Background: Response to rhGH treatment is variable among GH deficiency (GHD), GH insensitivity and several intermediate conditions.
Aims and objectives: To compare baseline parameters and response to rhGH treatment in eight diagnostic categories of patients with short stature.
Methods: We selected 125 prepubertal children presenting at least 2 years of rhGH treatment (mean 5.29 years, range 215.6 years), hormonal and clinic parameters alterations (0<IGF1 <−2 SDS and/or GH stimulated peak secretion <8 μg/l and H<−3 SDS or HV <−2 SDS or H<−2 SDS+HV<−1.5 SDS). We divided patients in eight groups: 19 organic-GHD (GHD-O), 19 isolated idiopathic-GHD (GHD-II), 16 neuroscretory dysfunction (NSD), 14 radio-treated-GHD patients (rt-GHD), 15 ISS, 11 SGA, 11 SHOX-Deficiency, 20 Turner syndrome (TS). Changes in height and in height velocity SDS at 1 year and at the last visit were evaluated in relation to years of treatment and average doses of rhGH.
Results: At the 1st year all groups gained >1 height SDS. At the last visit, GHD-O gain 1.78±1.58 SDS, SGA 1.07±0.4 SDS, NSD 0.90±0.65 SDS, ISS 0.84±0.66 SDS, GHD-II 0.72±0.55 SDS, SHOX-D 0.72±0.49 SDS, TS 0.23±0.61 SDS, rt-GHD 0.05±1.84 SDS. Statistical significant differences in height gain SDS at the last visit were found between: GHD-O vs rt-GHD (P 0.0014); GHD-O vs GHD-II (P 0.0246); rt-GHD vs GHD-II (P 0.0154); rt-GHD vs NSD (P 0.0112); SHOX-D vs TS (P 0.0318), while was not found between GHD-II vs BSI (P 0.3228).
Conclusions: rhGH treatment improves the short-term response in all groups and the long-term response in most patients: GHDO has the better response, radio-treated patients have the lowest, GHDII and ISS intermediate comparable response. Our data indicate that it is better review periodically the selection criteria and response to treatment.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology