Evaluation of the safety and efficacy of biosimilar recombinant growth hormone in children with growth hormone deficiency: non-inferiority, randomized, parallel, multicentric and Phase III trial

References

• Zapf J, Froesch E. Insulin-like growth factor I actions on somatic growth. In: Kostyo J, editor. Handbook of physiology. Philadelphia: American Physiological Society; 1999. p. 663–99.
   Google Scholar
• Sperling MA. Sperling pediatric endocrinology. 5th ed. Philadelphia: Elsevier; 2020. Available from: https://www.asia.elsevierhealth.com/sperling-pediatric-endocrinology-9780323625203.html
   Google Scholar
• Rohayem J, Drechsel H, Tittel B, et al. Long-term outcomes, genetics, and pituitary morphology in patients with isolated growth hormone deficiency and multiple pituitary hormone deficiencies: a single-centre experience of four decades of growth hormone replacement. Hormone Res Paediatrics. 2016;86(2):106–116. doi: 10.1159/000448098
   PubMed Web of Science ®Google Scholar
• Maghnie M, Ranke M, Geffner M, et al. Safety and efficacy of pediatric growth hormone therapy: results from the full KIGS cohort. J Clin Endocrinol Metab. 2022;107(12):3287–3301. doi: 10.1210/clinem/dgac517
   PubMed Web of Science ®Google Scholar
• Collett-Solberg PF, Jorge AA, Boguszewski M, et al. Growth hormone therapy in children; research and practice–a review. Growth Hormone IGF Res. 2019;44:20–32. doi: 10.1016/j.ghir.2018.12.004
   PubMed Web of Science ®Google Scholar
• Ranke MB, Wit JM. Growth hormone—past, present and future. Nat Rev Endocrinol. 2018;14(5):285–300. doi: 10.1038/nrendo.2018.22
   PubMed Web of Science ®Google Scholar
• Hage C, Gan H, Ibba A, et al. Advances in differential diagnosis and management of growth hormone deficiency in children. Nat Rev Endocrinol. 2021;17(10):608–624. doi: 10.1038/s41574-021-00539-5
   PubMed Web of Science ®Google Scholar
• Vanderlaan M, Maniatis A, Olney R, et al. Changes in manufacturing processes of biologic therapies can alter the immunogenicity profile of the product. Clin Pharmacol Ther. 2020;107(4):988–993. doi: 10.1002/cpt.1694
   PubMed Web of Science ®Google Scholar
• Arato T. Challenges for streamlining the development of biosimilars: a Japanese perspective. Clin Pharmacol Ther. 2023;113(1):30–36. doi: 10.1002/cpt.2762
   PubMed Web of Science ®Google Scholar
• Dudzinski DM, Kesselheim AS. Scientific and legal viability of follow-on protein drugs. Mass Medical Soc. 2008;358(8):843–849. doi: 10.1056/NEJMhle0706973
   Google Scholar
• Silva AC, Moreira J, Lobo JMS, et al. Current applications of pharmaceutical biotechnology. 2020;171:23–54. doi: 10.1007/10_2019_119
   Google Scholar
• Schellekens H, Moors E. Clinical comparability and European biosimilar regulations. Nature Biotechnol. 2010;28(1):28–31. doi: 10.1038/nbt0110-28
   PubMed Web of Science ®Google Scholar
• Noguera Peña A, Del Castillo Rodríguez C. Requirements for biosimilar authorization: a legal and comparative perspective–FDA versus EMA. Curr Sci. 2021;120(1):56. doi: 10.18520/cs/v120/i1/56-65
   Web of Science ®Google Scholar
• Saenger P. Ten years of biosimilar recombinant human growth hormone in Europe. Drug Des Devel Ther. 2017;11:1505–1507. doi: 10.2147/DDDT.S130317
   PubMed Web of Science ®Google Scholar
• Czepielewski M, Garret Q, Vencio S, et al. Efficacy and safety of a biosimilar recombinant human growth hormone (r-hGH cristalia) compared with reference r-hGH in children with growth hormone deficiency (CERES study): a randomized, multicentric, investigator-blind, phase 3 trial. Growth Hormone IGF Res. 2019;48:29–35. doi: 10.1016/j.ghir.2019.07.003
   PubMed Web of Science ®Google Scholar
• Marume A, Archary M, Mahomed S. Validation of growth standards and growth references: a review of literature. J Child Health Care. 2022;26(3):498–510. doi: 10.1177/13674935211024816
   PubMed Web of Science ®Google Scholar
• National Institute for Health and Care Excellence (NICE). Human growth hormone (somatropin) for the treatment of growth failure in children. Technology appraisal guidance [TA188]. [cited 2021 May 26]. 2010. Available from: https://www.nice.org.uk/guidance/ta188
   Google Scholar
• Lashari SK, Korejo HB, Memon YM. To determine frequency of etiological factors in short statured patients presenting at an endocrine clinic of a tertiary care hospital. Pak J Med Sci. 2014;30(4):858. doi: 10.12669/pjms.304.4619
   PubMed Web of Science ®Google Scholar
• Richmond E, Rogol AD. Treatment of growth hormone deficiency in children, adolescents and at the transitional age. Best Pract Res Clin Endocrinol Metab. 2016;30(6):749–755. doi: 10.1016/j.beem.2016.11.005
   PubMed Web of Science ®Google Scholar
• Butler G. Indications for growth hormone therapy. Paediatrics Child Health. 2007;17(9):356–360. doi: 10.1016/j.paed.2007.06.008
   Google Scholar
• Darendeliler F, Karagiannis G, Wilton P. Headache, idiopathic intracranial hypertension and slipped capital femoral epiphysis during growth hormone treatment: a safety update from the KIGS database. Hormone Res. 2007;68(Suppl. 5):41–47. doi: 10.1159/000110474
   PubMedGoogle Scholar
• Hughes IP, Harris M, Choong C, et al. Growth hormone regimens in Australia: analysis of the first 3 years of treatment for idiopathic growth hormone deficiency and idiopathic short stature. Clin Endocrinol. 2012;77(1):62–71. doi: 10.1111/j.1365-2265.2011.04230.x
   PubMed Web of Science ®Google Scholar
• Borrás Pérez MV, Kriström B, Romer T, et al. Ten Years of clinical experience with biosimilar human growth hormone: a review of safety data. Drug Des Devel Ther. 2017;Volume 11:1497–1503. doi: 10.2147/DDDT.S130909
   Web of Science ®Google Scholar
• Vance ML, Mauras N, Wood AJJ. Growth hormone therapy in adults and children. N Engl J Med. 1999;341(16):1206–1216. doi: 10.1056/NEJM199910143411607
   PubMed Web of Science ®Google Scholar
• Abucham J, Boguszewski MC. From the full KIGS cohort: on safety and efficacy of growth hormone treatment. J Clin Endocrinol Metab. 2023;108(1):e1–e2. doi: 10.1210/clinem/dgac625
   Google Scholar
• Youngster I, Rachmiel R, Pinhas-Hamiel O, et al. Treatment with recombinant human growth hormone during childhood is associated with increased intraocular pressure. J Paediatr. 2012;161(6):1116–1119. e1. doi: 10.1016/j.jpeds.2012.05.024
   PubMed Web of Science ®Google Scholar
• Ozturk T, Durmaz Engin C, Koksaldi S, et al. The short-term effects of intranasal steroids on intraocular pressure in pediatric population. Int Ophthalmol. 2022;42(12):3821–3827. doi: 10.1007/s10792-022-02402-6
   PubMed Web of Science ®Google Scholar