Objectives: PATRO Children is an observational, international, longitudinal study of the long-term safety of a recombinant human growth hormone (rhGH; Omnitrope®, Sandoz). The effect of rhGH treatment on growth and adult height (AH) is assessed as a secondary objective of the study.
Methods: This ongoing study is conducted in hospitals and specialised endocrinology clinics across 14 countries. The study population includes infants, children and adolescents receiving Omnitrope® therapy according to country-specific prescribing information. Height velocity (HV, cm/year), height standard deviation score (HSDS), and HVSDS are derived from height measurements and country-specific reference tables. It is anticipated that patients will be treated with Omnitrope® for growth promotion in the study until they achieve AH. Patients are considered to have reached AH if they discontinue the study due to reaching AH/bone age maturity, or reaching near AH.
Results: As of January 2019 (over 13 years), 5777 patients were enrolled and included in the effectiveness population. Overall, 85.0% of patients were rhGH naïve at study entry and 14.9% had previously received other rhGH treatment. To date, 1209 (20.9%) patients (male, n=626; female, n=583) have reached AH, of which 925 (76.5%) patients were rhGH naïve and 283 (23.4%) were not. Of the patients who reached AH, 772 (63.9%) had growth hormone deficiency (GHD), and 309 (25.6%) were born small for gestational age (SGA). Among GHD patients who reached AH, 479 (62.0%) were male and 293 (38.0%) were female; mean (SD) baseline HSDS was 2.12 (1.02); at AH, mean (SD) HSDS was 1.28 (1.09). The mean (SD) difference in AH and target height was 4.55 (7.20) cm, and the mean (SD) difference between AH SDS and target height SDS was 0.87 (1.05). In SGA patients who reached AH, 123 (39.8%) were male and 186 (60.2%) were female. Mean (SD) baseline HSDS was 2.26 (1.11); at AH, mean (SD) HSDS was 2.07 (1.20). The mean (SD) difference in AH and target height was 6.82 (6.97) cm, and the mean (SD) difference between AH SDS and target height SDS was 1.40 (1.08).
Conclusion: Based on this analysis, Omnitrope® treatment improves AH of children with GHD and SGA children in real-life clinical practice. The PATRO Children study is ongoing and will continue to provide further long-term information on AH in children receiving Omnitrope® in these and other approved indications.
19 Sep 2019 - 21 Sep 2019