ESPE Abstracts (2014) 82 P-D-2-3-442

Two-Year Results from Patro Children, a Multi-Centre, Non-Interventional Study of the Long-Term Efficacy and Safety of Omnitrope® in Children Requiring GH Treatment

Roland Pfafflea, Shankar Kanumakalab, Charlotte Höybyec, Berit Kriströmd, Ellen Schucke, Markus Zabranskye, Tadej Battelinof & Michel Colleg

aDepartment of Paediatric Endocrinology, University of Leipzig Medical School, Leipzig, Germany; bDepartment of Paediatrics, Royal Alexandra Children’s Hospital, Brighton, UK; cDepartment of Endocrinology, Metabolism and Diabetology, Karolinska University Hospital, Stockholm, Sweden; dDepartment of Clinical Science/Paediatrics, Umeå University, Umeå, Sweden; eSandoz International GmbH, Holzkirchen, Germany; fDepartment of Endocrinology, Diabetes and Metabolic Diseases, University Children’s Hospital, Llubljana, Slovenia; g25 Rue Boudet, Bordeaux, France

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.