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J Zhejiang Univ (Med Sci)
Efficacy and safety of aromatase inhibitor letrozole in treatment of male children with disorders of sex development
YU Bingqing, NIE Min, WU Xueyan, MAO Jiangfeng, WANG Xi, MA Wanlu, JI Wen, HUANG Qibin, ZHANG Rui
Department of Endocrinology, National Health Commission Key laboratory of Endocrinology, Peking Union Medical College Hospital, PekingUnion Medical College, Chinese Academy ofMedical Sciences, Beijing, China
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Abstract  Objective: To investigate the efficacy and safety of aromatase inhibitor letrozole in treatment of male children with disorders of sex development (DSD). Methods: Clinical data of 12 male DSD children with a mean age of 14.6 ±2.5 years admitted to Peking Union Medical College Hospital from January 2014 to January 2016 were retrospectively analyzed. The patients treated with treated with letrozole (1.25~2.5mg, q.d) for 3 months or longer, and followed up for 0.5~2.5 years. Clinical manifestation and laboratory test finding were documented and the efficacy and safety were evaluated. Results: The baseline luteinizing hormone (LH) level before treatment was 5.2 (3.7, 16.5) IU/L, follicle-stimulating hormone(FSH) level was 14.1 (5.3, 24.7) IU/L, testosterone level was 5.2 (3.8, 10.0) nmol/L, and E2 level was (120.7±51.0)pmol/L. After half a year of treatment, the blood LH, FSH and testosterone levels of patients increased (all P<0.05), and estrogen levels decreased from baseline (P<0.05). After 1 year of treatment, the blood testosterone level was significantly higher than that before treatment (P<0.05); the LH and FSH levels tended to increase and the estrogen level tended to decrease than the baseline, but there was no significant statistical difference (P>0.05). Semen was routinely detected in 8 patients, and sperms were detected in semen of 3 patients with hypospadias. There were no significant adverse events in all patients during the treatment, and no significant changes in biochemical results after treatment. Conclusion: Aromatase inhibitor letrozole can effectively increase testosterone levels in patients with disorders of sex development and promote spermatogenesis, it has no significant adverse effects in short-term administration.

Key wordsDisorders of sex development/drug therapy      Aromatase inhibitors/therapeutic use      Testosterone      Estrogens      Child     
Received: 15 December 2019      Published: 29 May 2020
CLC:  R588  
Cite this article:

YU Bingqing, NIE Min, WU Xueyan, MAO Jiangfeng, WANG Xi, MA Wanlu, JI Wen, HUANG Qibin, ZHANG Rui. Efficacy and safety of aromatase inhibitor letrozole in treatment of male children with disorders of sex development. J Zhejiang Univ (Med Sci), 0, 0(0): 2-0.

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目的:评价芳香化酶抑制剂来曲唑治疗男性性发育异常(DSD)患儿的疗效和安全性。方法:收集2014年1月至2016年1月就诊于北京协和医院内分泌科门诊的男性DSD患儿12例,其中来曲唑治疗≥ 3个月的患者采集其临床资料及实验室检查结果,比较治疗前后激素水平及生化指标的差异,并观察治疗中的不良反应。结果:本组患儿来曲唑治疗的平均年龄为(14.6±2.5)岁。来曲唑治疗半年后患者血促性腺激素黄体生成素(LH)、卵泡刺激素(FSH)及睾酮水平较基线升高(均P<0.05),雌二醇水平较基线下降(P<0.05);治疗1年后患者睾酮水平较基线高(P<0.05),LH及FSH水平较基线高,雌二醇水平较基线下降,但差异无统计学意义(均P>0.05)。8例患者进行精液常规检测,其中3例尿道下裂患者精液中可检测到精子。所有患者用药后无明显不良反应,监测治疗前后生化检查无明显变化。结论:来曲唑可有效升高性发育异常患者的睾酮水平,促进精子生成,且短期应用未见明显不良反应。

关键词: 性发育障碍/药物疗法,  芳香酶抑制剂/治疗应用,  睾酮,  雌激素类,  儿童 
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