Endocrine Abstracts

Background: The NordiNet^® International Outcome Study (IOS) (NCT00960128), a non-interventional study (2006–2016), assessed the effectiveness and safety of real-life treatment with Norditropin^®. Out of 20,548 enrolled patients, 20,195 (paediatric/adult; 17,711/2484) were included in the full analysis set (FAS) and 12,938 (11,967/971) in the effectiveness analysis set (EAS). Outcomes were assessed in children with growth hormone deficiency (GHD), born small for gestational age (SGA), Turner syndrome (TS), and Noonan syndrome (NS), and in adults with GHD (AGHD).

Methods: Patient information was entered by participating physicians using a web-based system. Among other endpoints, change from baseline in height standard deviation scores (ΔHSDS) and insulin-like growth factor-I (ΔIGF-I) SDS were assessed. Non-serious (NS) adverse reactions (NSARs), SARs, and serious adverse events (SAEs) were recorded. Data are mean (SD).

Results: Patient numbers by indication were (FAS/EAS): GHD, 9967/7141; SGA, 4274/3200; TS, 1374/936; NS, 154/106; AGHD, 2321/971; ‘other’, 2105/584. At treatment start, patients born SGA (7.9 (3.4) years) were the youngest (GHD, 9.1 (4.1)), TS, 8.7 (3.8), NS, 8.9 (3.8). Average GH dose (mg/kg per day) was lower for GHD (0.032 (0.008)) versus SGA (0.038 (0.009)), TS (0.044 (0.009)) or NS (0.040 (0.009)). Treatment follow-up (years) was longer for patients with TS (4.3 (2.8)) versus GHD (3.8 (2.9)), born SGA (3.6 (2.8)) or NS (3.4 (2.9)). Patients born SGA were shortest (height S.D.S.) at baseline (−2.97 (0.91)) (GHD, −2.55 (1.10), TS, −2.66 (0.93); NS, −2.83 (1.13)). ΔHSDS was greatest in year 1 (baseline to year 1 visit): GHD, 0.69 (0.56); SGA, 0.65 (0.44); TS, 0.54 (0.36); NS 0.51 (0.38). ΔHSDS (baseline to near adult height): GHD, 1.42 (1.19); SGA, 1.11 (0.81); TS, 0.83 (0.87); NS, 1.43 (0.59).

AGHD: age at treatment start (years), females, 46.6 (14.0), males, 49.3 (14.6); mean GH dose (mg/day), females, 0.338 (0.177), males, 0.289 (0.157); treatment follow-up (years), females, 4.9 (4.2), males, 5.0 (4.2) years; baseline IGF-I SDS, females, −1.09 (1.44), males, −1.12 (1.60). IGF-I SDS increased year-on-year from baseline (ΔIGF-I S.D.S. from baseline at year 1: female, 1.20 (1.51), male, 1.52 (1.47)). Safety: no new safety signals were observed. Number of events/number of patients were: paediatric, NSARs, 288/249; SARs, 133/90; SAEs, 352/224; adults, NSARs, 69/54; SARs, 38/29; SAEs, 200/119.

Conclusions: NordiNet IOS data showed that Norditropin was associated with increased HSDS in paediatric patients and increased IGF-I SDS in patients with AGHD supporting the effectiveness of GH therapy. No new safety signals were revealed.