ESPE Abstracts (2019) 92 P2-171

Final Height in GH-deficient Paediatric Patients: A Nationwide Experience

Stefano Zucchini1, Antonella Lonero2, Simonetta Bellone3, Mauro Bozzola4, Alessandra Cassio1, Maria Felicia Faienza5, Claudio Giacomozzi6, Anna Grandone7, Chiara Guzzetti8, Lorenzo Iughetti9, Maria Parpagnoli10, Mariacarolina Salerno11, Maria Elizabeth Street12, Gianluca Tornese13, Malgorzata Wasniewska14, Maurizio Delvecchio15

1Department of Medical and Surgical Sciences, University of Bologna, Bologna, Italy. 2Pediatrics Unit "Perrino" Hospital, Brindisi, Italy. 3Department of Pediatric Endocrinology,AOU "Maggiore della Carità", Novara, Italy. 4Pediatrics Unit "S Matteo" Hospital, Pavia, Italy. 5Pediatrics Department "B Trambusti" Giovanni XXIII Pediatric Hospital, Bari, Italy. 6Endocrinology & Diabetology Pediatric ASST Mantova, Hospital "Carlo Poma", Mantova, Italy. 7AOU Campania University "L. Vanvitelli", Napoli, Italy. 8Pediatric Hospital for Microcytaemia "A Cao" Cagliari, A.O. Brotzu, Cagliari, Italy. 9Pediatric Unit Department of Medical and Surgical Sciences for mothers, children and adults, Modena, Italy. 10Endocrinology Department "AUO Anna Meyer", Firenze, Italy. 11Department of Traslational Medical Sciences, "Federico II" University, Napoli, Italy. 12AUSL RE-IRCCS, Reggio Emilia, Reggio Emilia, Italy. 13Pediatrics Department - Endocrinology, Auxology and Diabetology Unit "Burlo Garofalo", Trieste, Italy. 14Department of Human Pathology of adult and Evolutive age "G. Barresi", Messina, Italy. 15Pediatrics Unit, "Giovanni XXIII Pediatric Hospital", Bari, Italy

Background: The primary goal of GH treatment in GHD children and adolescents is to normalize height, in order to attain an adult height within the target height (TH) range.

Aim: to investigate height improvement in GHD paediatric patients treated with GH in our Country.

Subjects: 737 patients with isolated GHD (39.5% females) from 13 tertiary Centres for Paediatric Endocrine Care of our Country diagnosed between 1991 and 2013. Near adult height (NAH) was recorded at GH treatment stop (growth velocity –GV- < 2 cm/year).

Inclusion criteria: in keeping with national laws, GHD was defined as 1. height < -3 SDS OR height < -2 SDS and GV < -1 SDS OR height < -1.5 SDS than TH and GV < -2 SDS AND 2. serum GH below 10 ng/ml after two standard stimulation tests (20 ng/ml if GHRG + arginine test).

Exclusion criteria: any condition which could affect linear growth.

Results: TH was -0.9 SDS. At start of treatment (baseline): males were older than females (10.6 vs 9.6 years, P <0.001); height was –2.2 SDS (vs TH P <0.001); mean GH dose 0.22 mg/kg/week. At puberty onset: age 11.3 and 12.5 years in females and males; height –1.3 SDS (vs baseline P <0.001; vs TH P <0.001). At the end of treatment: age 14.8 and 16.3 years in females and males; height -0.8 SDS (vs baseline and puberty onset P <0.001; vs TH P = ns); mean GH dose 0.18 mg/kg/week (vs baseline P <0.001). NAH was significantly and positively correlated with TH, baseline height and height at puberty onset (P <0.001 for each), but not with baseline age and GH dose. The regression analysis showed that baseline height and TH were the most important factors affecting NAH. Discussion: in our real-life nationwide study, the patients seem to be older than data from literature, but NAH is within the genetic growth potential. Most of them could have a transient prepubertal GHD (priming with sexual steroids was never performed), but a delay in referral to the tertiary Centres for Paediatric Endocrine Disease could also account for this finding. The baseline GH dose seems similar to what reported in literature and decreased during the follow-up. Patients recruitment and data collection about GH retesting, IGF1, and MRI findings are still ongoing.