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

M Cappa,Genesis National Board On Behalf Of The Genesis Italian Investigators ,S Loche,M Maghnie,L Iughetti,A Vottero

doi:10.1007/s40618-015-0418-0

Abstract

PurposeWe 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.MethodsWe 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.ResultsThe 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.ConclusionsData from the Italian cohort of GeNeSIS showed auxological changes and safety of GH therapy consistent with results from international surveillance databases.

Highlights

Short stature in children is an important problem that should be diagnosed and managed appropriately in order to promote normal height [1]
The diagnoses associated with short stature leading to initiation of growth hormone (GH) treatment are summarized in Table 1 for 662 patients with sufficient evaluable information
The effects of GH therapy at 1–4 years of treatment and at final height were studied in a country-specific cohort of pediatric patients treated for short stature

Summary

Introduction

Short stature in children is an important problem that should be diagnosed and managed appropriately in order to promote normal height [1]. GH therapy has subsequently been approved in the USA and Europe for other pediatric conditions that result in short stature, including Turner syndrome, being born small for gestational age (SGA) with failure to attain normal growth, Prader-Willi syndrome, chronic renal. GH therapy in pediatric patients is generally considered safe, with serious adverse events reported rarely, with currently approved doses [12,13,14,15]. Certain specific conditions such as benign intracranial hypertension, scoliosis and slipped capital femoral epiphysis have been observed shortly after starting GH in small numbers of treated patients [16]. Concerns have been raised about the possibility of long-term alterations in glucose metabolism and an association with the occurrence of neoplasms, but there is little evidence for either except in patients with preexisting risk factors [13, 17,18,19,20,21,22]

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