重组人生长激素治疗特发性矮小患儿的临床研究  

Clinical trial of recombinant human growth hormone in the treatment of children with idiopathic short stature

作　　者：张东光[1] 杨玉[1] 杨利[1] 邹海英[1] 胡云[1] ZHANG Dong-guang;YANG Yu;YANG Li;ZOU Hai-ying;HU Yun(Department of Endocrinology,Metabolism and Genetics,Jiangxi Provincial Children's Hospital,Nanchang 330038,Jiangxi Province,China)

机构地区：[1]江西省儿童医院内分泌遗传代谢科,江西南昌330038

出　　处：《中国临床药理学杂志》2025年第2期169-173,共5页The Chinese Journal of Clinical Pharmacology

摘　　要：目的观察重组人生长激素(rhGH)注射液治疗特发性矮小(ISS)患儿的临床疗效和安全性,及其对血清胰岛素样生长因子-1(IGF-1)、碱性磷酸酶(AKP)、胰岛素样生长因子结合蛋白-3(IGFBP-3)水平的影响。方法将ISS患儿随机分为对照组和试验组。对照组皮下注射0.15 U·kg^(-1)rhGH注射液,试验组皮下注射0.20 U·kg^(-1)rhGH注射液。2组患儿均连续治疗6个月。比较2组患儿的临床疗效,生长情况[生长速率、身高标准差积分(HtSDS)、预测成年身高(PAH)],骨代谢指标{AKP、骨钙素(OC)、25-羟基维生素D[25(OH)D]}水平及血清IGF-1、IGFBP3水平变化,并评价安全性。结果试验组入组46例,脱落6例,最终有40例纳入统计分析;对照组入组46例,脱落1例,最终有45例纳入统计分析。治疗6个月后,试验组和对照组的总有效率分别为85.00%(34例/40例)和64.44%(29例/45例),在统计学上差异有统计学意义(P<0.05)。治疗6个月后,试验组和对照组的生长速率分别为(7.96±1.62)和(6.84±1.56)cm·year^(-1),HtSDS分别为-2.38±0.24和-2.61±0.28,PAH分别为(156.86±4.18)和(155.02±4.25)cm,血清AKP水平分别为(278.42±47.46)和(257.14±42.79)U·L^(-1),OC水平分别为(76.92±10.17)和(72.43±10.32)μg·L^(-1),25(OH)D水平分别为(59.96±4.74)和(55.52±4.69)nmol·L^(-1),血清IGF-1水平分别为(296.77±28.32)和(251.47±24.96)ng·mL^(-1),血清IGFBP3水平分别为(5.76±1.22)和(4.86±0.89)μg·mL^(-1),试验组的上述指标与对照组比较,在统计学上差异均有统计学意义(均P<0.05)。2组患者的药物不良反应均以头痛、皮疹、关节痛为主。试验组和对照组的总药物不良反应发生率分别为12.50%(5例/40例)和6.67%(3例/45例),在统计学上差异无统计学意义(P>0.05)。结论rhGH注射液治疗ISS患儿的临床疗效确切,且0.20 U·kg^(-1)rhGH改善IGF-1、AKP、IGFBP3水平均显著优于0.15 U·kg^(-1)rhGH,更有利于促进患儿的骨骼发育、加快生长,且不增加药物不良反应Objective To observe the clinical efficacy and safety of recombinant human growth hormone injection(rhGH)in the treatment of children with idiopathic short stature(ISS)and its influence on levels of insulin-like growth factor 1(IGF-1),alkaline phosphatase(AKP)and insulin-like growth factor binding protein 3(IGFBP-3).Methods Children with ISS were randomized into control group and treatmentgroup.The control group was subcutaneously injected with 0.15 U·kg^(-1)of rhGH injection,while the treatment group was given subcutaneous injection of 0.20 U·kg^(-1)of rhGH injection,and both groups were continuously treated for 6 months.The clinical efficacy,growth status[growth velocity,height standard deviation score(HtSDS),predicted adultheight(PAH)],bonemetabolismindicators AKP,osteocalcin(OC),25-hydroxyvitamin D[25(OH)D]and serum IGF-1 and IGFBP3 levels were compared,and the safety was assessed.Results In the treatment group,46 cases were enrolled,6 cases were lost,and 40 cases were finally included in the statistical analysis.In the control group,46 cases were enrolled,1 case was lost,and 45 cases were finally included in the statistical analysis.The total effective rates in treatment group and control group after 6 months treatment were 85.00%(34 cases/40 cases)and 64.44%(29 cases/45 cases),respectively,with a statistical significance(P<0.05).After 6 months treatment,the growth rates in treatment group and control group were(7.96±1.62)and(6.84±1.56)cm·year^(-1);HtSDS values were-2.38±0.24 and-2.61±0.28;PAH values were(156.86±4.18)and(155.02±4.25)cm;serum AKP levels were(278.42±47.46)and(257.14±42.79)U·L^(-1);0C levels were(76.92±10.17)and(72.43±10.32)μg·L^(-1);25(0H)D levels were(59.96±4.74)and(55.52±4.69)nmol L^(-1);serum ICF-1 levels were(296.77±28.32)and(251.47±24.96)ng·mL^(-1);serum IGFBP3 levels were(5.76±1.22)and(4.86±0.89)μg·mL^(-1),respectively.Compared with the control group,the above indexes in the treatment group were statistically significant(all P<0.05).The adverse drug reactions in

关 键 词：重组人生长激素注射液 特发性矮小 胰岛素样生长因子-1 碱性磷酸酶 胰岛素样生长因子结合蛋白-3 

分 类 号：R977.1[医药卫生—药品]