生长激素治疗特纳综合征幼儿早期生长发育迟缓：一项随机、对照、多中心试验
Growth hormone treatment of early growth failure in toddlers with Turner syndrome: a randomized, controlled, multicenter trial.
作者信息
Davenport Marsha L, Crowe Brenda J, Travers Sharon H, Rubin Karen, Ross Judith L, Fechner Patricia Y, Gunther Daniel F, Liu Chunhua, Geffner Mitchell E, Thrailkill Kathryn, Huseman Carol, Zagar Anthony J, Quigley Charmian A
机构信息
Division of Pediatric Endocrinology, University of North Carolina, CB 7039, 3341 Medical Biomolecular Research Building, Chapel Hill, North Carolina 27599-7039, USA.
出版信息
J Clin Endocrinol Metab. 2007 Sep;92(9):3406-16. doi: 10.1210/jc.2006-2874. Epub 2007 Jun 26.
CONTEXT
Typically, growth failure in Turner syndrome (TS) begins prenatally, and height sd score (SDS) declines progressively from birth.
OBJECTIVE
This study aimed to determine whether GH treatment initiated before 4 yr of age in girls with TS could prevent subsequent growth failure. Secondary objectives were to identify factors associated with treatment response, to determine whether outcome could be predicted by a regression model using these factors, and to assess the safety of GH treatment in this young cohort.
DESIGN
This study was a prospective, randomized, controlled, open-label, multicenter clinical trial (Toddler Turner Study, August 1999 to August 2003).
SETTING
The study was conducted at 11 U.S. pediatric endocrine centers.
SUBJECTS
Eighty-eight girls with TS, aged 9 months to 4 yr, were enrolled.
INTERVENTIONS
Interventions comprised recombinant GH (50 mug/kg.d; n = 45) or no treatment (n = 43) for 2 yr.
MAIN OUTCOME MEASURE
The main outcome measure was baseline-to-2-yr change in height SDS.
RESULTS
Short stature was evident at baseline (mean length/height SDS = -1.6 +/- 1.0 at mean age 24.0 +/- 12.1 months). Mean height SDS increased in the GH group from -1.4 +/- 1.0 to -0.3 +/- 1.1 (1.1 SDS gain), whereas it decreased in the control group from -1.8 +/- 1.1 to -2.2 +/- 1.2 (0.5 SDS decline), resulting in a 2-yr between-group difference of 1.6 +/- 0.6 SDS (P < 0.0001). The baseline variable that correlated most strongly with 2-yr height gain was the difference between mid-parental height SDS and subjects' height SDS (r = 0.32; P = 0.04). Although attained height SDS at 2 yr could be predicted with good accuracy using baseline variables alone (R(2) = 0.81; P < 0.0001), prediction of 2-yr change in height SDS required inclusion of initial treatment response data (4-month or 1-yr height velocity) in the model (R(2) = 0.54; P < 0.0001). No new or unexpected safety signals associated with GH treatment were detected.
CONCLUSION
Early GH treatment can correct growth failure and normalize height in infants and toddlers with TS.
背景
通常，特纳综合征（TS）患者的生长发育迟缓始于产前，出生后身高标准差评分（SDS）逐渐下降。
目的
本研究旨在确定TS女童在4岁前开始生长激素（GH）治疗是否可预防随后的生长发育迟缓。次要目的是确定与治疗反应相关的因素，确定是否可用回归模型通过这些因素预测治疗结果，并评估GH治疗在该年轻队列中的安全性。
设计
本研究为前瞻性、随机、对照、开放标签、多中心临床试验（幼儿特纳研究，1999年8月至2003年8月）。
地点
该研究在美国11个儿科内分泌中心进行。
对象
招募了88名年龄在9个月至4岁的TS女童。
干预措施
干预措施包括重组GH（50μg/kg·d；n = 45）或不治疗（n = 43），为期2年。
主要结局指标
主要结局指标为基线至2年时身高SDS的变化。
结果
基线时即存在身材矮小（平均年龄24.0±12.1个月时，平均身长/身高SDS = -1.6±1.0）。GH组平均身高SDS从-1.4±1.0增至-0.3±1.1（增加1.1 SDS），而对照组从-1.8±1.1降至-2.2±1.2（下降0.5 SDS），导致两组2年的差异为1.6±0.6 SDS（P < 0.0001）。与2年身高增长相关性最强的基线变量是父母身高中位数SDS与受试者身高SDS的差值（r = 0.32；P = 0.04）。尽管仅使用基线变量就能较好地预测2年时达到的身高SDS（R² = 0.81；P < 0.0001），但预测身高SDS的2年变化需要在模型中纳入初始治疗反应数据（4个月或1年身高增长速度）（R² = 0.54；P < 0.0001）。未检测到与GH治疗相关的新的或意外的安全信号。
结论
早期GH治疗可纠正TS婴幼儿的生长发育迟缓并使身高正常化。
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