Evaluation of 451 Danish Boys With Delayed Puberty: Diagnostic Use of a New Puberty Nomogram and Effects of Oral Testosterone Therapy

Evaluation of 451 Danish Boys With Delayed Puberty: Diagnostic Use of a New Puberty Nomogram and Effects of Oral Testosterone Therapy
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DOI:
10.1210/jc.2014-3631
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发表时间:
2015-04-01
影响因子:
5.8
通讯作者:
Juul, Anders
Juul, Anders
中科院分区:
医学2区
文献类型:
--
作者:
Lawaetz, Jacob Gerner;Hagen, Casper P.;Juul, Anders

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背景:关于发育和青春期体质延迟(CDGP)男孩的诊断标准和青春期诱导作用的数据很少。目的:建立丹麦青春期发育正常男孩的青春期形态图。探讨口服十一酸睾酮(TU)对男孩CDGP的不同诊断标准及疗效。设计:对丹麦青春期发育正常的男孩进行横断面和纵向研究(COPENHAGEN青春期研究)。一项回顾性观察研究对1990年至2013年间451名男孩的青春期延迟进行了评估。机构:儿科内分泌科三级转诊中心。参与者:164名(36%)接受CDGP评估的男孩因缺少数据、重新分类或相关合并症而被排除,有287名(64%)符合分析条件。主要结局指标:用青春期nomogram(生殖器分期<年龄-2 SD)和经典标准(生殖器分期≥14岁)对CDGP患者进行分类的人数。一年口服TU治疗对青春期发展、循环激素、身高和预测成人身高(PAH)的影响。结果:根据经典标准,287名男孩中有78名(27%)存在青春期延迟,而根据青春期nomogram,有173名(60%)存在青春期发育障碍。这173名男孩中有96名(56%)接受口服TU治疗0.8年(0.5;1.3)[中位数(25;75百分位数)],对青春期发育产生了有益的影响。治疗1年后,身高从-1.9 SD(-2.5; -1.2)增加到-1.5 SD (-2.1; -0.7) (P < 0.001), PAH从172.3 cm(170.3; 182.8)增加到178.1 cm (171.4; 191.7) (P = 0.001)。结论:青春期形态图既可以评估青春期延迟开始,也可以评估青春期延迟进展,并可以区分正常和异常的青春期发育。口服TU治疗后,在不影响最终身高的情况下,青春期诱导和发展以及短期生长。
Context: Few data exist on the diagnostic criteria, and on the effects of puberty induction, in boys with constitutional delay in growth and puberty (CDGP).Objective: To develop puberty nomograms based on Danish boys with normal pubertal development. To evaluate the different diagnostic criteria and the effect of oral testosterone undecanoate (TU) in boys with CDGP.Design: Across-sectional and longitudinal study of Danish boys with normal pubertal development (COPENHAGEN puberty study). A retrospective observational study of 451 boys evaluated for delayed puberty between 1990 and 2013.Setting: Tertiary referral center for pediatric endocrinology.Participants: One hundred and sixty-four (36%) boys evaluated for CDGP were excluded due to missing data, reclassification, or associated comorbidities, yielding 287 (64%) eligible for analysis.Main Outcome Measures: The number of patients with CDGP classified by the puberty nomogram (genital stage < -2 SD for age) versus the classical criteria (genital stage 1 at >= 14 years). The effect of one year of oral TU treatment on pubertal progression, circulating hormones, height, and predicted adult height (PAH).Results: Seventy-eight (27%) of the 287 boys had delayed pubertal onset according to the classical criteria, whereas 173 (60%) of the 287 boys had impaired pubertal progression according to the puberty nomogram. Ninety-six (56%) of these 173 boys were treated with oral TU for 0.8 years (0.5; 1.3) [median (25th; 75th percentiles)], which resulted in beneficial effects on pubertal progression. Height increased from -1.9 SD (-2.5; -1.2) to -1.5 SD (-2.1; -0.7) (P < .001), and PAH increased from 172.3 cm (170.3; 182.8) to 178.1 cm (171.4; 191.7) (P = .001) following one year of treatment.Conclusions: The puberty nomogram evaluates both delayed pubertal onset as well as delayed pubertal progression and allows separation of normal versus abnormal pubertal development. Oral TU treatment was followed by pubertal induction and progression and short-term growth without compromising final height.