ESPE Abstracts

Patients with Turner syndrome (TS) have short stature, despite having normal growth hormone (GH) secretion. Treatment with recombinant human GH is recommended. The effectiveness and safety of Norditropin^® (somatropin, Novo Nordisk) over ≤10 years of follow-up were investigated in two non-interventional studies: NordiNet^® IOS (NCT00960128) and the ANSWER Program (NCT01009905). Of 2,409 children with TS, 2,377 were included in the full analysis set (FAS); 1,513 in the effectiveness analysis set (EAS). In EAS, 1,273 (84%) patients were prepubertal; mean (SD) age and dose at start of GH treatment were 8.8 (3.9) years and 0.045 (0.011) mg/kg/day. Baseline insulin-like growth factor-I (IGF-I) SD score (SDS) was –0.86 (1.52). Duration of GH treatment (FAS) was 3.8 (2.8) years. Concomitant medication was prescribed to 873 (36.7%) patients (FAS), most commonly oestradiol (455; 19.1%) and levothyroxine (299; 12.6%). Height SDS (HSDS) in EAS at baseline and during follow-up, based on national and three TS-specific references, are shown in the Table. TS-specific HSDS increased continually throughout follow-up, with near-adult HSDS reached by 264 (17%) patients (mean [SD] –1.99 [0.94]; change from baseline 0.90 [0.85]). During the study, 695 (46%) patients (EAS) entered puberty at mean age of 12.7 (1.9) years. Within FAS, mean IGF-I SDS at Year 10 was 0.91 (1.69); change from baseline 1.48 (1.70). Non serious adverse reactions were reported in 33 patients (mostly headache [13 events]), serious adverse reactions in 10 patients (namely epiphysiolysis [3 events]), and GH unrelated serious adverse events in 24 patients. Despite a relatively late start, TS patients responded well to GH treatment, without new safety signals identified. Our real-world data correlate well with previous studies.