Department of Pediatrics, Thomas Jefferson University, Philadelphia, Pa. 19107, USA.
Horm Res Paediatr. 2011;75(1):38-46. doi: 10.1159/000317529. Epub 2010 Aug 20.
BACKGROUND/AIMS: Untreated girls with Turner syndrome (TS) have growth failure, and adult height is, on average, 20 cm less than predicted height. Treatment with growth hormone (GH) is now standard of care. The objective of this study was to investigate the benefit of adding oxandrolone (Ox) to GH in a long-term, randomized, placebo (Pl)-controlled prospective trial to near adult height in TS.
prospective, randomized, Pl-controlled study: 76 girls with TS (ages 10-14.9 years) were randomized to receive Ox (0.06 mg/kg/day) or Pl in combination with GH (0.35 mg/kg/week, daily) over 2 years. Auxologic data, breast and pubic hair Tanner stages, and hormone and lipid levels were measured. Subjects who chose to continue were followed in a 2-year double-blind extension, also received estrogen therapy (years 3, 4), and had dual-energy X-ray absorptiometry evaluation of bone density (years 3, 4).
at year 4, the change in absolute height and height SDS was greater in the GH/Ox versus GH/Pl group [26.2 ± 6.7 vs. 22.2 ± 5.1 cm, analysis of covariance (ANCOVA) p < 0.001; 1.8 ± 0.9 vs. 1.2 ± 0.7 standard deviation scores, ANCOVA p < 0.001]. Bone mineral density (BMD) of the wrist (0.51 ± 0.17 vs. 0.54 ± 0.05 g/cm(2)) and spine (0.91 ± 0.34 vs. 0.96 ± 0.13 g/cm(2)) in the GH/Ox versus GH/Pl groups was similar after 4 years. Breast development was slower in the GH/Ox versus GH/Pl group [year 4: Tanner stage 2.9 ± 1.3 (Ox) vs. 4.1 ± 1.3 (Pl), p = 0.003], and menarche was approximately 1 year later.
the addition of Ox to GH at mean age 12.0 ± 1.7 year augmented height gain after 4 years of treatment, slowed breast development and did not affect BMD in girls with TS. Whether initiation of Ox prior to initiation of pubertal development would optimize height gain without impeding breast development will require further study.
背景/目的：未经治疗的特纳综合征（TS）女孩存在生长障碍，成人身高平均比预测身高低 20 厘米。目前，生长激素（GH）治疗是标准的治疗方法。本研究的目的是通过一项长期、随机、安慰剂（Pl）对照的前瞻性试验，研究在接近 TS 成年身高的情况下，添加氧雄龙（Ox）对 GH 的益处。
前瞻性、随机、Pl 对照研究：76 名年龄在 10-14.9 岁的 TS 女孩被随机分为 Ox（0.06 mg/kg/天）或 Pl 组，与 GH（0.35 mg/kg/周，每日）联合治疗 2 年。测量了生长指标、乳房和阴毛 Tanner 分期、激素和血脂水平。选择继续治疗的受试者在 2 年的双盲扩展期继续接受治疗，同时接受雌激素治疗（第 3、4 年），并进行双能 X 线吸收法骨密度评估（第 3、4 年）。
在第 4 年，GH/Ox 组与 GH/Pl 组的绝对身高和身高 SDS 变化更大[26.2 ± 6.7 比 22.2 ± 5.1 cm，协方差分析（ANCOVA）p < 0.001；1.8 ± 0.9 比 1.2 ± 0.7 标准差评分，ANCOVA p < 0.001]。GH/Ox 组与 GH/Pl 组的手腕（0.51 ± 0.17 比 0.54 ± 0.05 g/cm(2)) 和脊柱（0.91 ± 0.34 比 0.96 ± 0.13 g/cm(2)) 的骨密度（BMD）在 4 年后相似。GH/Ox 组的乳房发育速度比 GH/Pl 组慢[第 4 年：Tanner 分期 2.9 ± 1.3（Ox）比 4.1 ± 1.3（Pl），p = 0.003]，月经初潮晚约 1 年。
在平均年龄 12.0 ± 1.7 岁时，在 GH 中添加 Ox 可在治疗 4 年后增加身高增长，减缓乳房发育，对 TS 女孩的骨密度无影响。在青春期发育前开始使用 Ox 是否会在不影响乳房发育的情况下优化身高增长，还需要进一步研究。
