生长激素治疗儿童特发性矮小症疗效分析
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摘要
目的:探讨生长激素治疗儿童特发性矮小症(ISS)对成年终身高的改善情况。方法:回顾性分析已确诊为ISS并使用生长激素治疗,且目前已达到成年终身高的86例患儿,其中男50例,女36例。根据用药时间分为长疗程组(≥1年) 65例和短疗程组(<1年) 21例。观察患儿身高标准差积分(HSDS)、基线身高标准差积分(BHSDS)、遗传靶身高(TH)、遗传靶身高标准差积分(THSDS)、接近成年终身高(NAH)、接近成年终身高标准差积分(NAHSDS)等指标。结果:长疗程组患儿NAHSDS大于短疗程组(P<0. 05);长疗程组患儿NAHSDS大于THSDS(P<0. 05);短疗程组患儿NAHSDS小于THSDS(P<0. 05); 75. 6%(65/86)的患儿最终身高达到正常身高,其中长疗程组中95. 2%患儿最终身高达到正常身高。结论:使用生长激素治疗ISS患儿,长疗程(≥1年)较适宜。
Objective: To probe into the improvement of growth hormone on near-adult height of children with idiopathic short stature( ISS). Methods: Retrospective analysis was performed on 86 children diagnosed with ISS and treated with growth hormone,who had reached the near-adult height,including 50 males and 36 females.According to the duration of medication,there were 65 patients in the long course group( ≥1 year) and 21 patients in the short course group( <1 year).Height standard deviation scores( HSDS),baseline height standard deviation scores( BHSDS),genetic target height( TH),genetic target height standard deviation scores( THSDS),near-adult height( NAH),near-adult height standard deviation scores( NAHSDS) and other indicators of two groups were observed.Results: NAHSDS of the long course group was greater than that of the short course group( P<0. 05).NAHSDS was greater than THSDS in the long course group( P<0. 05).NAHSDS of short course group was less than THSDS( P<0. 05).A total of 75. 6%( 65/86) of the children eventually reached the normal height,and 95. 2% of the children in the long treatment group eventually reached the normal height.Conclusion: To set the course of treatment ≥1 year in children with ISS with growth hormone therapy is suitable.
Objective: To probe into the improvement of growth hormone on near-adult height of children with idiopathic short stature( ISS). Methods: Retrospective analysis was performed on 86 children diagnosed with ISS and treated with growth hormone,who had reached the near-adult height,including 50 males and 36 females.According to the duration of medication,there were 65 patients in the long course group( ≥1 year) and 21 patients in the short course group( <1 year).Height standard deviation scores( HSDS),baseline height standard deviation scores( BHSDS),genetic target height( TH),genetic target height standard deviation scores( THSDS),near-adult height( NAH),near-adult height standard deviation scores( NAHSDS) and other indicators of two groups were observed.Results: NAHSDS of the long course group was greater than that of the short course group( P<0. 05).NAHSDS was greater than THSDS in the long course group( P<0. 05).NAHSDS of short course group was less than THSDS( P<0. 05).A total of 75. 6%( 65/86) of the children eventually reached the normal height,and 95. 2% of the children in the long treatment group eventually reached the normal height.Conclusion: To set the course of treatment ≥1 year in children with ISS with growth hormone therapy is suitable.
引文
[1]LEE M M.Clinical practice.Idiopathic short stature[J].N Engl J Med,2006,354(24):2576-2582.
[2]COHEN P,ROGOL A D,DEAL C L,et al.Consensus statement on the diagnosis and treatment of children with idiopathic short stature:A summary of the Growth Hormone Research Society,the Lawson Wilkins Pediatric Endocrine Society,and the European Society for Paediatric Endocrinology Workshop[J].J Clin Endocrinol Metab,2008,93(11):4210-4217.
[3]SCHENA L,MEAZZA C,PAGANI S,et al.Efficacy of longterm growth hormone therapy in short non-growth hormonedeficient children[J].J Pediatr Endocrinol Metab,2017,30(2):197-201.
[4]TOPOR L S,FELDMAN H A,BAUCHNER H,et al.Variation in methods of predicting adult height for children with idiopathic short stature[J].Pediatrics,2010,126(5):938-944.
[5]应艳琴,罗小平.生长激素在儿科临床的应用---现状与循证[J].临床儿科杂志,2005(8):5-9.
[6]张雪霜,熊丰,朱岷,等.重组人生长激素治疗不同病因矮小症患儿效果观察[J].第三军医大学学报,2016,38(16):1889-1894.
[7]陈立黎,朱高慧,熊丰,等.重组人生长激素治疗儿童特发性矮小症远期疗效分析[J].儿科药学杂志,2018,24(6):13-16.
[8]MCCAUGHEY E S,MULLIGAN J,VOSS L D,et al.Randomised trial of growth hormone in short normal girls[J].Lancet,1998,351(9107):940-944.
[9]LESCHEKE W,ROSE S R,YANOVSKI J A,et al.Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature:A randomized,double-blind,placebo-controlled trial[J].J Clin Endocrinol Metab,2004,89(7):3140-3148.
[10]ALBERTSSON-WIKLAND K,ARONSON A S,GUSTAFSSON J,et al.Dose-dependent effect of growth hormone on final height in children with short stature without growth hormone deficiency[J].J Clin Endocrinol Metab,2008,93(11):4342-4350.
[11]WIT J M,BOERSMA B,DE MUINCK KEIZER-SCHRAMA S M,et al.Long-term results of growth hormone therapy in children with short stature,subnormal growth rate and normal growth hormone response to secretagogues[J].Clin Endocrinol(Oxf),1995,42(4):365-372.
[12]TRAVAGLINO P,BUZI F,MEAZZA C,et al.Response to long-term growth hormone therapy in short children with reduced GH bioactivity[J].Horm Res,2006,66(4):189-194.
[13]COUTANT R,ROULEAU S,DESPERT F,et al.Growth and adult height in GH-treated children with nonacquired GHdeficiency and idiopathic short stature:The influence of pituitary magnetic resonance imaging findings[J].J Clin Endocrinol Metab,2001,86(10):4649-4654.
[14]SOTOS J F,TOKAR N J.Growth hormone significantly increases the adult height of children with idiopathic short stature:Comparison of subgroups and benefit[J].Int J Pediatr Endocrinol,2014,2014(1):15.
[15]矮身材儿童诊治指南[J].中华医学信息导报,2008,23(24):20-21.
[16]吴迪,冯国双,巩纯秀.生长激素治疗特发性矮小儿童随访至接近成年终身高的治疗效果分析[J].首都医科大学学报,2018(1):92-97.
[17]ROSS J L,LEE P A,GUT R,et al.Attaining genetic height potential:Analysis of height outcomes from the ANSWERProgram in children treated with growth hormone over 5 years[J].Growth Horm IGF Res,2015,25(6):286-293.
[18]COHEN P,BRIGHT G M,ROGOL A D,et al.Effects of dose and gender on the growth and growth factor response to GH in GH-deficient children:Implications for efficacy and safety[J].JClin Endocrinol Metab,2002,87(1):90-98
[2]COHEN P,ROGOL A D,DEAL C L,et al.Consensus statement on the diagnosis and treatment of children with idiopathic short stature:A summary of the Growth Hormone Research Society,the Lawson Wilkins Pediatric Endocrine Society,and the European Society for Paediatric Endocrinology Workshop[J].J Clin Endocrinol Metab,2008,93(11):4210-4217.
[3]SCHENA L,MEAZZA C,PAGANI S,et al.Efficacy of longterm growth hormone therapy in short non-growth hormonedeficient children[J].J Pediatr Endocrinol Metab,2017,30(2):197-201.
[4]TOPOR L S,FELDMAN H A,BAUCHNER H,et al.Variation in methods of predicting adult height for children with idiopathic short stature[J].Pediatrics,2010,126(5):938-944.
[5]应艳琴,罗小平.生长激素在儿科临床的应用---现状与循证[J].临床儿科杂志,2005(8):5-9.
[6]张雪霜,熊丰,朱岷,等.重组人生长激素治疗不同病因矮小症患儿效果观察[J].第三军医大学学报,2016,38(16):1889-1894.
[7]陈立黎,朱高慧,熊丰,等.重组人生长激素治疗儿童特发性矮小症远期疗效分析[J].儿科药学杂志,2018,24(6):13-16.
[8]MCCAUGHEY E S,MULLIGAN J,VOSS L D,et al.Randomised trial of growth hormone in short normal girls[J].Lancet,1998,351(9107):940-944.
[9]LESCHEKE W,ROSE S R,YANOVSKI J A,et al.Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature:A randomized,double-blind,placebo-controlled trial[J].J Clin Endocrinol Metab,2004,89(7):3140-3148.
[10]ALBERTSSON-WIKLAND K,ARONSON A S,GUSTAFSSON J,et al.Dose-dependent effect of growth hormone on final height in children with short stature without growth hormone deficiency[J].J Clin Endocrinol Metab,2008,93(11):4342-4350.
[11]WIT J M,BOERSMA B,DE MUINCK KEIZER-SCHRAMA S M,et al.Long-term results of growth hormone therapy in children with short stature,subnormal growth rate and normal growth hormone response to secretagogues[J].Clin Endocrinol(Oxf),1995,42(4):365-372.
[12]TRAVAGLINO P,BUZI F,MEAZZA C,et al.Response to long-term growth hormone therapy in short children with reduced GH bioactivity[J].Horm Res,2006,66(4):189-194.
[13]COUTANT R,ROULEAU S,DESPERT F,et al.Growth and adult height in GH-treated children with nonacquired GHdeficiency and idiopathic short stature:The influence of pituitary magnetic resonance imaging findings[J].J Clin Endocrinol Metab,2001,86(10):4649-4654.
[14]SOTOS J F,TOKAR N J.Growth hormone significantly increases the adult height of children with idiopathic short stature:Comparison of subgroups and benefit[J].Int J Pediatr Endocrinol,2014,2014(1):15.
[15]矮身材儿童诊治指南[J].中华医学信息导报,2008,23(24):20-21.
[16]吴迪,冯国双,巩纯秀.生长激素治疗特发性矮小儿童随访至接近成年终身高的治疗效果分析[J].首都医科大学学报,2018(1):92-97.
[17]ROSS J L,LEE P A,GUT R,et al.Attaining genetic height potential:Analysis of height outcomes from the ANSWERProgram in children treated with growth hormone over 5 years[J].Growth Horm IGF Res,2015,25(6):286-293.
[18]COHEN P,BRIGHT G M,ROGOL A D,et al.Effects of dose and gender on the growth and growth factor response to GH in GH-deficient children:Implications for efficacy and safety[J].JClin Endocrinol Metab,2002,87(1):90-98