Cleemann Wang Amanda, Hagen Casper P, Nedaeifard Leila, Juul Anders, Jensen Rikke Beck
Department of Growth and Reproduction, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
J Clin Endocrinol Metab. 2020 Aug 1;105(8). doi: 10.1210/clinem/dgaa274.
Girls with Turner syndrome (TS) suffer linear growth failure, and TS is a registered indication for growth hormone (GH) treatment. GH is classically dosed according to body weight, and serum insulin-like growth factor-1 (IGF-1) concentrations are recommended to be kept within references according to international guidelines.
To assess the effect of long-term GH treatment in girls with TS following GH dosing by IGF-1 titration.
A retrospective, real-world evidence, observational study consisting of data collected in a single tertiary center from 1991 to 2018.
A cohort of 63 girls with TS treated with GH by IGF-1 titration with a median duration of 6.7 years (interquartile range [IQR]: 3.4-9.7 years).
Longitudinal measurements of height, IGF-1, and adult height (AH) following GH treatment were evaluated and compared between the different karyotypes (45,X, 45,X/46,XX, or miscellaneous).
Using GH dose titration according to IGF-1, only 6% of girls with TS had supranormal IGF-1 levels. Median dose was 33 µg/kg/day (IQR: 28-39 µg/kg/day) with no difference between the karyotype groups. AH was reached for 73% who attained a median AH of 1.25 standard deviation score (SDS) for age specific TS references (IQR: 0.64-1.50 SDS), and a median gain in height (ΔHSDS: AH SDS minus baseline height SDS of TS references) of 0.50 SDS, equal to 3.2 cm (SD 7.68) for all karyotypes.
Our real-world evidence study suggested that titration of GH dose to keep IGF-1 levels within the normal range resulted in a lower AH gain than in studies where a fixed dose was used.
特纳综合征（TS）女孩存在线性生长发育迟缓，TS是生长激素（GH）治疗的注册适应症。传统上，GH根据体重给药，国际指南建议血清胰岛素样生长因子-1（IGF-1）浓度应保持在参考范围内。
评估通过IGF-1滴定法调整GH剂量对TS女孩进行长期GH治疗的效果。
一项回顾性、基于真实世界证据的观察性研究，数据收集于1991年至2018年期间的一家单一三级中心。
一组63例接受IGF-1滴定法GH治疗的TS女孩，中位治疗时间为6.7年（四分位间距[IQR]：3.4 - 9.7年）。
评估并比较不同核型（45,X、45,X/46,XX或其他）的GH治疗后身高、IGF-1和成人身高（AH）的纵向测量值。
采用根据IGF-1滴定GH剂量的方法，只有6%的TS女孩IGF-1水平高于正常。中位剂量为33μg/kg/天（IQR：28 - 39μg/kg/天），各核型组之间无差异。73%的患者达到了AH，其年龄特异性TS参考值的中位AH为1.25标准差评分（SDS）（IQR：0.64 - 1.50 SDS），所有核型的身高中位数增加（ΔHSDS：AH SDS减去TS参考值的基线身高SDS）为0.50 SDS，相当于3.2 cm（标准差7.68）。
我们基于真实世界证据的研究表明，将GH剂量滴定以使IGF-1水平保持在正常范围内，与使用固定剂量的研究相比，AH增加较低。
