ESPE Abstracts

Background: The iNCGS Registry monitored long-term safety and effectiveness of NutropinAq^® (Somatropin injection) for paediatric growth disorders.

Objective: To report year-1 effectiveness and safety data from the iNCGS registry (NCT00455728).

Methods: Open-label, non-interventional, post-marketing surveillance study, in seven European countries from October 2005–December 2016. Measurements included height Standard Deviation Score (SDS) and height velocity. Investigators reported non-serious related treatment-emergent adverse events (TEAEs) and serious AEs, regardless of relationship to study treatment.

Results: Of 3657 screened patients, 2792 were enrolled. Mean (SD) age was 7.7 (4.1) years at diagnosis and 9.5 (3.6) years at first NutropinAq^® treatment. 85.3% patients were treatment-naïve, 67.9% naïve pre-pubertal [NPP], 14.6% non-naïve (status missing, n=4). GHD was present in 2082 patients (74.6%) and idiopathic in 1825. Non-GHD diagnoses included: Turner Syndrome (n=199); idiopathic short stature (n=90); small for gestational age (n=254). The starting dose of NutropinAq^® varied by indication and followed the label recommendations. Median duration of exposure was 38.2 months. At baseline, the Registry population (n=2714) had mean (SD) height SDS of -2.4 (1.0) and height velocity of 5.2 (2.4) cm/year. After 1 year of treatment, mean [95% CI] change from baseline in height SDS was: overall, 0.60 [0.58;0.62] (n=2125); treatment-naïve, 0.64 [0.61;0.66] (n=1839); NPP, 0.68 [0.65;0.70] (n=1465); NPP with organic GHD, 0.78 [0.67;0.88] (n=113). Height velocity after 1 year was: overall, 8.5 cm/year [8.4;8.6] (n=2131); treatment-naïve, 8.8 cm/year [8.7;8.9] (n=1843); NPP, 8.8 cm/year [8.7;8.9] (n=1467); NPP with organic GHD, 9.4 [8.9;10.0] (n=114). Improvements in effectiveness parameters occurred in all subgroups by disease aetiology, and were greatest in organic GHD. In the Safety Population (n=3493), 610 patients (17.5%) had ≥1 non-serious related TEAE, most frequently abnormal investigations (304 [8.7%]), including increased insulin-like growth factor (256 [7.3%]). 206 patients (5.9%) experienced ≥1 serious TEAE, considered related to NutropinAq^® in 27 (0.8%). 20 patients (0.6%) experienced a neoplasm considered as a serious event (4 [0.1%] considered treatment-related) but 14 had a prior history of neoplasm. Seven deaths occurred, all considered not related to NutropinAq^®.

Conclusions: After 1 year of treatment with NutropinAq^®, there was an improvement in mean height SDS and height velocity in all subgroups by prior treatment status and aetiologies, despite a >1-year delay between diagnosis and start of treatment. These data confirm that the benefit-risk profile for NutropinAq^® remains favourable, with no new safety concerns in children with growth failure treated within the recommended indication.