Background: PATRO Children is an international, open, longitudinal, non-interventional study designed to evaluate the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).
Objective and hypotheses: Long-term safety of Omnitrope® is the primary objective of PATRO Children (particularly the diabetogenic potential of GH in short children born small for gestational age, the risk of malignancies, and other safety signals associated with GH therapy in Prader-Willi syndrome). Long-term efficacy of Omnitrope® is a secondary objective.
Method: The study population includes infants, children and adolescents receiving treatment with Omnitrope® according to local 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 SD score (HSDS), height velocity (HV) and HVSDS are calculated using height measurements and country-specific reference tables.
Results: As of January 2015, 3928 patients have been recruited from 270 sites across 14 countries. The mean (SD) treatment duration is 27.0 (19.9) months. One case of new-onset diabetes has been reported. There have been no clinically relevant positive anti-hGH antibody titres in the patients tested so far. A total of 156 patients (4.0%) have experienced treatment-related AEs and 139 (3.5%) have experienced a serious AE (SAE). SAEs were considered treatment-related in eight (0.2%) patients. There have been no reports of GH-related malignancies and no additional safety concerns. Efficacy data indicate that Omnitrope® has a positive effect on growth parameters in prepubertal children across indications, irrespective of gender and pre-treatment status.
Conclusion: Results to date show that Omnitrope® is safe and well tolerated across paediatric indications, and is effective in the majority of children. PATRO Children will continue to extend the evidence base for Omnitrope®, and GH use in general, in the paediatric population.
Conflict of interest: ES and MZ are employees of Sandoz.
Funding: This work was supported by Sandoz International GmbH.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology