ESPE Abstracts (2019) 92 P1-87

Long-Term Safety Follow-up after Omnitrope® (recombinant human growth hormone) Treatment in Short Children Born Small for Gestational Age (SGA): Latest Results

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: The benefit of recombinant human growth hormone (rhGH) in improving height is widely recognised; however, rhGH therapy can affect carbohydrate metabolism and lead to impaired glucose tolerance during treatment. In addition, short children born SGA are predisposed to metabolic abnormalities. This study assessed the long-term safety of growth hormone (Omnitrope®) use in short children born SGA for up to 10 years after the end of treatment.

Methods: Conducted between June 2009 and October 2018, this was a follow-up observational study of patients from a phase IV study. All patients who participated in the phase IV study and received at least one dose of study medication were invited to enter a safety follow-up period of up to 10 years. The baseline visit was the final visit of the phase IV study. Further visits were planned after 6 months (F1), 1 year (F2), 5 years (F3) and 10 years (F4). The primary objective was to evaluate the long-term effect of rhGH treatment on the development of diabetes mellitus; secondary objectives included incidence/severity of adverse events (AEs).

Results: In total, 130 subjects were enrolled in the follow-up study; 99 completed F1, 88 completed F2 and 13 completed F3 (no subject reached F4). The full analysis set for evaluation comprised 118 patients (64 female). Mean (SD) age at baseline was 14.79 (2.85) years, mean weight was 44.97 (13.34) kg and mean (SD) BMI SDS was –0.84 (1.37). Mean (SD) duration of follow-up was 39.6 (24.4) months. No subject was newly diagnosed with diabetes. The results for carbohydrate metabolism parameters were consistent with this finding. A total of 144 AEs were reported in 54 subjects; these were mostly of mild-to-moderate intensity (96.5%) and not suspected to be related to previous rhGH treatment (94.4%). Serious AEs (n=18) have been reported in 8 patients; 3 (in 1 patient) were suspected as possibly related to previous rhGH treatment (anemia, menorrhagia, oligomenorrhoea). One fatal event occurred (sepsis), which was judged as not related to previous rhGH treatment.

Conclusions: None of the participating subjects, who had all been previously treated with Omnitrope® in a phase IV study, developed diabetes during this follow-up study. In addition, no other unexpected or concerning safety signals were observed.

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