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Abstract：
BACKGROUND: Country-specific information on pediatric GH therapy is available from multi-national studies.
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.
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.
摘要：
背景：儿童GH治疗的国家特异性信息可从多国研究中获得。
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。
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。
