Bizzarri Carla, Improda Nicola, Maggioli Chiara, Capalbo Donatella, Roma Silvia, Porzio Ottavia, Salerno Mariacarolina, Cappa Marco
Endocr Pract. 2017 May;23(5):546-556. doi: 10.4158/EP171751.OR. Epub 2017 Feb 22.
Poor linear growth is one of the main concerns in children with congenital adrenal hyperplasia (CAH). We aimed to analyze factors affecting growth trajectory in children with classical CAH.
Clinical records of children followed from infancy up to the end of growth at two Italian tertiary referral hospitals were reviewed. Fifty-seven patients (31 males), treated with hydrocortisone and fludrocortisone only, were included. Clinical observations were divided into three groups: 0 to 2 years, 172 observations; from 2 years to puberty onset, 813 observations; after puberty onset, 527 observations. Height velocity, pubertal growth spurt, and final height were evaluated as outcomes.
Final height standard deviation score (SDS) was lower than target height SDS (-0.74 ± 1.1 versus -0.31 ± 1.01; P<.001). Target-adjusted final height SDS was -0.44 ± 1.8 in males and -0.13 ± 1.1 in females (P = .001). Total pubertal growth was 21.9 ± 7.3 cm in males and 19.2 ± 8.2 cm in females (P = .19). Hydrocortisone dose increased and height-velocity SDS decreased during puberty. At multivariable analysis, height-velocity SDS was adversely affected by hydrocortisone dose (P = .038) and directly related to adrenocorticotropic hormone (ACTH) levels (P = .023). Target-adjusted final-height SDS was adversely affected by hydrocortisone dose (P<.001) and positively related to mineralocorticoid therapy (P = .001) and ACTH levels (P = .02). Total pubertal growth (cm) was positively related to ACTH levels (P = .01).
Height outcome of CAH patients is now better than previously reported. During puberty, the lowest effective dose of hydrocortisone should be used to optimize pubertal growth spurt and final height.
17-OHP = 17-alpha-hydroxyprogesterone ACTH = adrenocorticotropic hormone BMI = body mass index CAH = congenital adrenal hyperplasia GH = growth hormone HPA = hypothalamus-pituitary-adrenal PRA = plasma renin activity SDS = standard deviation score SV = simple virilizing SW = salt-wasting.
生长发育迟缓是先天性肾上腺皮质增生症（CAH）患儿的主要问题之一。我们旨在分析影响经典型CAH患儿生长轨迹的因素。
回顾了两家意大利三级转诊医院从婴儿期到生长结束的患儿临床记录。纳入仅接受氢化可的松和氟氢可的松治疗的57例患者（31例男性）。临床观察分为三组：0至2岁，172次观察；2岁至青春期开始，813次观察；青春期开始后，527次观察。将身高增长速度、青春期生长突增和最终身高作为观察指标。
最终身高标准差评分（SDS）低于目标身高SDS（-0.74±1.1对-0.31±1.01；P<0.001）。目标校正后的最终身高SDS男性为-0.44±1.8，女性为-0.13±1.1（P = 0.001）。男性青春期总生长量为21.9±7.3 cm，女性为19.2±8.2 cm（P = 0.19）。青春期氢化可的松剂量增加，身高增长速度SDS下降。多变量分析显示，身高增长速度SDS受氢化可的松剂量的负面影响（P = 0.038），且与促肾上腺皮质激素（ACTH）水平直接相关（P = 0.023）。目标校正后的最终身高SDS受氢化可的松剂量的负面影响（P<0.001），与盐皮质激素治疗（P = 0.001）和ACTH水平呈正相关（P = 0.02）。青春期总生长量（cm）与ACTH水平呈正相关（P = 0.01）。
CAH患者的身高结局目前优于先前报道。青春期应使用最低有效剂量的氢化可的松，以优化青春期生长突增和最终身高。
17-OHP = 17-α-羟孕酮；ACTH = 促肾上腺皮质激素；BMI = 体重指数；CAH = 先天性肾上腺皮质增生症；GH = 生长激素；HPA = 下丘脑-垂体-肾上腺；PRA = 血浆肾素活性；SDS = 标准差评分；SV = 单纯男性化型；SW = 失盐型
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