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特纳综合征患儿生长激素治疗的年龄和持续时间的影响。

Impact of age and duration of growth hormone therapy in children with Turner syndrome.

机构信息

Department of Pediatrics, Thomas Jefferson University DuPont Hospital for Children, Philadelphia, Pa., USA.

出版信息

Horm Res Paediatr. 2011;76(6):392-9. doi: 10.1159/000333073. Epub 2011 Nov 29.

Abstract

BACKGROUND/AIMS: To assess height standard deviation scores (HSDS) in patients with Turner syndrome (TS) by age at treatment initiation and varying durations of treatment with growth hormone (GH).

METHODS

GH treatment-naïve patients with TS from the American Norditropin Studies: Web-enabled Research (ANSWER) Program® Registry were analyzed at baseline, 4 months, and annually.

RESULTS

Three hundred and eighty-two patients with TS had a baseline mean (±SD) HSDS of -2.6 ± 0.9. Patients received short-term (1 year), long-term (<3 years), and extended GH treatment (≥3 years, mean = 4.54 years), resulting in 40.2% (n = 99/246), 60.5% (n = 69/114), and 62.3% (n = 86/138) of the patients achieving HSDS >-2. Patients starting GH at a younger age experienced better growth response, regardless of treatment duration. Change in HSDS from baseline (ΔHSDS) at 4 months correlated positively with ΔHSDS at 1 and 3 years, and ΔHSDS at 1 year with ΔHSDS at 3 years (p values from 0.0017 to<0.0001).

CONCLUSIONS

Height gains in patients with TS during short-term treatment were found to be highly predictive of longer-term results. Continuation of GH treatment (≥3 years) resulted in 62.3% of the patients achieving an HSDS within the normal population range, indicating the clinical importance of early initiation and continuation of GH treatment in patients with TS.

摘要

背景/目的:评估特纳综合征(TS)患者在不同起始治疗年龄和不同生长激素(GH)治疗持续时间时的身高标准差评分(HSDS)。

方法

对美国 Norditropin 研究:Web 支持的研究(ANSWER)计划®注册处的 GH 治疗初治 TS 患者进行分析,在基线、4 个月和每年进行分析。

结果

382 例 TS 患者的基线平均(±SD)HSDS 为-2.6±0.9。患者接受短期(1 年)、长期(<3 年)和延长 GH 治疗(≥3 年,平均=4.54 年),结果有 40.2%(n=99/246)、60.5%(n=69/114)和 62.3%(n=86/138)的患者 HSDS>-2。无论治疗持续时间如何,年龄较小开始 GH 治疗的患者生长反应更好。4 个月时 HSDS 的变化(ΔHSDS)与 1 年和 3 年时的ΔHSDS 呈正相关,1 年时的 ΔHSDS 与 3 年时的 ΔHSDS 也呈正相关(p 值均<0.0001)。

结论

在短期治疗期间,TS 患者的身高增长被发现与长期结果高度相关。继续 GH 治疗(≥3 年)导致 62.3%的患者 HSDS 达到正常人群范围,表明在 TS 患者中早期开始和继续 GH 治疗的临床重要性。

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