Clinical Characteristics and Treatment Outcomes of Growth Hormone Therapy in Pediatric Patients: A Single-Center Experience and Review of Current Literature
DOI:
https://doi.org/10.37609/srinmed.55Keywords:
Growth hormone, child, growth hormone replacement therapyAbstract
Objective: Recombinant human growth hormone (rhGH) became available for clinical trials, and growth hormone deficiency has been treated more safely and effectively since 1985. Growth hormone therapy enables patients to attain an adult height consistent with their genetic target height.
The present study was undertaken to evaluate patients’ characteristics, their response to therapy, factors influencing outcome, and side effects of treatment.
Method: A retrospective file review was conducted. Out of 149 patients followed up with a GHD diagnosis, 92 patients whose files were accessible and who received treatment for at least 1 year were included in the study. Patients’ chronological ages, ages at diagnosis, sex, pretreatment bone ages (BA), pubertal stages, annual growth velocities, auxological data during treatment, and side effects were recorded.
Results: Age at diagnosis, sex, etiologic distributions, and auxological data were found to be similar to the results of Western countries. Height gain was found to be significant in the first year of treatment, and then growth velocity declined gradually. Age at the initiation of GH treatment has been shown to be negatively correlated with the response to therapy, emphasizing the need for early diagnosis and treatment of the condition. No significant adverse effects were observed throughout the 16-year follow-up period.
Conclusion: Early detection of pathological short stature, particularly Growth Hormone Deficiency (GHD), and the initiation of treatment at an early stage, lead to a superior final height outcome. Patients with GHD can be treated both effectively and safely with recombinant growth hormone therapy
Downloads
References
Brook CGD,Dattani MT. Management of Growth Disorders. İn: Brook CGD,Dattani MT editors.Handbook of Clinical Pediatric Endocrinology. second ed. Boston:Jhon Wiley&Sons;2012. p70-98
UptoDate Padilla EJR, Rogol AD. Diagnosis of growth hormone deficiency in children.Oct 05, 2023.
Lifshitz F,Nathan MB,Allen BD. Growth hormone treatment. İn: Lifshitz F, editor. Pediatric Endocrinology. 5th ed. Newyork:İnforma Healtcare;2007.p.113-143
Guyda HJ.Four Decades of Growth Hormone Therapy for Short Children: What Have We Achieved? J Clin Endocrinol Metb.1999;84:4307-4316.
Fujieda K, Tanaka T. Diagnosis of children with short stature:insights from KIGS. Ranke MB, Price DA,Reiter EO, editors. Growth Hormone Therapy in Pediatrics-20 Years of KIGS. 1st ed. Basel:Karger;2007.p.16-22
Darendeliler F, Andıran N, Evliyaoğlu O, Uçkun Kitapçı A. Büyüme Hormonu eksikliğinin tedavisi. In Saka N, Akçay T. Çocuk Endokrinolojisinde Uzlaşı Çocuk Endokrinolojisi ve Diyabet Derneği Yayınları – V, Nobel Tıp Kitabevleri, Kasım 2014, İstanbul. Sayfa:11-24.
Divall, Sara A, Radovick S. Normal and Aberrant Growth in Children. İn:Melmed S, Auchus RJ, Goldfine AB , Rosen CJ, ve Kopp P., editors. Williams Textbook of Endocrinology 15th ed.Philadelphia: Elsevier, 2024.22, 915-1000.e40
Lindholm J. Growth hormone:historical notes. Pituitary 2006;9:5-10
Çocuk Endokrinolojisi ve Diyabet Derneği. Boy kısalığı ve büyüme hormonu tedavisi uzlaşıları. Ankara: Çocuk Endokrinolojisi ve Diyabet Derneği; 2024. Erişim adresi: https://cocukendokrindiyabet.org/wp-content/uploads/boy-kisaligi-ve-buyume-hormonu-tedavisi-uzlasilari-2024.pdf.Erişim: 01/05/2025
Thomas M, Massa G, Craen M, Zegher F, Bourguignon JP, Heinrichs C, et al. Prevalence and demographic features of childhood growth hormone deficiency in Belgium during the period 1986-2001. European Journal of Endocrinology; 151: 67-72.
Berberoğlu M, Şıklar Z, Darendeliler F, Poyrazoğlu S, Darcan S, Işıgüven P, et al. Evaluation of permanent growth hormone deficiency (GHD) in young adults with childhood onset GHD: a multicenter study. J Clin Res Ped Endo 2008; 1(1): 30-37.
Rachimel M, Rota V, Atenafu E, Danerman D, Hamilton J. Final height in children with idiopathic growth hormone deficiency treated with a fixed dose of recombinant growth hormone. Horm Res 2007; 68(5): 236-243.
Hilken J. UK audit of childhood growth hormone prescription, 1998. Arch Dis Child 2001; 84: 387-389.
Cacciari E, Zuccihini S, Carla G. Endocrine function and morphological findings in patients with disorders of the hypothalamo-pituitary area: a study with magnetic resonance Archives of Disease in Childhood 1990; 65: 1199-1202.
Abrahams JJ, Trefelner E, Boulware SD. MR imaging in idiopathic growth hormone deficiency. AJNR Am J Neuroradiol 1998; 19: 1609-1615.
Bozzola M, Mengarda F, Sartirana P, Tato L, Chaussain JL. Long-term follow-up evaluation of magnetic resonance imaging in the prognosis of permanent GH deficiency. European Journal of Endocrinology 2000; 143: 493-496.
Frisch H. Characteristics of idiopathic growth hormone deficiency at the start of growth hormone therapy and the response to growth hormone. In: Ranke MB, Price DA, Reiter EO, editors. Growth Hormone Therapy in Pediatrics-20 Years of KIGS. 1st ed. Basel: Karger; 2007.p.108-115.
Ranke MB, Lindberg A, Albertsson-Wikland K, Wilton P, Price DA, Reiter EO. Increased response, but lower responsiveness, to growth hormone (GH) in very young children (aged 0-3 years) with idiopathic GH deficiency: analysis of data from KIGS. J Clin Endocrinol Metab 2005; 90(4): 1966-1971.
Bajpai A, Menon PS. Growth hormone therapy. Indian J Pediatr 2005; 72(2): 139-144.
Reiter EO, Cutfield WS. Growth hormone deficiency: puberty and final height. In: Ranke MB, Price DA, Reiter EO, editors. Growth Hormone Therapy in Pediatrics-20 Years of KIGS. 1st ed. Basel: Karger; 2007. p.136-145.
Bajpai A, Kabra M, Gupta AK, Menon PSN. Growth pattern and skeletal maturation following growth hormone therapy in growth hormone deficiency: factors influencing outcome. Indian J Pediatr 2006; 43: 593-599.
Reiter EO, Price AD, Wilton P, Albertsson-Wikland K, Ranke MB. Effect of growth hormone (GH) treatment on the near-final height of 1258 patients with idiopathic GH deficiency: analysis of a large international database. J Clin Endocrinol Metab 2006; 91(6): 2047-2054.
Bell J, Parker KL, Swinford RD, Hoffman AR, Maneatis T, Lippe B. Long-term safety of recombinant human growth hormone in children. J Clin Endocrinol Metab. 2010;95 (1): 167-77.
Maghnie M, Ranke MB, Geffner ME, Vlachopapadopoulou E, Ibáñez L, Carlsson 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
Swerdlow AJ, Cooke R, Beckers D, Borgström B, Butler G, Carel JC, et al. Cancer Risks in Patients Treated With Growth Hormone in Childhood: The SAGhE European Cohort Study. J Clin Endocrinol Metab. 2017;102(5):1661-72.
Sävendahl L, Cooke R, Tidblad A, et al. Long-term mortality after childhood growth hormone treatment: the SAGhE cohort study. Lancet Diabetes Endocrinol. 2020; 8: 683.
Wilton P, Mattsson AF, Darendeliler F. Growth hormone treatment in children is not associated with an increase in the incidence of cancer: experience from KIGS (Pfizer International Growth Database). J Pediatr. 2010;157 (2): 265-70
Child CJ, Zimmermann AG, Chrousos GP, et al. Safety outcomes during pediatric GH therapy: final results from the prospective GeNeSIS observational program. J Clin Endocrinol Metab. 2019;104(2):379–389.
Downloads
Published
Issue
Section
License
Copyright (c) 2025 Scientific Reports in Medicine

This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.
The Journal and content of this website is licensed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0) License. This is in accordance with the Budapest Open Access Initiative (BOAI) definition of open access. The Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0) allows users to copy, distribute and transmit unmodified article, and make noncommercial use of the article. The CC BY license permits non-commercial re-use of an open access article, as long as the author is properly attributed.