ECEESPE2025 ePoster Presentations Growth Axis and Syndromes (132 abstracts)
1Department of Pediatrics of Second Faculty of Medicine Charles University in Prague and Motol University Hospital, Prague, Czech Republic
JOINT2470
Introduction: Growth hormone (GH) stimulation tests that are used as a golden standard for diagnosis of GH deficiency (GHD) are known to have poor reliability. Children with diagnosed GHD are therefore believed to have heterogeneous etiology of their short stature and consequently heterogeneous response to GH treatment. The aim of the study was to analyze the response to GH therapy in a single center cohort and to search for the factors predicting treatment outcomes.
Methods: Children treated with idiopathic GHD in our center that achieved their final height in years 2022-2024 were enrolled to the study. The diagnosis of GHD was made according to the current guidelines, maximal stimulated concentration of GH (GHmax) <10 mg/l in both clonidine and insulin hypoglycemia tests were used to confirm the diagnosis. To search for factors affecting response to GH treatment, correlation analysis and ANOVA Kruskal-Wallis test were used for continuous and categorical variables, respectively. P-values <0.05 were considered significant.
Results: In total, 138 children diagnosed with GHD and available final height were enrolled to the analysis. At GH treatment initiation, their median age was 6.1 years (IQR 4.1-7.8 years), height-SDS -2.8 (-3.2 to -2.5), IGF-1-SDS -1.5 (-1.8 to -1.1) and GHmax 5.9 (4.5-7.6) mg/L. Sixteen children had combined pituitary hormones deficiency (CPHD), 11 anterior pituitary hypoplasia and 26 more severe abnormal cerebral midline anatomy on MRI. The children were treated with GH for 8.8 (7.2-10.6) years with an average dose 31 (24-33) mg/kg/day. The height SDS increased by 1.3 (0.8-1.9) SD to an adult height -1.5 (-2.1 to -0.9) SD. The height SDS improvement showed a weak correlation with maximal stimulated GH concentration (correlation coefficient [CC] -0,21; P = 0.014). Children with GHmax <3 mg/L showed better height SDS improvement compared to those with GHmax ≥3 mg/L (2.3 vs 1.3 SD, P = 0.008), but, interestingly, no correlation between GHmax and height SDS improvement was found in children with GHmax >3 mg/L (CC 0.06, P = 0.51). Furthermore, height SDS gain weakly correlated with pre-treatment IGF-1 concentration (CC -0,24; P = 0.006) and was higher in children with CPHD (P = 0.0009). No correlation between effect of GH treatment and birth parameters, parents height and brain MRI was found.
Conclusions: Height outcomes following GH treatment in children with idiopathic GHD had great interindividual variability. Better treatment outcomes were associated with lower GHmax and pre-treatment IGF-1 concentration and presence of CPHD. No correlation between height improvement and GHmax beyond 3 mg/L was observed.