ESPE Abstracts (2019) 92 P1-387

Omnitrope® (Recombinant Human Growth Hormone) in Short Children Born Small for Gestational Age (SGA): A Long-Term, Phase IV Study

Mieczyslaw Walczak1, Tomasz Giemza2, Shrihari Jathanakodi3, Hichem Zouater4, Markus Zabransky4


1Department of Paediatric Endocrinology and Diabetology, Pomeranian Medical University, Szczecin, Poland. 2Sandoz Polska, Warsaw, Poland. 3Hexal AG, Holzkirchen, Germany. 4Sandoz GmbH, Holzkirchen, Germany


Background: Short children born SGA are predisposed to metabolic abnormalities. While the benefit of recombinant human growth hormone in improving height is widely recognised, it can affect carbohydrate metabolism and lead to impaired glucose tolerance during treatment. This ongoing, prospective study is assessing the long-term safety and efficacy of Omnitrope® (somatropin) in children born SGA. Here we present data from an interim analysis conducted in April 2017.

Methods: Prepubertal children born SGA were recruited according to standard criteria; those included are treated with Omnitrope® and followed at predetermined time intervals until final height is reached. Non-responders to treatment (height velocity standard deviation score [HVSDS] < +1) were withdrawn from the study after one year.

Results: Overall, 278 children were enrolled in the study; 13 discontinued after one year due to non-response to treatment; 249 completed their 2-year assessment; and 132 remained in the study at the interim analysis timepoint (median treatment duration 72.7 months). Mean (SD) age at baseline and latest visit was 7.86 (2.74) and 13.08 (3.36) years, respectively. To date, there have been no confirmed cases of new-onset diabetes mellitus. Oral glucose tolerance (2h), HbA1c, and fasting glucose levels have remained stable from baseline to latest visit; however, mean (SD) fasting insulin levels increased from 35.65 (34.69) pmol/L at baseline to 53.68 (35.93) pmol/L after 1 year and to 70.46 (45.60) pmol/L at latest visit. Auxological measurements demonstrated improvements in height parameters from baseline to latest visit, including mean height SDS (–3.39 at baseline, –2.57 at Year 1, –2.15 at year 2 and –1.74 at latest visit) and peak-centred HVSDS (–2.11 at baseline, +4.16 at Year 1, +2.23 at Year 2, and +0.19 at latest visit). Similarly, mean (SD) IGF-I SDS increased from –1.08 (1.02) at baseline to +0.64 (1.21) after 1 year and +0.53 (1.27) at latest visit. In total, 2075 adverse events (AEs) have been reported in 242 patients. The vast majority of these AEs were of mild-to-moderate intensity (99%) and not suspected to be related to study drug (96%). Serious AEs (n=125) have been reported in 68 patients; 4 (in 3 patients) were suspected as possibly related to study treatment, and one (osteonecrosis) resulted in study drug being permanently discontinued.

Conclusions: This long-term study confirms the effectiveness of Omnitrope® treatment for improving height in short children born SGA. In addition, there have been no concerning or clinically relevant safety findings to date.

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