中国全科医学2026，Vol.29Issue(6)：733-740,8.DOI:10.12114/j.issn.1007-9572.2024.0709
生长激素缺乏症的临床特征与重组人生长激素疗效分析:一项十年纵向队列研究
Clinical Characteristics of Growth Hormone Deficiency and Analysis of the Efficacy of Recombinant Human Growth Hormone:a Ten-year Longitudinal Cohort Study
摘要
Abstract
Background Growth hormone deficiency(GHD)can lead to growth and development disorders in children.Its clinical features are complex and diverse,and diagnosis and treatment are difficult.Recombinant human growth hormone(rhGH)is the first-choice drug for treating GHD,but there are significant individual differences in treatment efficacy,and the influencing factors are not yet clear.Objective Based on a ten-year longitudinal cohort study,to investigate the clinical characteristics of children with GHD and the efficacy of rhGH therapy.Methods A total of 1 050 children who were hospitalized in the Department of Endocrinology,Genetics and Metabolism,Affiliated Hospital of Jining Medical University from 2013 to 2022 and diagnosed with GHD were selected as the study subjects.The general clinical characteristics of children with GHD and the treatment effects of rhGH were analyzed.Multivariate logistic regression analyzed factors affecting first-year rhGH treatment effects.Results Among the 1 050 children with GHD,there were 737(70.19%)boys and 313(29.81%)girls.The average age was(10.3±3.5)years,the average bone age was(8.4±3.6)years,the average height standard deviation score(SDS)was(-2.64±0.63),the median insulin-like growth factor 1(IGF-1)SDS was-1.06(-1.79 to-0.28),the median peak growth hormone level was 5.51(3.64 to 7.28)ng/mL,and most(72.76%,764 cases)of the children were prepubertal.Compared with children who had not received rhGH treatment,children who received rhGH treatment had a younger average age,a higher average height SDS,and a lower average genetic target height,and the differences were statistically significant(P<0.05).rhGH treatment can significantly improve the height SDS in children with GHD.After 0.5 years of rhGH treatment,there was a significant improvement in the height SDS of the children(β=0.34,95%CI=0.29 to 0.39,P<0.001).As the treatment duration increased,the maximum improvement was achieved after 8 years of rhGH treatment(β=2.28,95%CI=1.84 to 2.72,P<0.001).Age(OR=0.84,95%CI=0.78 to 0.91,P<0.001),bone age(OR=0.87,95%CI=0.81 to 0.94,P<0.001),height SDS(OR=0.24,95%CI=0.12 to 0.49,P<0.001),insulin-like growth factor binding protein 3(OR=0.73,95%CI=0.60 to 0.89,P<0.001),and peak growth hormone level(OR=0.98,95%CI=0.95 to 0.99,P=0.004)had a negative impact on the efficacy of rhGH treatment.Genetic target height SDS(OR=1.22,95%CI=1.02 to 1.46,P=0.032),genetic target height SDS-height SDS(OR=1.34,95%CI=1.13 to 1.59,P=0.001),and IGF-1(OR=1.01,95%CI=1.01 to 1.22,P=0.019)had a positive impact on the efficacy of rhGH treatment.Conclusion This study revealed that children with GHD exhibited delayed bone age and low levels of IGF-1.rhGH significantly improves height in children with GHD,with more favorable outcomes observed in longer the treatment duration,younger the chronological age and bone age,the lower the baseline height SDS,the lower the insulin-like growth factor binding protein 3 level with lower peak growth hormone levels and higher genetic target heights.Early diagnosis,timely intervention,and appropriate treatment are essential for children with severe GHD to enhance their height and overall quality of life.关键词
生长激素缺乏症/重组人生长激素/身材矮小/治疗效果/队列研究Key words
Growth hormone deficiency/Recombinant human growth hormone/Short stature/Therapeutic effect/Cohort study分类
医药卫生引用本文复制引用
赵倩倩,张梅,李艳英,张艳红,晏文华,潘慧,班博..生长激素缺乏症的临床特征与重组人生长激素疗效分析:一项十年纵向队列研究[J].中国全科医学,2026,29(6):733-740,8.基金项目
山东省自然科学基金资助项目(ZR2022MH284) （ZR2022MH284）
济宁医学院附属医院博士后研究项目(JYFY399354) （JYFY399354）
