生长激素缺乏症患儿对每周一次索马帕西坦治疗的生长反应与生长激素刺激试验中的生长激素阈值相关。
Growth Response to Weekly Somapacitan Therapy in Children With GH Deficiency Is Related to GH Thresholds in GH Stimulation Testing.
作者信息
Backeljauw Philippe F, Boettcher Claudia, Højby Michael, Kelepouris Nicky, Jeppesen Mathias Lerbech, Mori Jun, Pietropoli Alberto, Miller Bradley S
机构信息
Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH 45229, USA.
Paediatric Endocrinology and Diabetology, Julie-von-Jenner Haus, University of Bern Faculty of Medicine, 3010 Bern, Switzerland.
出版信息
J Endocr Soc. 2025 Feb 27;9(4):bvaf038. doi: 10.1210/jendso/bvaf038. eCollection 2025 Mar 3.
CONTEXT
The lack of a severity definition and standardized GH cutoff level for GH deficiency (GHD) diagnosis in children leads to ambiguity in the interpretation of GH stimulation tests and treatment recommendations.
OBJECTIVE
To investigate treatment response differences among children with GHD treated with daily GH (somatropin) (year 1) or once-weekly somapacitan (years 1 and 2) based on GH peak concentrations assessed at diagnosis.
METHODS
This was a subgroup analysis of 200 patients with GHD aged ≥2.5 years participating in the REAL4 randomized, phase 3 trial. Height velocity (HV; cm/year) and changes in height SD score (SDS) and IGF-I SDS from baseline were compared for 3 GH peak groups: ≤ 3, >3 to <7, and ≥7 to ≤10 μg/L.
RESULTS
The ≤3 μg/L GH peak concentration group had the greatest HV at weeks 52 and 104. Mean change in height SDS ranged from 1.89 to 1.59, 1.17 to 1.06, and 0.92 to 1.07 at week 52 and 2.79 to 2.30, 1.64 to 1.54, and 1.33 to 1.51 at week 104 for the 2 treatment groups across the 3 GH peak concentrations, respectively. Mean change in IGF-I SDS ranged from 3.13 to 3.01, 2.11 to 1.96, and 1.87 to 2.26 at week 52 and from 2.81 to 2.11, 1.85 to 1.62, and 1.28 to 1.71 at week 104 for the 3 GH peak concentrations.
CONCLUSION
Patients with GHD in the ≤3 μg/L GH peak concentration group had greater HV and greater changes in height SDS and IGF-I SDS from baseline.
背景
儿童生长激素缺乏症（GHD）诊断中缺乏严重程度定义和标准化的生长激素（GH）截断水平，导致对GH刺激试验结果的解读和治疗建议存在模糊性。
目的
根据诊断时评估的GH峰值浓度，研究每日使用GH（生长激素）治疗1年的GHD患儿与每周使用一次索马普坦治疗1年和2年的GHD患儿的治疗反应差异。
方法
这是一项对参与REAL4随机3期试验的200例年龄≥2.5岁的GHD患者进行的亚组分析。比较了3个GH峰值组（≤3、>3至<7、≥7至≤10μg/L）的身高增长速度（HV；cm/年）以及身高标准差评分（SDS）和IGF-I SDS较基线的变化。
结果
GH峰值浓度≤3μg/L组在第52周和第104周时HV最高。在第52周时，3个GH峰值浓度下2个治疗组的身高SDS平均变化分别为1.89至1.59、1.17至1.06、0.92至1.07；在第104周时分别为2.79至2.30、1.64至1.54、1.33至1.51。在第52周时，3个GH峰值浓度下IGF-I SDS平均变化分别为3.13至3.01、2.11至1.96、1.87至2.26；在第104周时分别为2.81至2.11、1.85至1.62、1.28至1.71。
结论
GH峰值浓度≤3μg/L的GHD患者的HV更高，且身高SDS和IGF-I SDS较基线的变化更大。
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