Introduction: Peripubertal children with delayed puberty frequently display a poor growth rate prompting endocrine work-up. Whether priming with sex steroids should routinely be performed in these patients to improve specificity of growth hormone stimulation tests (GHST) is unclear. Treatment with sex steroids in constitutional delay of growth and puberty (CDGP) is also debated.
Patients and Methods: This multicenter retrospective study included 151 normal weight children (54 females) presenting with pubertal delay and short stature/poor growth rate (aged 12.55±1.82 years). Patients were diagnosed as CDGP if GH peak after a GHST was ≥ 8 µg/L or isolated growth hormone deficiency (IGHD) if < 8 µg/L. CDGP patients received low-dose sex steroids (LDSS) for 3-51 months, or no treatment. All IGHD patients received rhGH (25-35 µg/kg/day). Within the IGHD cohort, 77/85 were retested at final height (FH). Patients were divided into 6 groups: untreated CDGP diagnosed with or without priming (1A n = 31; 2A = 21), CDGP receiving LDSS diagnosed with or without priming (1B = 12; 2B = 2), IGHD diagnosed with or without priming (1C = 42; 2C = 43). Standard deviation score (SDS) FH, Δ SDS FH- SDS target height (TH) and degree of success (defined as - 1 ≤Δ SDS FH- SDS TH ≤ 1) were outcomes of interest.
Results: SDS FH was higher in group 1C than 1A (-0.86 vs -1.43, P= 0.014) and similar trend was found for ΔSDS FH-TH (1C -0.07 vs 1A -0.74; P = 0.005). SDS FH and ΔSDS FH-TH were comparable between groups 2C and 2A (-0.93 vs -0.99, P= 0.85; -0.31 vs -0.46, P=0.508).
IGHD patients showed the highest degree of success [group C (1C + 2C) 89% vs group B (1B + 2 B) 86% vs group A (1A+2A) 63% P= 0.0009], and above all group 1C (1C 93%, 2C 86%, 1B 83%, 1A 68%, 2A 57%, P= 0.005).
At retesting, a higher proportion of permanent IGHD was documented in group 1C (1C 27% vs 2C 17.5%; N.S.), not reaching statistical significance possibly due to low sample size.
Conclusion: Our data suggest that priming with sex steroids in peripubertal short subjects improves the ability to select those patients who would more likely benefit from rhGH therapy. Indeed, rhGH treatment in IGHD seems to give advantages in terms of final height compared to untreated CDGP especially in those patients diagnosed upon a primed GHST.
19 - 21 Sep 2019
European Society for Paediatric Endocrinology