Rosenfeld R G, Attie K M, Frane J, Brasel J A, Burstein S, Cara J F, Chernausek S, Gotlin R W, Kuntze J, Lippe B M, Mahoney C P, Moore W V, Saenger P, Johanson A J
Department of Pediatrics, Oregon Health Sciences University, Portland 97201, USA.
J Pediatr. 1998 Feb;132(2):319-24. doi: 10.1016/s0022-3476(98)70452-4.
To carry out a multicenter, prospective, randomized trial of human growth hormone (GH), alone or in combination with oxandrolone (OX), in patients with Turner's syndrome (TS).
In an initial phase lasting 12 to 24 months, 70 girls with TS, verified by karyotype, were randomly assigned to one of four groups: (1) observation, (2) OX, (3) GH, or (4) GH plus OX. After completion of the first phase, group 3 subjects continued to receive GH only. All other subjects were treated with GH plus OX. Subjects were followed up until attainment of adult height and/or cessation of treatment. Data from this trial were compared with growth characteristics of 25 American historical subjects with TS (matched for age, height, parental target height, and karyotype) who never received either GH or androgens.
Of the 70 subjects enrolled, 60 completed the clinical trial. The 17 subjects receiving GH alone all completed the trial and reached a height of 150.4+/-5.5 cm (mean +/- SD), 8.4+/-4.5 cm taller than their mean projected adult height at enrollment (95% confidence interval [CI]: 6.3 to 10.6 cm). The 43 subjects receiving GH plus OX attained a mean height of 152.1+/-5.9 cm, 10.3+/-4.7 cm taller than their mean projected adult height (95% CI: 8.9 to 11.7 cm). The historical control subjects had a mean adult height of 144.2+/-6.0 cm, precisely matching their original projected adult height of 144.2+/-6.1 cm.
GH, either alone or in combination with OX, is capable of stimulating short-term growth and augmenting adult height in girls with TS. With early diagnosis and initiation of treatment, an adult height of more than 150 cm is a reasonable goal for most girls with TS.
开展一项关于重组人生长激素（GH）单独使用或联合氧雄龙（OX）治疗特纳综合征（TS）患者的多中心、前瞻性、随机试验。
在为期12至24个月的初始阶段，70名经核型分析确诊为TS的女孩被随机分为四组之一：（1）观察；（2）OX；（3）GH；或（4）GH加OX。第一阶段结束后，第3组受试者继续仅接受GH治疗。所有其他受试者接受GH加OX治疗。对受试者进行随访，直至达到成人身高和/或停止治疗。将该试验的数据与25名从未接受过GH或雄激素治疗的美国TS历史受试者（年龄、身高、父母目标身高和核型匹配）的生长特征进行比较。
70名入组受试者中，60名完成了临床试验。17名单独接受GH治疗的受试者均完成了试验，身高达到150.4±5.5厘米（均值±标准差），比入组时预测的成人平均身高高8.4±4.5厘米（95%置信区间[CI]：6.3至10.6厘米）。43名接受GH加OX治疗的受试者平均身高为152.1±5.9厘米，比预测的成人平均身高高10.3±4.7厘米（95%CI：8.9至11.7厘米）。历史对照受试者的成人平均身高为144.2±6.0厘米，与最初预测的成人身高144.2±6.1厘米完全匹配。
GH单独使用或联合OX能够刺激TS女孩的短期生长并增加成人身高。通过早期诊断和开始治疗，对于大多数TS女孩来说，超过150厘米的成人身高是一个合理的目标。
