ESPE Abstracts (2018) 89 RFC15.6

ESPE2018 Rapid Free Communications Growth and syndromes (6 abstracts)

Latest Results from PATRO Children, a Multi-Centre, Non-Interventional Study of the Long-Term Safety and Efficacy of Omnitrope® in Children Requiring Growth Hormone Treatment

Shankar Kanumakala a , Roland Pfäffle b , Charlotte Höybye c , Berit Kriström d , Tadej Battelino e , Markus Zabransky f & Hichem Zouater f

aDepartment of Paediatrics, Royal Alexandra Children’s Hospital, Brighton, UK; bDepartment of Paediatric Endocrinology, University of Leipzig Medical School, Leipzig, Germany; cPatient Area Endocrinology and Nephrology, Inflammation and Infection Theme, Karolinska University Hospital, Stockholm, Sweden; dDepartment of Clinical Science/Paediatrics, Umeå University, Umeå, Sweden; eDepartment of Endocrinology, Diabetes and Metabolic Diseases, University Children’s Hospital, Ljubljana, Slovenia; fSandoz GmbH, Holzkirchen, Germany

Objectives: PATRO Children is a non-interventional, international, longitudinal study of the long-term safety of a recombinant human growth hormone (rhGH; Omnitrope®, Sandoz). In particular, the study will assess the diabetogenic potential of rhGH and the risk of malignancies. The long-term efficacy is a secondary objective of the study.

Methods: The study population includes infants, children and adolescents receiving Omnitrope® therapy according to local prescribing information. All adverse events (AEs) are monitored and recorded for evaluation of rhGH safety. Laboratory values (including glucose metabolism and anti-hGH antibodies) are requested at least once a year. Height standard deviation score (HSDS), height velocity (HV) and HVSDS are calculated using height measurements and country-specific reference tables to evaluate rhGH efficacy.

Results: As of March 2018, 6214 patients were recruited from 299 sites in 14 countries. The mean (standard deviation [S.D.]) Omnitrope® treatment duration was 36.9 (25.8) months (approx. 3 years), with 1541 (24.8%) patients completing 5 years of treatment. Overall, 84.4%% of patients were rhGH naïve and 15.1% had previously received rhGH treatment. Since September 2006, 2930 (47.2%) patients reported 11408 AEs, with 583 AEs in 415 (6.7%) patients suspected to be related to treatment. Overall, 1261 AEs in 668 (10.7%) patients were regarded as serious; of these, 52 events in 39 (0.6%) patients were suspected to be related to treatment. Drug-related serious AEs included type 1 diabetes mellitus (n=1 SGA patient, rhGH treatment discontinued), impaired glucose tolerance (n=1 SGA patient, rhGH treatment discontinued), craniopharyngioma (n=1 GHD patient, no change to rhGH treatment), germ cell cancer (n=1 GHD patient, rhGH treatment discontinued), neoplasm progression (n=1 GHD patient, rhGH treatment interrupted) and malignant astrocytoma recurrence (n=1 patient with other indication, rhGH treatment interrupted). No clinically relevant positive anti-hGH antibody titers have been found after the start of rhGH therapy in patients tested so far. Efficacy data indicate that rhGH treatment has a positive impact on growth parameters in the majority of pediatric indications. Following 3 years of treatment, the improvement in HSDS from baseline was +1.31 and +1.40 in prepubertal treatment-naïve GHD and SGA patients, respectively. At 3 years, HVSDS improved from baseline by +4.74 and +4.14 in prepubertal naïve GHD and SGA patients, respectively.

Conclusion: The latest data snapshot from the ongoing PATRO Children study suggests that rhGH is well tolerated and efficacious across pediatric indications; these findings will be confirmed by further analyses.

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