Background: PATRO Children is an international, open, longitudinal, non-interventional study of the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).
Objective and hypotheses: The primary objective of PATRO Children is to assess long-term safety of Omnitrope® (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 PraderWilli syndrome). Long-term efficacy of Omnitrope® is a secondary objective.
Methods: The study population includes infants, children and adolescents treated 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 standard deviation score, height velocity and height velocity standard deviation score are calculated using height measurements and country-specific reference tables.
Results: As of January 2016, 4675 patients were recruited from 291 sites (14 countries). The mean (S.D.) treatment duration is 30.2 (22.0) months. One case of new-onset type 1 diabetes has been linked to study treatment. No clinically relevant positive anti-hGH antibody titres have been found in patients tested so far. In total, 234 patients (5.0%) have experienced treatment-related AEs and 248 (5.3%) have experienced a serious AE (SAE). SAEs were considered treatment-related in 22 (0.5%) patients. There are 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 all 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 extend the evidence base for Omnitrope®, and rhGH use in general, in the paediatric population.
10 - 12 Sep 2016
European Society for Paediatric Endocrinology