Background: PATRO children is an international, open, longitudinal, noninterventional study of the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).
Objective and hypotheses: The primary objective is to assess the long-term safety of Omnitrope®, particularly the diabetogenic potential of GH in short children born small for gestational age, the risk of malignancies, and potential risks of GH therapy in Prader-Willi syndrome. The long-term efficacy of Omnitrope® is a secondary objective.
Method: PATRO children includes infants, children, and adolescents who are receiving treatment with Omnitrope® according to country-specific prescribing information. To evaluate safety, all adverse events (AEs) are monitored and recorded. Laboratory values (including glucose metabolism and anti-hGH antibodies) are requested at least once a year. To evaluate efficacy, height SDS (HSDS), height velocity (HV), and HVSDS are derived from height measurements and country-specific reference tables.
Results: To date, 2816 patients have been recruited from 250 sites across 14 countries. The mean (S.D.) treatment duration is 22.5 (18.6) months. There has been one case of new-onset diabetes. No patients have tested positive for anti-hGH antibodies so far (83 tests in 43 patients). In total, 119 patients (4.3%) have experienced treatment-related adverse events (AEs) and 77 (2.8%) have experienced a serious AE (SAE). SAEs were treatment-related in 4 (0.1%) patients. There have been no reports of GH-related malignancies and no additional safety concerns. Efficacy data at 2 years indicate a positive effect of Omnitrope® on growth parameters in prepubertal children across all indications, irrespective of gender and pre-treatment status.
Conclusion: This 2-year analysis shows that Omnitrope® was safe and well tolerated in a wide range of paediatric indications. Omnitrope® was effective in the majority of children. This ongoing study will extend the evidence base for Omnitrope®, and GH in general, in paediatric indications.
20 - 22 Sep 2014
European Society for Paediatric Endocrinology