ESPE Abstracts

Background: NordiNet^® International Outcome Study ([IOS]; NCT00960128), a non-interventional study (2006–2016), assessed the effectiveness and safety of real-world treatment with Norditropin^®. Outcomes were assessed in children with growth hormone deficiency (GHD), born small for gestational age (SGA), Turner syndrome (TS), chronic renal disease (CRD), idiopathic short stature (ISS), Noonan syndrome (NS) and Prader-Willi syndrome (PWS).

Methods: Patient information was entered using a web-based system. 17,995 paediatric patients enrolled: 17,711 included in the full analysis set (FAS) (safety evaluation); 11,967 in the effectiveness analysis set (EAS). Endpoints included change from baseline in height standard deviation scores (ΔHSDS) and near-adult height (NAH) SDS (height at age >16 [boys]/>15 [girls] and height velocity <2 cm/year, or height at >18 years). Non-serious adverse reactions (NSARs), SARs and serious adverse events (SAEs) were recorded. Data are mean (standard deviation).

Results: Patient numbers by indication were FAS/EAS: GHD, 9967/7141; SGA, 4274/3200; TS, 1374/936; CRD, 290/200; ISS, 485/317; NS, 154/106; PWS, 132/67. At treatment start, patients with PWS (4.7 [5.00] years) were the youngest: GHD, 9.1 (4.1); SGA, 7.9 (3.4); TS, 8.7 (3.8); CRD, 8.3 (4.4); ISS, 10.1 (3.5); NS, 8.9 (3.8). Patients born SGA were shortest (HSDS) at baseline (−2.97 [0.91]): GHD, −2.55 (1.10); TS, −2.66 (0.93); CRD, −2.74 (1.17); ISS, −2.82 (0.99); NS, −2.83 (1.13); PWS, −1.94 (1.48). Average GH dose (mg/kg/day) was lower for PWS (0.026 [0.008]) versus GHD (0.032 [0.008]); SGA, 0.038 (0.009); TS, 0.044 (0.009); CRD, 0.041 (0.011); ISS, 0.038 (0.014); NS, 0.040 (0.009). Treatment follow-up (years) was longest for patients with TS (4.3 [2.8]): GHD, 3.8 (2.9); SGA, 3.6 (2.8); CRD, 2.8 (2.6); ISS, 3.3 (2.4); NS, 3.4 (2.9); PWS, 4.0 (3.5). ΔHSDS was greatest in year 1: GHD, 0.69 (0.56); SGA, 0.65 (0.44); TS, 0.54 (0.36); CRD, 0.61 (1.19); ISS, 0.52 (0.38); NS, 0.51 (0.38); PWS, 0.85 (0.90). Proportion (%) of patients with HSDS >−2 was (baseline/year 3): GHD, 26.2/79.3; SGA, 9.3/64.4; TS, 22.1/63.5; CRD, 23.5/59.4; ISS, 18.3/56.8; NS, 17.9/67.5; PWS, 59.7/89.3. NAH SDS was: GHD, −1.16 (1.22) (n=943); SGA, −1.97 (0.95) (n=190); TS, −2.08 (0.84) (n=189); small numbers of patients achieved NAH during the observation period in other indications. Safety: no new signals were observed. Number of events/number of patients were: NSARs, 288/249; SARs, 133/90; SAEs, 352/224.

Conclusions: In paediatric patients, growth hormone was associated with increased HSDS and increased proportion with HSDS >−2. No new safety signals were revealed.