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Multicenter Study
. 2016 Jun;39(6):667-77.
doi: 10.1007/s40618-015-0418-0. Epub 2015 Dec 28.

Efficacy and safety of growth hormone treatment in children with short stature: the Italian cohort of the GeNeSIS clinical study

M Cappa  1 , L Iughetti  2 , S Loche  3 , M Maghnie  4 , A Vottero  5 ; GeNeSIS National Board on behalf of the GeNeSIS Italian Investigators
Collaborators, Affiliations
Multicenter Study

Efficacy and safety of growth hormone treatment in children with short stature: the Italian cohort of the GeNeSIS clinical study

M Cappa et al. J Endocrinol Invest. 2016 Jun.

Abstract

Purpose: We examined auxological changes in growth hormone (GH)-treated children in Italy using data from the Italian cohort of the multinational observational Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS) of pediatric patients requiring GH treatment.

Methods: We studied 711 children (median baseline age 9.6 years). Diagnosis associated with short stature was as determined by the investigator. Height standard deviation score (SDS) was evaluated yearly until final or near-final height (n = 78). Adverse events were assessed in all GH-treated patients.

Results: The diagnosis resulting in GH treatment was GH deficiency (GHD) in 85.5 % of patients, followed by Turner syndrome (TS 6.6 %). Median starting GH dose was higher in patients with TS (0.30 mg/kg/week) than patients with GHD (0.23 mg/kg/week). Median (interquartile range) GH treatment duration was 2.6 (0.6-3.7) years. Mean (95 % confidence interval) final height SDS gain was 2.00 (1.27-2.73) for patients with organic GHD (n = 18) and 1.19 (0.97-1.40) for patients with idiopathic GHD (n = 41), but lower for patients with TS, 0.37 (-0.03 to 0.77, n = 13). Final height SDS was >-2 for 94 % of organic GHD, 88 % of idiopathic GHD and 62 % of TS patients. Mean age at GH start was lower for organic GHD patients, and treatment duration was longer than for other groups, resulting in greater mean final height gain. GH-related adverse events occurred mainly in patients diagnosed with idiopathic GHD.

Conclusions: Data from the Italian cohort of GeNeSIS showed auxological changes and safety of GH therapy consistent with results from international surveillance databases.

Keywords: Final height; Growth; Pediatric GH treatment; Safety; Short stature.

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Figures

Fig. 1
Fig. 1
Flow chart showing numbers of patients in Italy available for specific analyses
Fig. 2
Fig. 2
Change in height SDS a, gain in height SDS from baseline b, and change in height velocity c, by duration of GH treatment, for patients with idiopathic GH deficiency (GHD, n = 54), organic GHD (n = 15) or Turner syndrome (n = 9), who were GH treatment naïve at study entry and had at least 4 years of follow-up
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