| Contributors: |
Cappa, M.; Iughetti, L.; Loche, S.; Maghnie, M.; Vottero, A; Genesis National Board On Behalf Of The Genesis Italian Investigators; Antoniazzi, Franco; Beccaria, Luciano; Bernasconi, Sergio; Caggiano, Domenico; Caruso-Nicoletti, Manuela; Catucci, Alessandra; Chiarelli, Francesco; Cianfarani, Stefano; Colucci, Annarita; Francesca De Rienzo; Raffaele Di Pumpo; Alessandra Di Stasio; Farello, Giovanni; Felici, Leonardo; Femiano, Pasquale; Garagantini, Luigi; Giavoli, Claudia; Greggio, Nellaaugusta; Guazzarotti, Laura; Larizza, Daniela; Licenziati, Mariarosaria; Lonero, Antonella; Maggio, Mariacristina; Marsciani, Alberto; Matarazzo, Patrizia; Mazzanti, Laura; Messini, Beatrice; Napoli, Flavia; Pasquino, Annamaria; Perrone, Laura; Pilia, Sabrina; Pilotta, Alba; Piran, Marzia; Pozzobon, Gabriella; Predieri, Barbara; Sacco, Michele; Salerno, Mariacarolina; Tirendi, Antonina; Ubertini, Graziamaria; Vannelli, Silvia; Wasniewska, Malgorzata; Zampolli, Maria; Zanotti, Martina; Zuccotti, Gianvincenzo |
| Description: |
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 9.6years). 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 (0.30mg/kg/week) than patients with GHD (0.23mg/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. |