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J Zhejiang Univ (Med Sci)
Efficacy and safety of letrozole in treatment of McCune-Albright Syndrome girls with peripheral precocious puberty
XU De, LU Wenli, WANG Xueqing, WANG Junqi, XIE Yiwen, DONG Zhiya, WANG Wei
Department of Pediatrics, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China
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Abstract  Objective: To evaluate the efficacy and safety of the third-generation aromatase inhibitor letrozole in the treatment of McCune-Albright syndrome (MAS) girls with peripheral precocious puberty. Methods: Twenty-one MAS girls with peripheral precocious puberty treated in Pediatrics Department of Ruijin Hospital, Shanghai Jiao Tong University School of Medicine from March 2012 to June 2017 were enrolled in the study. Patients presented with repeated vaginal bleeding, premature breast enlargement, café-au-lait spots or dysplasia of bone fibers, and low levels of LH and FSH; and the congenital adrenal hyperplasia, estrogen-producing tumors, and exogenous estrogen intake were excluded. Letrozole were administrated at a dose of 0.5-2mg/m2·d for 6 to 12 months. The patients were observed for changes in breast staging, vaginal bleeding, sex hormone levels, liver function and bone age changes, and changes in uterine and ovarian volume. Results: After treatment, bone age/chronological age (BA/CA)was decreased from 1.23±0.30 to 1.11±0.18 (P=0.005); the predicted adult height (PAH) increased from 156.18 ±5.86 cm to 157.4 ±2. 06 cm after treatment (P=0.023), the E2 level decreased significantly, while the teststosterone (T) level did not increase significantly and the uterus showed no significant increase, and no adverse reactions such as ovarian torsion occurred. Conclusion: Precocious puberty is one of the most common endocrine manifestations in MAS. Our findings suggest that letrozole may be an effective and safe therapy to precocious puberty in girls with McCune-Albright Syndrome.

Key wordsPuberty, precocious      McCune-Albright syndrome      Aromatase inhibitors      Letrozole      Girls     
Received: 05 December 2019      Published: 29 May 2020
CLC:  R588  
Cite this article:

XU De, LU Wenli, WANG Xueqing, WANG Junqi, XIE Yiwen, DONG Zhiya, WANG Wei. Efficacy and safety of letrozole in treatment of McCune-Albright Syndrome girls with peripheral precocious puberty. J Zhejiang Univ (Med Sci), 0, 0(0): 3-0.

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目的:探讨应用第三代芳香化酶抑制剂来曲唑治疗纤维性骨营养不良综合征(MAS)女童外周性性早熟的疗效及安全性。方法:选取2012年3月至2017年6月上海交通大学医学院附属瑞金医院儿科的MAS女童21例。患儿反复阴道出血、乳房增大同时伴或不伴牛奶咖啡斑或骨纤维发育不良,LH和FSH水平低下,排除先天性肾上腺皮质增生症、分泌雌激素的肿瘤及外源性雌激素摄入所致等情况。给予来曲唑0.5~2 mg·(m2·d)治疗,疗程6~12个月,观察患儿乳房分期变化、阴道出血次数、性激素水平、肝功能及骨龄变化及子宫卵巢容积变化及有无不良事件发生。结果:经过来曲唑治疗后,患儿骨龄/实际年龄放缓(1.23±0.30降至1.11±0.18,P<0.01);患儿预测成人身高从治疗前的(156.2±5.9)cm增加至治疗后的(158.4±2.1)cm(P<0.05);患儿阴道出血次数明显减少,雌二醇水平下降,睾酮未见明显升高;治疗后患儿子宫未见明显增大,也未发生卵巢扭转及肝酶升高等不良反应。结论:儿童MAS的内分泌表现以性早熟最为多见,第三代芳香化酶抑制剂来曲唑治疗MAS相关的外周性性早熟可延缓骨龄进展,减少阴道出血的频率,也未出现肝酶升高等肝功能受损的不良反应。

关键词: 青春期,早熟,  纤维性骨营养不良综合征,  芳香化酶抑制剂,  来曲唑,  女童 
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