Background: Growth hormone (GH) deficiency(GHD) in childhood is associated with impairment in linear growth. GH therapy enables the achievement of normal adult height in most cases. The response is variable and factors influencing height outcome are still not clearly defined.
Objective: To evaluate near adult height (NAH) in a single center cohort of childhood onset GHD patients treated with GH and investigate main predictors of final height (FH).
Patients and methods: 80 GHD-patients (41M-39F) followed from the diagnosis to the achievement of NAH were enrolled. Mean age at diagnosis was 9.9±4.0 years. 79% of patients (63/80) had isolated GHD (IGHD), 21% (17/80) had multiple pituitary hormone deficiency(MPHD) and received hormone replacement as necessary. Structural abnormalities of the pituitary gland were detected in 40 patients. Height, Height velocity (HV) and IGF-1 were evaluated at diagnosis, at the end of the first year of treatment, at onset of puberty and at the attainment of NAH and were expressed as standard deviation score (SDS) according to reference standards. Change in HSDS (Δ) from baseline to the different end points was calculated.Multiple regression analysis was used to evaluate predictor of FH.
Results: At diagnosis HSDS (−2.53±0.94), HVSDS (−2.47±1.69) and IGF1SDS (−1.23±1.24) were below normal ranges. After 1 year of GH, HSDS (−1.86±0.84, P<0.0001), HVSDS (3.03±2.79, P<0.0001) and IGFISDS (0.34±1.24, P<0.0001) significantly improved. At puberty onset, mean age of patients was 12.52±1.67 years. ΔHSDS from baseline to pubertal onset was significantly higher in MPHD vs IGHD (3.03±1.66 vs 0.6±0.68, P<0.0001) while was comparable between males and females. NAHSDS (−0.87±0.98), achieved at a mean age of 16.94±1.51 years, was significantly higher compared to baseline (P<0.0001), and was higher in patients with MPHD vs patients with IGHD (−0.47±1.15 vs −0.98±0.91, P<0.05). Although NAHSDS was higher in males vs females (−0.65±0.9 vs −1.11±1.02, P<0.05), the total gain in HSDS was comparable between the two sexes (1.66±0.91 vs 1.64±1.09, P=ns). Multiple regression analysis showed that NAH correlated with sex (P<0.05), severity of GHD (P<0.05), diagnosis (P<0.0001), age at diagnosis (P<0.0001), HV in the 1° year of treatment (P<0.0001), H at onset of puberty (P<0.0001), prepubertal ΔHSDS (P<0.0001) and duration of treatment (P<0.0001).
Conclusion: GH treatment enables the achievement of normal NAH.Early treatment and optimization of prepubertal growth are important to obtain a better growth response. Patients with severeGHD and MPHD have a greater gain in height and NAH than patients with mildGHD and IGHD.
18 - 21 May 2019
European Society of Endocrinology