Roland Pfäffle1, Martin Bidlingmaier2, Ilonka Kreitschmann-Andermahr3, Christof Land4, Carl Joachim Partsch5, Karl Otfried Schwab6, Heide Sommer7, Philippe Backeljauw8, Shankar Kanumakala9, Sandro Loche10, Hichem Zouater11, Christian J Strasburger12. 1. Department of Pediatrics, Leipzig University, Leipzig, Germany, rpfaeffle@medizin.uni-leipzig.de. 2. Medizinische Klinik und Poliklinik IV, Klinikum der Universität München, Munich, Germany. 3. Department of Neurosurgery, University Medicine Essen, Essen, Germany. 4. Praxis für Kinder-Endokrinologie und Diabetologie, Gauting, Germany. 5. Department of Paediatric Endocrinology, Endokrinologikum Hamburg, Hamburg, Germany. 6. Department of Pediatrics, University Hospital Freiburg, Freiburg, Germany. 7. Sandoz Germany c/o HEXAL AG, Holzkirchen, Germany. 8. Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA. 9. Royal Alexandra Children's Hospital, Brighton and Sussex University Hospitals NHS Trust, Brighton, United Kingdom. 10. SSD Endocrinologia Pediatrica e Centro Screening Neonatali Ospedale Pediatrico Microcitemico "A. Cao," AO Brotzu, Cagliari, Italy. 11. Sandoz Biopharmaceutical c/o HEXAL AG, Holzkirchen, Germany. 12. Department of Medicine for Endocrinology, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Abstract
INTRODUCTION: Omnitrope® was approved as a biosimilar recombinant human growth hormone (rhGH) in 2006. OBJECTIVE: The purpose of this work was to evaluate the long-term safety and effectiveness of Omnitrope® in PATRO Children - an ongoing, international, longitudinal, non-interventional study in children who require rhGH treatment. METHODS: The study population includes infants, children, and adolescents receiving Omnitrope®. Adverse events (AEs) are monitored for safety and rhGH effectiveness is evaluated by calculation of the height standard deviation score (HSDS), height velocity (HV), and HVSDS using height measurements and country-specific references. RESULTS: As of November 2017, 6,009 patients from 298 centers across 14 countries were enrolled in PATRO Children. Overall, 57.7% of patients had growth hormone deficiency (GHD), 25.8% were born small for gestational age (SGA), and 4.8% had Turner syndrome (TS). In total, 84.1% were rhGH treatment naïve at study entry. The mean duration of Omnitrope® treatment in the study was 36.1 months (range 0-133.7). Overall, 10,360 AEs were reported in 2,750 patients (45.8%). Treatment-related AEs were reported in 396 patients (6.6%; 550 events), and serious AEs (SAE) in 636 patients (10.6%; 1,191 events); 50 SAEs in 37 patients (0.6%) were considered treatment related. Following 5 years of therapy in patients who were rhGH treatment naïve at study entry, improvement from baseline in mean HSDS was +1.85 in GHD, +1.76 in SGA, and +1.0 in TS patients. In total, 912 (17.9%) patients reached adult height (n = 577 GHD, n = 236 SGA, n = 62 TS). CONCLUSIONS: This analysis of PATRO Children indicates that biosimilar rhGH is well tolerated and effective in real-world clinical practice.
INTRODUCTION:Omnitrope® was approved as a biosimilar recombinant humangrowth hormone (rhGH) in 2006. OBJECTIVE: The purpose of this work was to evaluate the long-term safety and effectiveness of Omnitrope® in PATRO Children - an ongoing, international, longitudinal, non-interventional study in children who require rhGH treatment. METHODS: The study population includes infants, children, and adolescents receiving Omnitrope®. Adverse events (AEs) are monitored for safety and rhGH effectiveness is evaluated by calculation of the height standard deviation score (HSDS), height velocity (HV), and HVSDS using height measurements and country-specific references. RESULTS: As of November 2017, 6,009 patients from 298 centers across 14 countries were enrolled in PATRO Children. Overall, 57.7% of patients had growth hormone deficiency (GHD), 25.8% were born small for gestational age (SGA), and 4.8% had Turner syndrome (TS). In total, 84.1% were rhGH treatment naïve at study entry. The mean duration of Omnitrope® treatment in the study was 36.1 months (range 0-133.7). Overall, 10,360 AEs were reported in 2,750 patients (45.8%). Treatment-related AEs were reported in 396 patients (6.6%; 550 events), and serious AEs (SAE) in 636 patients (10.6%; 1,191 events); 50 SAEs in 37 patients (0.6%) were considered treatment related. Following 5 years of therapy in patients who were rhGH treatment naïve at study entry, improvement from baseline in mean HSDS was +1.85 in GHD, +1.76 in SGA, and +1.0 in TS patients. In total, 912 (17.9%) patients reached adult height (n = 577 GHD, n = 236 SGA, n = 62 TS). CONCLUSIONS: This analysis of PATRO Children indicates that biosimilar rhGH is well tolerated and effective in real-world clinical practice.
Authors: Pekka Kurki; Hye-Na Kang; Niklas Ekman; Ivana Knezevic; Martina Weise; Elena Wolff-Holz Journal: BioDrugs Date: 2022-05-21 Impact factor: 7.744
Authors: Juan P López-Siguero; Maria J Martínez-Aedo; Jose Antonio Bermúdez de la Vega; Jordi Bosch-Muñoz; Alfonso M Lechuga-Sancho; Triana Villalobos Journal: Clin Endocrinol (Oxf) Date: 2021-12-09 Impact factor: 3.523
Authors: Lars Sävendahl; Michel Polak; Philippe Backeljauw; Joanne C Blair; Bradley S Miller; Tilman R Rohrer; Anita Hokken-Koelega; Alberto Pietropoli; Nicky Kelepouris; Judith Ross Journal: J Clin Endocrinol Metab Date: 2021-05-13 Impact factor: 5.958