Paediatric Endocrinology and Diabetology, University Children's Hospital, Tuebingen, Germany.
Horm Res Paediatr. 2012;77(4):214-21. doi: 10.1159/000336806. Epub 2012 Mar 16.
Prediction algorithms suggest factors determining short- and long-term growth response to growth hormone (GH) in Turner's syndrome (TS). A total of 133 patients (group A; 53% with karyotype 45,X) completed 1 year of treatment and 77 patients (group B) reached adult height (AH) after >4 years on GH treatment. The patients were analysed for factors determining the outcomes, and in addition, the validity of published algorithms was tested.
In group A [values are given as medians (10th-90th percentiles)], starting age was 9.4 (4.8-14) years, height was -3.2 (-4.4 to -1.9) SDS (Prader references), and GH dose was 38 (23-48) μg/kg/day. Observed height velocity was 7.7 (5.2-9.8) cm/years and was equal to the predicted height velocity. In group B, projected adult height (PAH) was 147.4 (139.5-154.8) cm. Total gain in height over PAH of 6.1 (2.0-12.6) cm was negatively correlated with height at start, but positively correlated with GH duration, first year Δheight SDS, or index of responsiveness. Observed AH was 153.5 (146.6-160.1) cm and predicted AH was 155.0 (147.4-161.0) cm, which is statistically not different. On GH <5% IGF-I levels were >2 SDS.
The published prediction algorithms were found to be valid. If normal AH is to be reached at the lowest costs and risks, probably only TS children with a good growth potential and a high responsiveness to GH can be treated successfully with GH doses of <50 μg/kg/day.
预测算法提示了决定特纳综合征(TS)患者短期和长期生长激素(GH)反应的因素。共有 133 名患者(A 组;53%的核型为 45,X)完成了 1 年的治疗,77 名患者(B 组)在 GH 治疗>4 年后达到成人身高(AH)。对这些患者进行了分析,以确定影响治疗结果的因素,并对已发表的算法的有效性进行了测试。
在 A 组中[数值以中位数(10-90 百分位数)表示],起始年龄为 9.4 岁(4.8-14 岁),身高为-3.2(-4.4 至-1.9)SDS(Prader 参考值),GH 剂量为 38(23-48)μg/kg/天。观察到的身高增长速度为 7.7(5.2-9.8)cm/年,与预测的身高增长速度相等。在 B 组中,预测的成人身高(PAH)为 147.4(139.5-154.8)cm。超过 PAH 的身高总增长 6.1(2.0-12.6)cm 与起始身高呈负相关,但与 GH 持续时间、第一年身高 SDS 增长值或反应指数呈正相关。观察到的 AH 为 153.5(146.6-160.1)cm,预测的 AH 为 155.0(147.4-161.0)cm,统计学上无差异。在 GH<5%IGF-I 水平时,>2 SDS。
已发表的预测算法被证明是有效的。如果要以最低的成本和风险达到正常的 AH,可能只有具有良好生长潜力和对 GH 高度反应的 TS 儿童才能成功接受<50μg/kg/天的 GH 剂量治疗。