Swendiman Robert A, Coons Barbara E, Alter Craig A, Bamba Vaneeta, Nance Michael L, Vogiatzi Maria G
Department of Surgery, University of Pennsylvania, Philadelphia, PA, US.
Department of Surgery, Columbia University, New York, NY, US.
J Endocr Soc. 2019 Nov 13;4(2):bvz004. doi: 10.1210/jendso/bvz004. eCollection 2020 Feb 1.
Children with congenital adrenal hyperplasia (CAH) because of 21 hydroxylase deficiency (21OHD) are at risk for early or precocious puberty and a short adult height compared to population means and midparental height. The effect of histrelin in suppressing puberty and improving growth in these children has not been reported.
Retrospective cohort analysis of all patients (age ≤ 20) at our institution who underwent histrelin implantation between 2008 and 2017. Treated patients with CAH (classic and nonclassic forms of 21OHD) were identified and their growth data analyzed.
Fifteen children with CAH were treated with histrelin for a median of 3 years (range 2-5; age at first implantation 7.7 ± 1.5 years). Bone age (BA) to chronologic age (CA) decreased from 1.57 ± 0.4 to 1.25 ± 0.25 ( < .01), while predicted adult height (PAH) increased by 7.1 ± 6.6 cm ( < .01). A subgroup of 10 children reached adult height. Similar changes in BA/CA and PAH were observed with therapy ( = .02). Adult height z improved compared to pretreatment PAH z (-1.42 ± 0.9 vs. -1.96 ± 1.1 respectively, < .01), but remained lower than midparental height z ( = .01).
In this retrospective cohort study of children with CAH due to 21OHD and early or precocious puberty, histrelin implantation resulted in a decrease in BA progression compared to CA and an improvement in PAH. In the subgroup who completed growth, adult height remained significantly lower than midparental. These results need to be confirmed with prospective controlled studies.
由于21-羟化酶缺乏(21OHD)导致先天性肾上腺皮质增生(CAH)的儿童,与总体人群均值及父母平均身高相比,有出现性早熟或青春期提前以及成人身高偏矮的风险。尚无关于组氨瑞林对这些儿童抑制青春期发育及促进生长作用的报道。
对2008年至2017年间在我院接受组氨瑞林植入治疗的所有患者(年龄≤20岁)进行回顾性队列分析。确定接受治疗的CAH患者(21OHD的经典型和非经典型),并分析其生长数据。
15例CAH儿童接受了组氨瑞林治疗,中位治疗时间为3年(范围2 - 5年;首次植入时年龄7.7±1.5岁)。骨龄(BA)与实际年龄(CA)的比值从1.57±0.4降至1.25±0.25(P<0.01),而预测成人身高(PAH)增加了7.1±6.6 cm(P<0.01)。10名儿童的亚组达到了成人身高。治疗后观察到BA/CA和PAH有类似变化(P = 0.02)。与治疗前PAH z评分相比,成人身高z评分有所改善(分别为-1.42±0.9和-1.96±1.1,P<0.01),但仍低于父母平均身高z评分(P = 0.01)。
在这项针对因21OHD导致CAH且有性早熟或青春期提前的儿童的回顾性队列研究中,组氨瑞林植入导致BA进展相对于CA有所下降,且PAH有所改善。在完成生长的亚组中,成人身高仍显著低于父母平均身高。这些结果需要通过前瞻性对照研究来证实。