Zegarra Walter, Ranadive Sayali, Toulan Diane, Neely E Kirk
Division of Pediatric Endocrinology and Diabetes, Stanford University School of Medicine, Stanford, CA 94305-5660, USA.
Pediatric Endocrinology, Palo Alto Medical Foundation, Palo Alto, CA 94301, USA.
J Endocr Soc. 2024 Aug 27;8(10):bvae141. doi: 10.1210/jendso/bvae141.
Insufficient efficacy and safety data for off-label use of aromatase inhibitors to augment height in boys with short stature.
To compare anastrozole and letrozole in treatment of idiopathic short stature in pubertal boys.
Open-label trial with 2 treatment arms.
Pediatric Endocrine Clinic at Stanford.
A total of 79 pubertal males ≥10 years with bone age (BA) ≤ 14 years, predicted adult height (PAH) < 5th percentile or >10 cm below mid-parental height.
Anastrozole 1.0 mg or letrozole 2.5 mg daily for up to 3 years.
Annual hormone levels and growth parameters during treatment and a year posttherapy; annual BA and PAH (primary outcome measure); spine x-rays and dual energy X-ray absorptiometry at baseline and 2 years.
Compared with anastrozole (n = 35), letrozole (n = 30) resulted in higher testosterone levels, lower estradiol and IGF-1 levels, and slower growth velocity and BA advance. The PAH increase observed at year 1 in both groups did not persist at years 2 and 3. Change in PAH from baseline was not different between treatment groups. In groups combined, PAH gain over 3 years vs baseline was +1.3 cm ( = .043) in linear mixed models.
Letrozole caused greater deviations than anastrozole in hormone levels, growth velocity, and BA advancement, but no group differences in PAH or side effects were found. Change in PAH after 2 to 3 years of treatment was minimal. The efficacy of AI as monotherapy for height augmentation in pubertal boys with idiopathic short stature may be limited, and safety remains an issue.
芳香化酶抑制剂用于身材矮小男孩增高的标签外使用的疗效和安全性数据不足。
比较阿那曲唑和来曲唑治疗青春期男孩特发性身材矮小的效果。
开放标签试验，设2个治疗组。
斯坦福大学儿科内分泌诊所。
共79名年龄≥10岁的青春期男性，骨龄（BA）≤14岁，预测成年身高（PAH）低于第5百分位数或比父母平均身高低10 cm以上。
阿那曲唑1.0 mg或来曲唑2.5 mg，每日1次，治疗长达3年。
治疗期间及治疗后1年的年度激素水平和生长参数；年度BA和PAH（主要观察指标）；基线及2年时的脊柱X光片和双能X线吸收测定法。
与阿那曲唑组（n = 35）相比，来曲唑组（n = 30）的睾酮水平更高，雌二醇和IGF-1水平更低，生长速度和骨龄进展更慢。两组在第1年观察到的PAH增加在第2年和第3年没有持续。治疗组间PAH相对于基线的变化没有差异。综合两组，线性混合模型显示3年期间PAH相对于基线增加了1.3 cm（P = 0.043）。
来曲唑在激素水平、生长速度和骨龄进展方面比阿那曲唑引起的偏差更大，但在PAH或副作用方面未发现组间差异。治疗2至3年后PAH的变化很小。芳香化酶抑制剂作为青春期特发性身材矮小男孩增高单一疗法的疗效可能有限，安全性仍是一个问题。
