Nemours Children's Clinic, Jacksonville, Florida 32207, USA.
J Clin Endocrinol Metab. 2011 Nov;96(11):3502-10. doi: 10.1210/jc.2011-1449. Epub 2011 Aug 31.
The type, dose, and route of 17β-estradiol (E(2)) used to feminize girls with Turner syndrome (TS) is not well established.
The objective of the study was to characterize pharmacokinetics and pharmacodynamics of oral vs. transdermal E(2).
The study was conducted at a clinical research center.
Ten girls with TS, mean age 17.7 ± 0.4 (se) yr and 20 normally menstruating controls (aged 16.8 ± 0.4 yr) participated in the study.
TS subjects were randomized 2 wk each to: low-dose daily oral (0.5 mg) and biweekly transdermal E(2) (0.0375 mg) with 2 wk washout in between or high-dose oral (2.0 mg) and transdermal (0.075 mg), studied for 24 h each. Tandem mass spectrometry E(2) and estrone (E(1)) assays and a recombinant cell bioassay were used.
Controls consisted of the following: E(2), 96 ± 11 pg/ml (se), E(1), 70 ± 7 (mean follicular/luteal). TS consisted of the following: E(2), average concentration on low-dose oral, 18 ± 2.1 pg/ml, low-dose transdermal, 38 ± 13, high-dose oral, 46 ± 15, high-dose transdermal, 114 ± 31 pg/ml. E(1) concentrations were much higher on oral E(2) (low or high dose) than transdermal in TS and higher than controls. Bioestrogen was closest to normal in the high-dose transdermal group. LH and FSH decreased more in transdermal than oral low-dose routes and similarly in the high-dose oral and transdermal groups. IGF-I concentrations were variable (P = NS) among groups, and low-density lipoprotein/high-density lipoprotein cholesterol responses were variable.
Transdermal E(2) results in E(2), E(1), and bioestrogen concentrations closer to normal and achieves greater suppression of LH/FSH in lower doses compared with normal. Whether the long-term metabolic effects of estrogen differ using the same form of E(2), depending on route, awaits further study in TS.
用于使 Turner 综合征(TS)女孩女性化的 17β-雌二醇(E2)的类型、剂量和途径尚未得到很好的确立。
本研究的目的是描述口服与透皮 E2 的药代动力学和药效学特征。
该研究在临床研究中心进行。
10 名 TS 女孩,平均年龄 17.7±0.4(se)岁,20 名正常月经的对照者(年龄 16.8±0.4 岁)参与了这项研究。
TS 受试者随机接受 2 周的低剂量每日口服(0.5mg)和双周透皮 E2(0.0375mg)治疗,两种方案之间有 2 周的洗脱期,或者高剂量口服(2.0mg)和透皮(0.075mg)治疗,每种方案均进行 24 小时研究。采用串联质谱 E2 和雌酮(E1)检测和重组细胞生物测定法。
对照组的 E2 为 96±11pg/ml(se),E1 为 70±7(卵泡期/黄体期平均值)。TS 组的 E2 平均浓度为:低剂量口服,18±2.1pg/ml;低剂量透皮,38±13;高剂量口服,46±15;高剂量透皮,114±31pg/ml。在 TS 中,口服 E2(低剂量或高剂量)的 E1 浓度均高于透皮,且高于对照组。高剂量透皮组的生物雌激素最接近正常。与口服低剂量相比,透皮给药的 LH 和 FSH 下降更明显,而高剂量口服和透皮组之间的下降程度相似。IGF-I 浓度在各组之间差异无统计学意义(P=NS),低密度脂蛋白/高密度脂蛋白胆固醇反应也存在差异。
与正常相比,透皮 E2 可使 E2、E1 和生物雌激素浓度更接近正常,并在较低剂量下实现更大程度的 LH/FSH 抑制。根据途径不同,相同形式的 E2 是否会对 TS 产生不同的长期代谢影响,还需要进一步研究。