ESPE Abstracts

Keywords: growth hormone, small for gestational age, genetic defects; Silver-Russell syndrome; growth hormone deficiency

Objectives: To evaluate the influencing factors of GH treatment in Chinese short stature children born SGA.

Methods: This was a single-center, retrospective, cross-sectional survey in China. Of 101 patients were identified born SGA and 66 short stature children born SGA were participant in the GH therapy. Forty-one participants were receiving GH treatment and complete at least 6-month of follow-up. Main outcome measures were height standard deviation score (HTSDS), heigh, growth velocity (GV), and change of height standard deviation score (△HTSDS). HTSDS was converted by using Chinese reference data. The generalized estimating equation (GEE) was used to identified the potential factors, including GH treatment time, age at GH therapy initiation, sex, SGA related gene mutation, GH deficiency and birth weight, that influencing the effectiveness of GH treatment. Enrolled patients born SGA were categorized into three age groups based on their age at GH treatment initiation (2 to <4 years, 4 to <6 years, and ≥ 6 years). SGA related gene mutations and so as whether the gene mutation was belonged to Silver-Russell syndrome, whether patients were associated with GH deficiency (<10 ng/mL) were also used to evaluate the impact on GH efficacy.

Results: The mean age at GH treatment initiation was 5.6 years old. Results from GEE indicated that GH treatment time were significantly affected HTSDS, height, GV, and ΔHTSDS among patients born SGA. The increase in GV caused by GH treatment was highest in the first half of the year and gradually weakened later. SGA related gene mutation was significantly affected GV (β=-1.25, 95% confidence interval, CI: -2.38, -0.12), and GH deficiency was significantly affected both GV and ΔHTSDS (β=-1.40, 95% CI: -2.21, -0.58, and β=-0.36, 95% CI: -0.66, -0.07, respectively). There was no statistical difference in GV or ΔHTSDS comparison between gene mutation and normal, or SRS gene mutations and not at a single time point. Patients who associated with GH deficiency had significantly higher ΔHTSDS after 2 years and 2.5 years of GH therapy.

Conclusions: Chinese short stature children born SGA without SGA related gene mutations, and those with natural GH secretion deficiency, may have better GH efficacy that those with gene defect and normal natural GH secretion. *Correspondence: Feihong Luo, luofh@fudan.edu.cn