TRANSDERMAL VS ORAL ESTROGEN THERAPY ON ADOLESCENTS WITH TURNER'S SYNDROME

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。特纳综合征(TS)的雌激素替代治疗最常用的是雌激素制剂。根据初步数据，我们推测，在生长激素(GH)治疗的青少年TS中，透皮与口服雌二醇会对近终末成人身高(FAH)和近峰值骨量(PBM)有更有利的影响。本研究的目的是评价透皮雌激素和口服雌激素对生长和骨量的影响，以及它们与生长因子水平、骨转换指标和性激素水平的相关性。这项为期两年的选择性随机前瞻性研究包括两个治疗组：等量的口服雌二醇组和透皮给药组，并结合标准生长激素治疗。12-15岁的TS青少年将根据骨龄被选择性地随机分为每组。在这两年中，两组的雌激素剂量都将在模拟正常青春期的情况下每6个月逐渐增加一次。每组样本量为12个，检验显著性水平为0.05，我们将有80%的能力来检测两组之间25%的增长差异。在骨量方面没有初步数据来评估样本大小估计，因为两组之间有显著差异。结果计量的统计分析将通过重复计量方差分析进行。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Estrogen replacement in Turner Syndrome (TS) is accomplished most commonly using estrogen preparations. Based on preliminary data we hypothesize that transdermal vs. oral estradiol will have more favorable effect on near final adult height (FAH) and near peak bone mass (PBM) in growth hormone (GH) treated adolescents with TS. The aim of the study is to evaluate the effect of transdermal vs. oral estrogen on growth and bone mass and their correlation with growth factor levels, markers of bone turnover and sex steroid levels. This 2 year selectively randomized prospective study involves two treatment groups: equivalent doses of oral vs. transdermal estradiol in combination with standard growth hormone therapy. The TS adolescents ages 12-15 years will be selectively randomized to each group by bone age. Estrogen dose will be gradually increased every 6 months over the two years in both groups mimicking normal puberty. With a sample size of 12 in each group and test significance level of 0.05, we will have an 80% power to detect a 25% difference in growth of two groups. There is no preliminary data in terms of bone mass to evaluate sample size estimation for signigicant difference between two groups. Statistical analysis of outcome measures will be done with repeated measures analysis of variance.