ESPE Abstracts (2019) 92 P1-227

Latest Results From PATRO Children, a Multi-Centre, Observational Study of the Long-Term Safety and Effectiveness of Omnitrope® in Children Requiring Growth Hormone Treatment

Shankar Kanumakala1, Roland Pfäffle2, Charlotte Höybye3, Berit Kriström4, Tadej Battelino5, Markus Zabransky6, Hichem Zouater6


1Department of Paediatrics, Royal Alexandra Children's Hospital, Brighton, United Kingdom. 2Department of Paediatric Endocrinology, University of Leipzig Medical School, Leipzig, Germany. 3Patient Area Endocrinology and Nephrology, Inflammation and Infection Theme, Karolinska University Hospital, Stockholm, Sweden. 4Department of Clinical Science/Paediatrics, Umeå University, Umeå, Sweden. 5Department of Endocrinology, Diabetes and Metabolic Diseases, University Children's Hospital, Ljubljana, Slovenia. 6Sandoz GmbH, Holzkirchen, Germany


Objectives: PATRO Children is an observational, international, longitudinal study of the long-term safety of a recombinant human growth hormone (rhGH; Omnitrope®, Sandoz). In particular, the study will assess the impact of rhGH on glucose metabolism and risk of malignancies. Long-term effectiveness is a secondary objective.

Methods: The study population includes infants, children and adolescents receiving Omnitrope® therapy according to country-specific 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 according to country-specific reference tables.

Results: Over 13 years up to January 2019, data were included from 6710 patients at 301 sites in 14 countries. The mean (SD) Omnitrope® treatment duration was 39.3 (27.0) months (approx. 3.3 years), with 1844 (27.5%) patients completing 5 years of treatment. Overall, 85.1% of patients were rhGH naïve and 14.5% had previously received rhGH treatment (0.4% data missing). Since the study start in September 2006, 3336 (49.7%) patients have reported 13588 AEs, with 674 AEs in 480 (7.2%) patients suspected to be treatment-related. Overall, 1553 AEs in 807 (12.0%) patients were regarded as serious; of these, 81 events in 53 (0.8%) patients were suspected to be treatment-related. Drug-related serious AEs included type 1 diabetes mellitus (n=1 SGA patient, treatment discontinued), impaired glucose tolerance (n=2 SGA patients, treatment discontinued; n=1 GHD patient, dose reduction), malignant germ cell cancer (n=1 GHD patient, treatment discontinued), craniopharyngioma (n=1 GHD patient, no treatment interruption), and progression of a pre-existing neoplasm (n=1 GHD patient, n=1 patient with other indication; treatment interrupted in both). A positive anti-hGH antibody titer was reported post-baseline in one treatment-naïve patient (1.3% of patients tested post-baseline). Effectiveness data: following 4 years of treatment, the delta HSDS was +1.55 and +1.57 in prepubertal treatment-naïve GHD and SGA patients, respectively. At 4 years, delta peak-centered HVSDS was +4.42 and +3.68 in prepubertal treatment-naïve GHD and SGA patients, respectively.

Conclusion: The latest results from the ongoing PATRO Children study suggest that Omnitrope® is well tolerated and effective across pediatric indications; these findings will be reviewed by further analyses in the future.

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