Philippe Backeljauw1, Shankar Kanumakala2, Sandro Loche3, Karl Otfried Schwab4, Roland Werner Pfäffle5, Charlotte Höybye6,7, Elena Lundberg8, Tadej Battelino9, Berit Kriström8, Tomasz Giemza10, Hichem Zouater11. 1. Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. 2. Royal Alexandra Children's Hospital, Brighton, United Kingdom. 3. Hospital for Children Microcitemico "A. Cao", Cagliari, Italy. 4. Department of Pediatrics, University Medical Center, Freiburg, Germany. 5. Department of Pediatrics, University of Leipzig, Leipzig, Germany. 6. Department of Endocrinology, Karolinska University Hospital, Solna, Sweden. 7. Department of Molecular Medicine and Surgery, Karolinska Institute, Stockholm, Sweden. 8. Institute of Clinical Science/Pediatrics, Umeå University, Umeå, Sweden. 9. Department of Paediatric Endocrinology, Diabetes and Metabolic Diseases, University Medical Centre-University Children's Hospital, and University of Ljubljana, Ljubljana, Slovenia. 10. Sandoz Poland, Warsaw, Poland. 11. Sandoz GmbH, Holzkirchen, Germany.
Abstract
INTRODUCTION: PATRO Children is an international, observational, postmarketing surveillance study for a biosimilar recombinant human growth hormone (rhGH; somatropin, Omnitrope®; Sandoz), approved by the European Medicines Agency in 2006. We report safety and effectiveness data for patients with Turner syndrome (TS). METHODS: The study population included infants, children, and adolescents with TS who received Omnitrope® treatment according to standard clinical practice. Adverse events (AEs) were monitored for safety evaluation, and height velocity (HV), height standard deviation score (HSDS), and HVSDS were calculated to evaluate treatment effectiveness. RESULTS: As of August 2019, 348 TS patients were enrolled from 130 centers. At baseline, 314 patients (90.2%) were prepubertal and 284 patients (81.6%) were rhGH treatment naïve. The mean (range) age at baseline was 9.0 (0.7-18.5) years, and mean (SD) treatment duration in the study was 38.5 (26.8) months. Overall, 170 patients (48.9%) reported AEs, which were considered treatment related in 25 patients (7.2%). One treatment-related serious AE was reported (intracranial hypertension). Mean ΔHSDS after 3 years of therapy was +1.17 in treatment-naïve prepubertal patients and +0.1 in pretreated prepubertal patients. In total, 51 patients (31.1%) reached adult height (AH), 35 of whom were rhGH treatment naïve; in these patients, mean (SD) HSDS was -2.97 (1.03) at the start of Omnitrope® treatment, and they achieved a mean (SD) AHSDS of -2.02 (0.9). CONCLUSION: These data suggest that biosimilar rhGH is well tolerated and effective in TS patients managed in real-life clinical practice. Optimization of rhGH dose may contribute to a higher AH. The Author(s). Published by S. Karger AG, Basel.
INTRODUCTION: PATRO Children is an international, observational, postmarketing surveillance study for a biosimilar recombinant human growth hormone (rhGH; somatropin, Omnitrope®; Sandoz), approved by the European Medicines Agency in 2006. We report safety and effectiveness data for patients with Turner syndrome (TS). METHODS: The study population included infants, children, and adolescents with TS who received Omnitrope® treatment according to standard clinical practice. Adverse events (AEs) were monitored for safety evaluation, and height velocity (HV), height standard deviation score (HSDS), and HVSDS were calculated to evaluate treatment effectiveness. RESULTS: As of August 2019, 348 TS patients were enrolled from 130 centers. At baseline, 314 patients (90.2%) were prepubertal and 284 patients (81.6%) were rhGH treatment naïve. The mean (range) age at baseline was 9.0 (0.7-18.5) years, and mean (SD) treatment duration in the study was 38.5 (26.8) months. Overall, 170 patients (48.9%) reported AEs, which were considered treatment related in 25 patients (7.2%). One treatment-related serious AE was reported (intracranial hypertension). Mean ΔHSDS after 3 years of therapy was +1.17 in treatment-naïve prepubertal patients and +0.1 in pretreated prepubertal patients. In total, 51 patients (31.1%) reached adult height (AH), 35 of whom were rhGH treatment naïve; in these patients, mean (SD) HSDS was -2.97 (1.03) at the start of Omnitrope® treatment, and they achieved a mean (SD) AHSDS of -2.02 (0.9). CONCLUSION: These data suggest that biosimilar rhGH is well tolerated and effective in TS patients managed in real-life clinical practice. Optimization of rhGH dose may contribute to a higher AH. The Author(s). Published by S. Karger AG, Basel.
Authors: M B Ranke; A Lindberg; P Chatelain; P Wilton; W Cutfield; K Albertsson-Wikland; D A Price Journal: J Clin Endocrinol Metab Date: 2000-11 Impact factor: 5.958
Authors: Martha P D Zeger; Kavita Shah; Karen Kowal; Gordon B Cutler; Harvey Kushner; Judith L Ross Journal: Horm Res Paediatr Date: 2010-08-20 Impact factor: 2.852
Authors: Cheri Deal; Susan Kirsch; Jean-Pierre Chanoine; Sarah Lawrence; Elizabeth Cummings; Elizabeth T Rosolowsky; Seth D Marks; Nan Jia; Christopher J Child Journal: CMAJ Open Date: 2018-09-10