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J Zhejiang Univ (Med Sci)
    
Efficacy of letrozole in treatment of children with congenital adrenal hyperplasia due to steroid 21-hydroxylase deficiency
WANG Qian1, ZHANG Shule2, MA Xue2, LI Guimei1, WANG Zengmin1, WANG Fengxue1
1. Department of Pediatric Endocrine, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan 250014, China;
2. Department of Pediatric Endocrine, Shandong Provincial Hospital Affiliated to Shandong University, Jinan 250014, China
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Abstract  Objective: To assess the efficacy of letrozole (Lz) in treatment of children with congenital adrenal hyperplasia (CAH) due to steroid 21-hydroxylase deficiency (21-OHD). Methods: Twenty eight children, including 19 boys and 9 girls aged 4-10y, with CAH due to 21-OHD were enrolled in the study. At the first six months of study, all children received conventional treatment with hydrocortisone (HC) and fludrocortisone, then letrozole was added to original regimen and used for 12 months. The height velocity (HV), difference between bone age and chronological age (BA-CA), height standard diviation score (Ht-SDSBA), PAH (predicted adult height), Tanner phase, sex hormone, and possible adverse reaction were evaluated and compared between those before and after letrozole treatment. Results: There was significant increase of Ht-SDSBA after 12 months of Lz-treated phase (P=0.036,0.017,0.004) and PAH of female children was significantly increased in the Lz-treated phase (P=0.017, 0.006, 0.002), whereas PAH of male children was significantly increased 18 months after Lz-treatment (P=0.02,0.003). No changes occurred in HV. Levels of FSH and LH were significantly increased (P=0.01, 0.04); and E2 was significantly decreased (P=0.002); no changes in TO or possible adverse reactions. Conclusion: Treatment with the aromatase inhibitor letrozole delays bone maturation and improves PAH in children with CAH due to 21-OHD. No adverse effects on bone mineralization are observed after 2 years of treatment.

Key wordsAdrenal hyperplasia, congenital      21-hydroxylase deficiency      Child      Aromatase inhibitors      Predicted adult height      Bone age      Adverse reaction     
Received: 04 December 2019      Published: 29 May 2020
CLC:  R588  
  R725.8  
Cite this article:

WANG Qian, ZHANG Shule, MA Xue, LI Guimei, WANG Zengmin, WANG Fengxue. Efficacy of letrozole in treatment of children with congenital adrenal hyperplasia due to steroid 21-hydroxylase deficiency. J Zhejiang Univ (Med Sci), 0, 0(0): 4-0.

URL:

http://www.zjujournals.com/med/10.3785/j.issn.1008-9292.2020.04.04     OR     http://www.zjujournals.com/med/Y0/V0/I0/4


来曲唑对21-羟化酶缺乏型先天性肾上腺皮质增生症患儿生长发育的影响

目的:探讨来曲唑联合常规方法治疗21-羟化酶缺乏型(21-OHD)先天性肾上腺皮质增生症(CAH)的可行性及合理治疗方案。方法:选取2010年.12月至2016年1月确诊的4~10岁的21-羟化酶缺乏型CAH患儿共28例,其中女性患儿9例。入组患儿前6个月为常规治疗期(氢化可的松、氟氢可的松),第6个月末开始加用来曲唑治疗。通过自身前后对照的方法,对加用来曲唑联合治疗前后患儿的肾上腺皮质激素替代剂量、骨龄进展速度、预期成年身高(PAH)、性征发育程度及性激素水平、可能引起的不良反应等指标进行统计。结果:加用来曲唑治疗后,相比于单用氢化可的松、氟氢可的松的常规治疗,患儿生长速度在短期(6个月内)有所下降,但很快恢复正常;联合治疗12个月后(入组18个月末)患儿Ht-SDSBA较入组时及常规治疗时均显著改善(P<0.05或P<0.01);骨龄与实际年龄差值(BA-CA)至联合治疗18个月(24个月末)较常规治疗时显著改善;女性患儿PAH自联合治疗12个月(入组18个月末)较常规治疗期显著改善(P<0.05或P<0.01),男性患儿组直至联合治疗18个月后(入组24个月末)PAH亦显著改善(P<0.05或P<0.01);患儿卵泡刺激素、促黄体生成素升高,但未达到中枢性性早熟标准;患儿雌二醇水平降低(P<0.01),考虑与加用来曲唑有关;患儿睾酮水平无显著改变。来曲唑联合治疗24个月,患儿未见多毛、严重痤疮、头疼、骨痛、肥胖、高血压、皮疹等不良反应。结论:来曲唑联合常规治疗的方案可用于21-OHD型CAH患儿长期治疗,特别是骨龄明显超期、预期成年身高受损的患儿,可实现抑制骨龄进展、改善预期成年身高的治疗目的。

关键词: 肾上腺增生,先天性,  21-羟化酶缺乏症,  儿童,  芳香化酶抑制剂,  预期成年身高,  骨龄,  不良反应 
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