHD), and to predict treatment outcomes using the nomogram model. Methods　A total of 86 children with GHD who were admitted to Shangluo Central Hospital from February 2020 to February 2022 were selected as the study subjects. All the children received recombinant human growth hormone treatment. After the treatment, they were followed up every 3 months for 1 year. Based on the follow-up results, the children were divided into the ineffective group (17 cases) and the effective group (69 cases). The clinical data of the patients were collected, including general information (age, gender, body mass index, bone age index, height standard deviation score, region, guardian's educational level, family economic status, sleep duration) and laboratory indicators [frozen-thawed follicle stimulating hormone (FSH), estradiol (E2), testosterone (T), luteinizing hormone (LH), insulin-like growth factor-1 (IGF-1), fasting blood glucose (FBG), bone alkaline phosphatase (BAP), free triiodothyronine (FT3), free thyroxine (FT4) levels, and FT3/FT4 before treatment]. Independent sample t test and χ² test were used for statistical analysis. Multivariate logistic regression analysis was used to identify the influencing factors for the ineffective treatment of children with GHD; the R3.4.3 software was employed to construct a nomogram model based on the results of the multivariate logistic regression analysis; calibration curves and Hosmer-Lemeshow tests were used to assess the calibration degree of the model; the receiver operating characteristic curve was used to analyze the predictive efficacy of the model for the ineffective treatment of children with GHD, and the area under the curve was calculated. Results　A total of 86 children with GHD were included in this study, and 17 of them (19.77%) were ineffective. The effective group had higher values for bone age index, height standard deviation score, and BAP level than the ineffective group (all P<0.05). The results of the multivariate logistic regression analysis showed that the bone age index (OR=0.114, 95%CI: 0.021-0.620), height standard deviation score (OR=0.019, 95%CI: 0.002-0.185), and BAP level (OR=0.070, 95%CI: 0.009-0.544) were all factors influencing the ineffectiveness of treatment in children with GHD (all P<0.05). Using bone age index, height standard deviation score, and BAP level as predictive variables, a nomogram model was constructed. The calibration and goodness-of-fit of this model were both good. The receiver operating characteristic curve showed that the area under the curve of this model was 0.972 (95%CI: 0.931-1.000), with a sensitivity of 94.1% and a specificity of 97.1%. Conclusion　Bone age index, height standard deviation score, and BAP level are all factors that affect the therapeutic effect of recombinant human growth hormone in children with GHD. The nomogram model can effectively predict the therapeutic effect and is helpful for guiding clinical practice.

Key words: Growth hormone deficiency,  , Recombinant human growth hormone,  , Clinical efficacy,  , Nomogram