Department of Growth and Reproduction, Copenhagen University Hospital - Rigshospitalet, 2100 Copenhagen, Denmark.
Department of Clinical Medicine, University of Copenhagen, 2200 Copenhagen, Denmark.
J Clin Endocrinol Metab. 2023 Apr 13;108(5):1043-1052. doi: 10.1210/clinem/dgac694.
Early initiation of growth hormone (GH) therapy is recommended for short children born small for gestational age (SGA); however, real-world data indicate that treatment is often delayed.
We aimed to assess the impact of patient age at GH therapy initiation on long-term growth outcomes and safety in short children born SGA.
Analysis of pooled data from NordiNet® International Outcome Study (NCT00960128; 469 European clinics) and the ANSWER Program (NCT01009905; 207 US clinics), two large, complementary observational studies. Patients received GH as prescribed by their treating physician. Enrolled patients born SGA were categorized into three groups based on their age at GH treatment initiation: 2 to <4 years, 4 to <6 years, and ≥6 years. Patient characteristics at birth and GH initiation, auxology, and safety data were evaluated.
The effectiveness analysis (treatment-naïve and prepubertal patients at GH initiation) included 3318 patients: 10.7% aged 2 to <4 years at therapy initiation, 31.6% aged 4 to <6 years, and 57.7% aged ≥6 years. Following 8 years of therapy, the mean improvement in height standard deviation score from baseline was significantly greater in the 2 to <4 years group vs the 4 to <6 years (+2.5 vs +2.2; P = 0.0054) and ≥6 years groups (+2.5 vs +1.7; P < 0.0001). No unexpected safety events were reported.
Early initiation of GH therapy in short children born SGA may be an important contributor to height optimization. The data are reassuring regarding the long-term safety of GH therapy in this population.
对于因宫内生长受限而身材矮小的患儿，建议早期开始生长激素（GH）治疗；然而，实际数据表明，治疗往往会延迟。
我们旨在评估患儿开始 GH 治疗时的年龄对因宫内生长受限而身材矮小的患儿的长期生长结局和安全性的影响。
对 NordiNet®国际结果研究（NCT00960128；469 家欧洲诊所）和 ANSWER 计划（NCT01009905；207 家美国诊所）的汇总数据进行分析，这两项大型、互补的观察性研究。患者接受其治疗医生规定的 GH 治疗。根据开始 GH 治疗时的年龄，将接受 GH 治疗的因宫内生长受限而身材矮小的患儿归入以下三组：2 岁至<4 岁、4 岁至<6 岁和≥6 岁。评估患儿出生时和开始 GH 治疗时的特征、生长情况和安全性数据。
在有效性分析（开始 GH 治疗时为治疗初治和青春前期的患者）中，共纳入 3318 例患者：10.7%的患儿在治疗开始时年龄为 2 岁至<4 岁，31.6%的患儿年龄为 4 岁至<6 岁，57.7%的患儿年龄为≥6 岁。经过 8 年的治疗，与 4 岁至<6 岁组（+2.2；P=0.0054）和≥6 岁组（+1.7；P<0.0001）相比，2 岁至<4 岁组的身高标准差评分从基线的平均改善更显著（+2.5）。未报告意外的安全性事件。
对于因宫内生长受限而身材矮小的患儿，早期开始 GH 治疗可能是优化身高的重要因素。这些数据对于该人群中 GH 治疗的长期安全性是令人安心的。
