METHODS: A total of 1294 children in Spain enrolled in the observational Genetics and Neuroendocrinology of Short-stature International Study (GeNeSIS). Adverse events were assessed in all GH-treated patients (n=1267) and effectiveness in those with GH deficiency (GHD, 78%).
RESULTS: Mean age at time of entry to the study was 9.8 years. GH was initiated at a median (Q1-Q3) 0.22 (0.20-0.25) mg/kg/week and administered for 2.8 (1.6-4.4) years. For 262 patients with GHD and 4-year data, mean (95% CI) height velocity was 4.3 (4.1 - 4.6) cm/year at baseline, 9.0 (8.7 to 9.4) cm/year at 1-year, and 5.5 (5.2 to 5.8) cm/year at 4-years. Height standard deviation score (SDS) was -2.48 (-2.58 to -2.38) at baseline and -1.18 (-1.28 to -1.08) at 4 years. Final height SDS minus target height SDS (n=241) was -0.09 (-0.20 to 0.02). In 1143 GH-treated patients with ≥1 year follow-up, 93 (8.1%) reported treatment-emergent adverse events. Serious events were reported for 7 children, with 2 considered GH-related.
CONCLUSIONS: These data confirm the benefit of GH replacement therapy on height gain for the patients in Spain. The safety profile was consistent with that already known for GH therapy.
方法:西班牙共有1294名儿童参加了观察性短身材遗传学和神经内分泌学国际研究(GeNeSIS)。在所有GH治疗的患者(n=1267)中评估不良事件,并在GH缺乏患者中评估有效性(GHD,78%)。
结果:进入研究时的平均年龄为9.8岁。GH以中位数(Q1-Q3)0.22(0.20-0.25)mg/kg/周开始,并持续2.8(1.6-4.4)年。对于262例GHD患者和4年数据,基线时平均(95%CI)身高速度为4.3(4.1-4.6)厘米/年,9.0(8.7至9.4)厘米/年,1年和5.5(5.2至5.8)厘米/年在4年。身高标准差评分(SDS)在基线时为-2.48(-2.58至-2.38),在4年时为-1.18(-1.28至-1.08)。最终高度SDS减去目标高度SDS(n=241)为-0.09(-0.20至0.02)。在1143名接受GH治疗的患者中,随访时间≥1年,93例(8.1%)报告了因治疗引起的不良事件。报告了7名儿童的严重事件,与2认为GH相关。
结论:这些数据证实了GH替代疗法对西班牙患者身高增长的益处。安全性与已知的GH治疗一致。