Basal characteristics and first year responses to human growth hormone (GH) vary according to diagnostic criteria in children with non-acquired GH deficiency (naGHD): observations from a single center over a period of five decades.

Background Children with non-acquired (na) growth hormone deficiency (GHD) diagnosed over decades in one center may provide perspective insight. Methods naGHD is divided into idiopathic GHD (IGHD), GHD of known cause (cGHD) and GHD neurosecretory dysfunction (NSD); time periods: <1988 (I); 1988-1997 (II); 1998-2007 (III); 2008-2015 (IV). Descriptive analyses were performed at diagnosis and during first year GH treatment. Results Patients (periods, N): I, 87; II, 141; III, 356; IV, 51. In cGHD (all), age, maximum GH, insulin-like growth factor-I (IGF-I), and insulin-like growth factor-binding protein-3 (IGFBP-3) (5.1 years, 3.6 μg/L, -5.3 standard deviation score [SDS], -3.7 SDS) were lower than in IGHD (all) (6.8 years 5.8 μg/L, -2.5 SDS, -1.0 SDS), but not height (-3.1 vs. -3.2 SDS). Characteristics of NSD were similar to that of IGHD. Patients with IGHD - not cGHD - diagnosed during 2008-2015 (IV) were the youngest with most severe GHD (maxGH, IGF-I, IGFBP-3), and first year height velocity (HV) and ∆ IGF-I (10.5 cm/year, 4.0 SDS) but not ∆ height SDS were the highest on recombinant human growth hormone (rhGH) (27 μg/kg/day). Conclusions Although during 1988-2007 patient characteristics were similar, the recently (>2008) stipulated more stringent diagnostic criteria - HV before testing, sex steroid priming, lower GH cut-off - have restricted diagnoses to more severe cases as they were observed before the rhGH era.