PEG-rhGH治疗特发性矮小症患儿对生长速率影响的临床研究  

Clinical trial of PEG-rhGH in the treatment of children with idiopathic short stature

作　　者：王忻 邓茜 李敏 王娟娟 WANG Xin;DENG Qian;LI Min;WANG Juan-juan(Department of Endocrinology and Metabolism,Anhui Provincial Children's Hospital,Hefei 23001l,Anhui Province,China)

机构地区：[1]安徽省儿童医院内分泌代谢科,安徽合肥230011

出　　处：《中国临床药理学杂志》2024年第18期2685-2689,共5页The Chinese Journal of Clinical Pharmacology

摘　　要：目的探讨特发性矮小症(ISS)患儿聚乙二醇重组人生长激素(PEGrhGH)治疗后生长速率(GV),分析GV影响因素。方法ISS患儿均先后接受PEG-rhGH治疗,每周0.16~0.17mg·kg^(-1),皮下注射,注射部位为脐周、上臂外侧、大腿外侧,持续治疗1年,观察患儿治疗前后生长参数差异,记录治疗后患儿GV水平,通过多元逐步线性回归分析患儿治疗后1年GV水平影响因素。结果ISS患儿治疗前及治疗1年后身高(Ht)分别为(109.51±12.59)和(123.16±13.07)cm,身高标准差积分(HtSDS)水平分别为-2.24±0.25和-1.71±0.21,GV水平分别为(3.81±0.52)和(9.88±1.04)cm·year^(-1),25羟维生素D水平分别为(27.19±3.14)和(33.05±3.46)ng·mL^(-1),Ⅰ型前胶原氨基端原肽(PINP)水平分别为(490.29±54.30)和(598.45±57.18)μg·L^(-1),胰岛素生长因子-Ⅰ(IGF-1)水平分别为(114.86±19.14)和(213.73±20.03)ng·mL^(-1),胰岛素样生长因子结合蛋白-3(IGFBP-3)水平分别为(6817.27±716.30)和(7230.39±721.45)ng·mL^(-1),治疗前上述指标与治疗组后比较,在统计学上差异均有统计学意义(均P<0.05)。ISS患儿治疗前实际年龄(CA)为(8.05±1.09)岁,治疗前骨龄(BA)为(7.14±1.01)岁,治疗前骨龄差(BAD)为(-0.98±0.18)岁,治疗前骨龄指数(BAI)为0.86±0.12,遗传身高(MPH)为(167.31±5.73)cm。Pearson相关性分析显示ISS患儿治疗后GV水平与治疗前CA、治疗前BA、治疗前BAI呈负相关,与治疗前BAD、MPH呈正相关(均P<0.05);多元逐步线性回归分析结果显示治疗前高CA、BA、BAI水平为患儿PEG-rhGH治疗后低GV水平的危险因素,治疗前高BAD及MPH水平为保护因素。结论ISS患儿PEG-rhGH治疗开始时年龄小、骨龄小者可获得较佳的GV应答,且遗传靶身高可影响患儿治疗后GV水平。Objective To explore growth velocity(GV)and analyze its influencing factors in children with idiopathic short stature(ISS)after treatment with polyethylene glycol recombinant human growth hormone(PEG-rhGH).Methods The clinical data of children with ISS were retrospectively analyzed.All children were given subcutaneous injection of PEG-rhGH(0.16-0.17 mg·kg^(-1)each week)at periumbilical,lateral upper arm and thigh sites for 1 year.The differences in growth parameters before and after treatment were observed,and GV after treatment was recorded.The influencing factors of GV at 1 year after treatment were analyzed by multivariate stepwise linear regression analysis.Results Before and after 1 year of treatment,height(Ht)was(109.51±12.59)and(123.16±13.07)cm,height standard deviation scores(HtSDS)were-2.24±0.25 and-1.71±0.21,GV levels were(3.81±0.52)and(9.88±1.04)cm·year^(-1),levels of 25-hydroxy-vitamin-D[25(OH)D]were(27.19±3.14)and(33.05±3.46)ng·mL^(-1),levels of propeptide of type I procollagen(PINP)were(490.29±54.30)and(598.45±57.18)μg·L-1,levels of insulin-like growth factor-1(IGF-1)were(114.86±19.14)and(213.73±20.03)ng·mL^(-1),levels of insulin-like growth factor binding protein-3(IGFBP-3)were(6817.27±716.30)and(7230.39±721.45)ng·mL^(-1),the differences were statistically significant(all P<0.05).Before treatment,chronological age(CA),bone age(BA),bone age delay(BAD),bone age index(BAI)and mid-parental height(MPH)were(8.05±1.09)years,(7.14±1.01)years,(-0.98±0.18)years,0.86±0.12 and(167.31±5.73)cm,respectively.Pearson correlation analysis showed that GV level after treatment was negatively correlated with CA,BA and BAI before treatment,while positively correlated with BAD and MPH before treatment(P<0.05).The results of multivariate stepwise linear regression analysis showed that high levels of CA,BA and BAI before treatment were risk factors of low GV level after PEG-rhGH treatment,while high levels of BAD and MPH before treatment were protective factors.Conclusion GV response is good in IS

关 键 词：聚乙二醇重组人生长激素 特发性矮小症 生长速率 骨龄 

分 类 号：R977[医药卫生—药品]