Arroyo Ruiz Ramón, Ballester Pérez Aránzazu, Leiva-Gea Isabel, Martínez-Aedo MªJosé, López-Siguero Juan Pedro
Department of Pediatric Endocrinology, Hospital Regional Universitario de Málaga, Málaga, Spain.
Department of Pediatric Endocrinology, Hospital Regional Universitario de Málaga, 29010 Málaga, Spain.
Ther Adv Endocrinol Metab. 2022 Apr 2;13:20420188221083534. doi: 10.1177/20420188221083534. eCollection 2022.
The objective was to analyze the efficacy of recombinant human growth hormone (rhGH) treatment in children born small for gestational age (SGA) without catch-up growth treated before the onset of puberty, with follow-up until adult height. The influence of demographic and auxological factors on the final response evaluated as adult height and height gain was assessed.
A prospective longitudinal observational study performed in a tertiary hospital, involving SGA patients, who started treatment with rhGH between October 2003 and April 2015. Potential response predictors were evaluated by multiple regression analysis and receiver operating characteristic curves.
Of the initial 96 patients included, 61 patients (28 boys and 33 girls) reached adult height. Adult height gain in standard deviation (SDS) was 0.99 (0.8) and 1.49 (0.94), respectively ( < 0.05). An adult height greater than -2 SDS was reached in 75% of the girls but only in 53% of the boys. The pubertal height gain was 22.6 (5.8) cm in boys and 18.8 (4.5) cm in girls. The multiple regression model obtained for total height gain explained 42% of the variability in this variable including sex, height gain during the first year, and the difference from target height at the start of treatment. A first-year height gain of 0.69 SDS was the optimal point for assessing a final height gain greater than 1.5 SDS with a specificity of 70% and a sensitivity of 71%.
Most SGA patients achieve normalization of height above -2 SD, the percentage being higher in girls. According to our predictive model, height gain in the first year is the most important variable for predicting good response to treatment. During puberty, there is a loss of height SDS, probably due to a lower total pubertal gain with respect to the reference population, which is more marked in boys.
本研究旨在分析重组人生长激素(rhGH)治疗青春期前出生时小于胎龄(SGA)且无追赶生长的儿童的疗效,并随访至成年身高。评估人口统计学和人体测量学因素对以成年身高和身高增长评估的最终反应的影响。
在一家三级医院进行的前瞻性纵向观察研究,纳入2003年10月至2015年4月期间开始接受rhGH治疗的SGA患者。通过多元回归分析和受试者工作特征曲线评估潜在的反应预测因素。
最初纳入的96例患者中,61例患者(28例男孩和33例女孩)达到成年身高。成年身高标准差(SDS)增加分别为0.99(0.8)和1.49(0.94)(P<0.05)。75%的女孩成年身高大于-2 SDS,而男孩仅为53%。男孩青春期身高增长为22.6(5.8)cm,女孩为18.8(4.5)cm。获得的总身高增长多元回归模型解释了该变量42%的变异性,包括性别、第一年身高增长以及治疗开始时与目标身高的差异。第一年身高增长0.69 SDS是评估最终身高增长大于1.5 SDS的最佳点,特异性为70%,敏感性为71%。
大多数SGA患者身高达到高于-2 SD的正常水平,女孩的比例更高。根据我们的预测模型,第一年的身高增长是预测治疗良好反应的最重要变量。在青春期,身高SDS有所下降,可能是由于相对于参考人群青春期总增长较低,这在男孩中更为明显。