ESPE Abstracts (2022) 95 P1-513

ESPE2022 Poster Category 1 Growth and Syndromes (85 abstracts)

Long-term effectiveness and safety of childhood growth hormone treatment in Turner syndrome from two large observational studies

Philippe Backeljauw 1 , Alberto Pietropoli 2 & Tilman Rohrer 3


1Cincinnati Center for Growth Disorders and Center for Pediatric and Adult Turner Syndrome Care, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, USA; 2Rare Endocrine Disorders, Novo Nordisk Health Care AG, Zurich, Switzerland; 3Department of Pediatrics and Neonatology, Division of Pediatric Endocrinology, University Children’s Hospital, Saarland University Medical Center, Homburg, Germany


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.

Table: Mean (SD) HSDS
    Follow-up (years)
Reference data Baseline 1 2 3 4 5 6 7 8 9 10
National –2.62 (0.95) –2.09 (0.95) –1.80 (0.96) –1.68 (0.94) –1.65 (0.94) –1.57 (0.88) –1.53 (0.87) –1.60 (0.88) –1.70 (0.87) –1.88 (0.85) –2.00 (0.86)
Westerlaken, 1997 0.08 (1.16) 0.87 (1.09) 1.34 (1.13) 1.62 (1.12) 1.77 (1.14) 1.93 (1.12) 2.10 (1.12) 2.15 (1.15) 2.20 (1.08) 2.14 (0.95) 2.01 (0.91)
Cabrol, 1996 0.23 (1.15) 1.02 (1.09) 1.48 (1.14) 1.74 (1.12) 1.86 (1.13) 2.00 (1.11) 2.11 (1.11) 2.15 (1.08) 2.20 (1.02) 2.17 (0.89) 2.05 (0.85)
Ranke, 1988 0.55 (1.10) 1.28 (1.07) 1.73 (1.11) 1.99 (1.11) 2.12 (1.14) 2.28 (1.11) 2.44 (1.13) 2.48 (1.16) 2.49 (1.09) 2.40 (0.98) 2.24 (0.95)
Patients 1513
1475*
1142
1140*
965 772 597 460 326 238 165 97 52
*Ranke only

Volume 95

60th Annual ESPE (ESPE 2022)

Rome, Italy
15 Sep 2022 - 17 Sep 2022

European Society for Paediatric Endocrinology 

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