ESPE Abstracts

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.