比卡鲁胺联合阿那曲唑治疗睾丸中毒症男孩的促性腺激素非依赖性性早熟：一项II期开放标签试验研究（BATT）
Bicalutamide plus anastrozole for the treatment of gonadotropin-independent precocious puberty in boys with testotoxicosis: a phase II, open-label pilot study (BATT).
作者信息
Reiter Edward O, Mauras Nelly, McCormick Ken, Kulshreshtha Bindu, Amrhein James, De Luca Francesco, O'Brien Sandra, Armstrong Jon, Melezinkova Helena
机构信息
Baystate Children's Hospital, Tufts University School of Medicine, Springfield, MA, USA.
出版信息
J Pediatr Endocrinol Metab. 2010 Oct;23(10):999-1009. doi: 10.1515/jpem.2010.161.
OBJECTIVE
To evaluate the efficacy, tolerability, and pharmacokinetics of bicalutamide plus anastrozole in young males with testotoxicosis.
METHODS
This was a multicenter, open-label, single-arm, 12-month, Phase II pilot trial in 14 males (2-9 years) with testotoxicosis treated with bicalutamide (12.5, 25, 50, or 100 mg) and anastrozole (0.5 or 1 mg) daily. The primary outcome was change in growth rate.
RESULTS
At 1 year, the mean (standard deviation) change from baseline in growth rate was -1.6 (+/- 5.1) cm/year and -0.1 (+/- 1.8) SD units, and in bone maturation was -2.3 (+/- 0.5) years. The bone age/chronological age ratio was reduced from 2.1 (+/- 0.6) at baseline to 1.0 (+/- 0.4) (p = 0.00013). Steady-state trough R-bicalutamide and anastrozole concentrations were attained by Day 21 and 8, respectively. Gynecomastia (42.9%) and breast tenderness (12.5%) were the most common treatment-related adverse events.
CONCLUSIONS
Treatment of testotoxicosis with bicalutamide plus anastrozole resulted in slower growth rate.
目的
评估比卡鲁胺联合阿那曲唑治疗男性性早熟的疗效、耐受性及药代动力学。
方法
这是一项多中心、开放标签、单臂、为期12个月的II期试点试验，14名年龄在2至9岁的男性性早熟患者接受比卡鲁胺（12.5、25、50或100毫克）和阿那曲唑（0.5或1毫克）每日治疗。主要结局指标为生长速率的变化。
结果
1年后，生长速率较基线的平均（标准差）变化为-1.6（±5.1）厘米/年和-0.1（±1.8）标准差单位，骨成熟度变化为-2.3（±0.5）年。骨龄/实际年龄比值从基线时的2.1（±0.6）降至1.0（±0.4）（p = 0.00013）。分别在第21天和第8天达到比卡鲁胺和阿那曲唑的稳态谷浓度。男性乳房发育（42.9%）和乳房压痛（12.5%）是最常见的与治疗相关的不良事件。
结论
比卡鲁胺联合阿那曲唑治疗男性性早熟导致生长速率减慢。
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