乌鲁木齐市第一人民医院儿童内分泌科 新疆 乌鲁木齐 830000
闻钰,女,1976—,副主任医师,主要从事儿童内分泌研究,E‑mail: byf1234@163.com
收稿:2023-05-08,
纸质出版:2025-05-25
移动端阅览
闻钰,千茜茜,朱宁等.重组人生长激素对儿童特发性矮小症的疗效及预测模型建立[J].武汉大学学报(医学版),2025,46(05):655-661.
WEN Yu,QIAN Xixi,ZHU Ning,et al.Curative effect of recombinant human growth hormone on children with idiopathic short stature and establishment of prediction model[J].Medical Journal of Wuhan University,2025,46(05):655-661.
闻钰,千茜茜,朱宁等.重组人生长激素对儿童特发性矮小症的疗效及预测模型建立[J].武汉大学学报(医学版),2025,46(05):655-661. DOI: 10.14188/j.1671-8852.2023.0286.
WEN Yu,QIAN Xixi,ZHU Ning,et al.Curative effect of recombinant human growth hormone on children with idiopathic short stature and establishment of prediction model[J].Medical Journal of Wuhan University,2025,46(05):655-661. DOI: 10.14188/j.1671-8852.2023.0286.
目的
2
观察重组人生长激素(rhGH)对儿童特发性矮小症(ISS)的治疗效果,建立rhGH治疗后生长速率的预测模型并加以验证。
方法
2
选取2020年6月至2021年12月在乌鲁木齐市第一人民医院采用rhGH治疗的152例ISS患儿为研究对象,比较治疗前后各时期生长指标变化。以治疗12月后年生长速率(GV)为因变量,采用多元逐步回归方法确定rhGH疗效的相关预测因素并构建预测模型,并对模型准确度予以验证。
结果
2
rhGH治疗6个月、12个月的身高(Ht)、身高标准差比值(HtSDS)均逐渐升高(
P
<
0.05),rhGH治疗12个月的身高增长(△Ht)、GV均低于治疗6个月(
P
<
0.05);同时治疗6个月与12个月GV均与生活年龄(CA)、Ht、骨龄指数(BAI)、GH峰、垂体高度呈负相关(
P
<
0.05),与遗传靶身高(TH)、骨龄差(BAD)呈正相关(
P
<
0.05),而治疗6个月GV还与HtSDS呈正相关(
P
<
0.05);将治疗12个月GV作为因变量,治疗前Ht(
X
1
)、BAD(
X
2
)、GH峰(
X
3
)及治疗6个月GV
6
(
X
4
)被纳入方程,建立预测模型:
Y
=4.338-0.062
X
1
+0.012
X
2
-0.030
X
3
+0.772
X
4
,
R
2
=0.731,并通过外部验证。
结论
2
rhGH对ISS患儿的生长发育具有较好疗效,且治疗中身高的增长是非匀速连续生长。治疗后患儿GV与多因素相关。以治疗前Ht、BAD、GH峰,及治疗6个月GV为预测因素构建的多元回归模型具有较好的预测效能。
Objective
2
To observe the curative effect of recombinant human growth hormone (rhGH) on idiopathic short stature (ISS) children
and to establish a predictive model of growth rate after rhGH treatment and verify it.
Methods
2
A total of 152 ISS children treated with rhGH in Urumqi First People's Hospital from June 2020 to December 2021 were selected as the study objects. The growth indexes were compared before and after treatment. And with growth velocity (GV) at 12 months as the dependent variable
the multiple stepwise regression method was used to determine the relevant predictors of rhGH efficacy and construct a prediction model
and the accuracy of the model was verified.
Results
2
Height (Ht) and height standard deviation scores (HtSDS) gradually increased at 6 and 12 months of rhGH treatment (
P
<
0.05)
and △Ht and GV at 12 months of rhGH treatment were lower than those at 6 months of treatment (
P
<
0.05). At the same time
GV at 6 and 12 months of treatment was negatively correlated with chronological age (CA)
Ht
bone age index (BAI)
GH peak
and pituitary height (
P
<
0.05) and positively correlated with target height (TH) and bone age difference (BAD) (
P
<
0.05)
while GV at 6 months of treatment was also positively correlated with HtSDS (
P
<
0.05). In addition
GV at 12 months of treatment was used as the dependent variable
and Ht (
X
1
)
BAD (
X
2
)
GH peak (
X
3
)
and GV
6
(
X
4
) at 6 months of treatment were included in the equation to establish a prediction model:
Y
=4.338-0.062
X
1
+0.012
X
2
-0.030
X
3
+0.772
X
4
R
2
=0.731
and the model was verified externally.
Conclusion
2
The rhGH treatment has a good curative effect on the height growth of ISS children
and the increase in height during treatment is non‑uniform
with continuous growth. After treatment
GV was associated with multiple factors. The multiple regression model constructed with Ht
BAD
GH peak before treatment
and GV at 6 months after treatment as predictors had good predictive power.
COLLETT‑SOLBERG P F , AMBLER G , BACKELJAUW P F , et al . Diagnosis, genetics, and therapy of short stature in children: A growth hormone research society international perspective [J ] . Horm Res Paediatr , 2019 , 92 ( 1 ): 1 ‑ 14 .
RAPAPORT R , WIT J M , SAVAGE M O . Growth failure: 'idiopathic' only after a detailed diagnostic evaluation [J ] . Endocr Connect , 2021 , 10 ( 3 ): R125 ‑ R138 .
HALAS J G , GRIMBERG A . Dilemmas of growth hormone treatment for GH deficiency and idiopathic short stature: Defining, distinguishing, and deciding [J ] . Minerva Pediatr , 2020 , 72 ( 3 ): 206 ‑ 225 .
DANOWITZ M , GRIMBERG A . Clinical indications for growth hormone therapy [J ] . Adv Pediatr , 2022 , 69 ( 1 ): 203 ‑ 217 .
陈倩 , 杨亭 , 张萱 , 等 . 生长激素治疗儿童特发性矮小症疗效分析 [J ] . 儿科药学杂志 , 2019 , 25 ( 6 ): 6 ‑ 9 .
CHEN Q , YANG T , ZHANG X , et al . Growth hormone on children with idiopathic short stature [J ] . J Pediatr Pharm , 2019 , 25 ( 6 ): 6 ‑ 9 .
王煜娇 , 武苏 , 袁雪雯 , 等 . 重组人生长激素治疗特发性矮小患儿的疗效及生长速率预测模型的建立 [J ] . 现代生物医学进展 , 2020 , 20 ( 22 ): 4244 ‑ 4248 .
WANG Y J , WU S , YUAN X W , et al . The curative effect of recombinant human growth hormone on children with idiopathic short stature and establishment of growth rate prediction model [J ] . Prog Mod Biomed , 2020 , 20 ( 22 ): 4244 ‑ 4248 .
SAVAGE M O , STORR H L . GH resistance is a component of idiopathic short stature: Implications for rhGH therapy [J ] . Front Endocrinol , 2021 , 12 : 781044 .
YING Y Q , HOU L , LIANG Y , et al . Efficacy and safety of recombinant human growth hormone in treating Chinese children with idiopathic short stature [J ] . Growth Horm IGF Res , 2018 , 42 / 43 : 80 ‑ 85 .
KE X A , LIANG H T , MIAO H , et al . Clinical characteristics of short‑stature patients with an NPR2 mutation and the therapeutic response to rhGH [J ] . J Clin Endocrinol Metab , 2021 , 106 ( 2 ): 431 ‑ 441 .
DE ARRIBA MUÑOZ A , MUÑIZ V C , SAEZ J J A , et al . Impact of adherence on growth response during the first 2 years of growth hormone treatment [J ] . Endocrine , 2021 , 72 ( 2 ): 513 ‑ 523 .
王婷 , 郑荣秀 , 刘靓 , 等 . 重组人生长激素对矮身材儿童身高增长疗效分析及预测模型的建立 [J ] . 天津医科大学学报 , 2018 , 24 ( 3 ): 232 ‑ 235 .
WANG T , ZHENG R X , LIU L , et al . Effect of recombinant human growth hormone therapy on GHD and ISS patients and establishment of prediction model [J ] . J Tianjin Med Univ , 2018 , 24 ( 3 ): 232 ‑ 235 .
韩晓伟 , 董治亚 , 张婉玉 , 等 . 矮小症病因及临床特征分析 [J ] . 临床儿科杂志 , 2019 , 37 ( 1 ): 39 ‑ 42 .
HAN X W , DONG Z Y , ZHANG W Y , et al . Analysis of the etiology and clinical characteristics of short stature [J ] . J Clin Pediatr , 2019 , 37 ( 1 ): 39 ‑ 42 .
INZAGHI E , REITER E , CIANFARANI S . The challenge of defining and investigating the causes of idiopathic short stature and finding an effective therapy [J ] . Horm Res Paediatr , 2019 , 92 ( 2 ): 71 ‑ 83 .
SODERO G , MARIANI F , CAPRARELLI M , et al . Growth hormone responses during arginine and clonidine stimulation test: Correlations with patients' auxological and metabolic parameters in a single centre study [J ] . Growth Horm IGF Res , 2023 , 68 : 101522 .
王玉 , 解欣 , 柏金秀 , 等 . 生长激素治疗前后矮小症儿童血清生长激素释放肽、摄食抑制因子1水平的变化及意义 [J ] . 实用医学杂志 , 2019 , 35 ( 19 ): 3038 ‑ 3041 .
WANG Y , XIE X , BAI J X , et al . Significance of changes of serum ghrelin and nesfatin‑1 level in children with short stature before and after the process of growth hormone therapy [J ] . J Pract Med , 2019 , 35 ( 19 ): 3038 ‑ 3041 .
李辉 , 季成叶 , 宗心南 , 等 . 中国0~18岁儿童、青少年身高、体重的标准化生长曲线 [J ] . 中华儿科杂志 , 2009 , 47 ( 7 ): 487 ‑ 492 .
LI H , JI C Y , ZONG X N , et al . Height and weight standardized growth charts for Chinese children and adolescents aged 0 to 18 years [J ] . Chin J Pediatr , 2009 , 47 ( 7 ): 487 ‑ 492 .
YUAN J H , DU Z , WU Z W , et al . A novel diagnostic predictive model for idiopathic short stature in children [J ] . Front Endocrinol , 2021 , 12 : 721812 .
MAGHNIE M , RANKE M B , GEFFNER M E , et al . Safety and efficacy of pediatric growth hormone therapy: Results from the full KIGS cohort [J ] . J Clin Endocrinol Metab , 2022 , 107 ( 12 ): 3287 ‑ 3301 .
MIGLIARETTI G , DITARANTO S , GUIOT C , et al . Long‑term response to recombinant human growth hormone treatment: A new predictive mathematical method [J ] . J Endocrinol Invest , 2018 , 41 ( 7 ): 839 ‑ 848 .
YING Y Q , HOU L , LIANG Y , et al . Efficacy and safety of recombinant human growth hormone in treating Chinese children with idiopathic short stature [J ] . Growth Horm IGF Res , 2018 , 42 / 43 : 80 ‑ 85 .
黄旗振 . 重组人生长激素治疗儿童特发性矮小症的临床效果 [J ] . 中国医药科学 , 2023 , 13 ( 4 ): 102 ‑ 105 .
HUANG Q Z . Observation on clinical effect of recombinant human growth hormone on children with idiopathic short stature [J ] . China Med Pharm , 2023 , 13 ( 4 ): 102 ‑ 105 .
VYAS V , MENON R K . Management of short stature: Use of growth hormone in GH‑deficient and non‑GH‑deficient conditions [J ] . Indian J Pediatr , 2021 , 88 ( 12 ): 1203 ‑ 1208 .
SÄVENDAHL L , COOKE R , TIDBLAD A , et al . Long‑term mortality after childhood growth hormone treatment: The SAGhE cohort study [J ] . Lancet Diabetes Endocrinol , 2020 , 8 ( 8 ): 683 ‑ 692 .
KOSTERIA I , ALOUMANIS K , KANAKA‑GANTENBEIN C , et al . Pediatric growth hormone therapy in Greece: Analysis of the Hellenic cohort of the GeNeSIS study [J ] . Hormones , 2019 , 18 ( 4 ): 423 ‑ 434 .
CUTFIELD W S , ALBERT B B . Growth hormone treatment for idiopathic short stature [J ] . Pediatr Endocrinol Rev , 2018 , 16 ( Suppl 1 ): 113 ‑ 122 .
任娟娟 , 任巧 . rhGH治疗儿童生长激素缺乏症和特发性矮小症的疗效及影响因素研究 [J ] . 临床医学 , 2020 , 40 ( 10 ): 89 ‑ 91 .
REN J J , REN Q . Effect of rhGH on children with growth hormone deficiency and idiopathic dwarf and its influencing factors [J ] . Clin Med , 2020 , 40 ( 10 ): 89 ‑ 91 .
0
浏览量
7
下载量
0
CSCD
关联资源
相关文章
相关作者
相关机构
京公网安备11010802024621