New directions in growth hormone treatment in children

Renata Stawerska

doi:10.5114/pedm.2025.158544

eISSN: 2083-8441
ISSN: 2081-237X

Pediatric Endocrinology Diabetes and Metabolism

Current issue Archive Manuscripts accepted About the journal Supplements Editorial board Reviewers Abstracting and indexing Subscription Contact Instructions for authors Publication charge Ethical standards and procedures

Editorial System

Submit your Manuscript

Editorial Policies

Sarajevo Declaration on Integrity and Visibility of Scholarly Publications

Open access

Journal integrated with

4/2025
vol. 31

Send email

Copy url:

Editorial

New directions in growth hormone treatment in children

Renata Stawerska

^{1, 2}

Department of Paediatric and Adult Endocrinology, Medical University of Lodz, Poland
Department of Endocrinology and Metabolic Diseases, Polish Mother’s Memorial Hospital – Research Institute, Lodz, Poland

Pediatr Endocrinol Diabetes Metab 2025; 31 (4): 143-154

DOI: https://doi.org/10.5114/pedm.2025.158544

Online publish date: 2026/01/27

Article file

- Editorial New directions.pdf [0.69 MB]

Get citation

PlumX metrics:

1. Ranke MB, Wit JM. Growth hormone – past, present and future. Nat Rev Endocrinol. 2018; 14: 285–300. doi: 10.1038/nrendo.2018.22.

2. Maghnie M, Ranke MB, Geffner ME, et al. Safety and efficacy of pediatric growth hormone therapy: results from the full KIGS cohort. J Clin Endocrinol Metab 2022; 107: 3287–3301. doi: 10.1210/clinem/dgac517.

3. Sävendahl L, Polak M, Backeljauw P, et al. Long-term safety of growth hormone treatment in childhood: two large observational studies (NordiNet IOS and ANSWER). J Clin Endocrinol Metab 2021; 106: 1728–1741. doi: 10.1210/clinem/dgab080.

4. Aydın BK, Aycan Z, Sıklar Z, et al. Adherence to growth hormone therapy: results of a multicenter study. Endocr Pract 2014; 20: 46–51. doi: 10.4158/EP13194.OR.

5. Gomez R, Ahmed SF, Maghnie M, et al. Treatment adherence to injectable treatments in pediatric growth hormone deficiency compared with other chronic pediatric conditions: a systematic review. Front Endocrinol (Lausanne) 2022; 13: 795224. doi: 10.3389/fendo.2022.795224.

6. Rivolo S, Loftus J, Peter B, Fahey M, Kongnakorn T. Cost-effectiveness and cost-utility analysis of once-weekly somatrogon versus daily growth hormone for pediatric GHD in Ireland. J Med Econ 2023; 26: 963–972. doi: 10.1080/13696998.2023.2228167.

7. European Medicines Agency. Skytrofa (lonapegsomatropin): Summary of Product Characteristics. EMA/740430/2021 Rev.5; 2022. Accessed May 17, 2025.

8. European Medicines Agency. Sogroya (somapacitan): Summary of Product Characteristics. EMEA/H/005030 Rev.2; 2021. Accessed May 17, 2025.

9. European Medicines Agency. Ngenla (somatrogon): Summary of Product Characteristics. EMEA/H/C/005633 Rev.4; 2022. Accessed May 17, 2025.

10. Chatelain P, Malievskiy O, Radziuk K, et al. A randomized phase 2 study of long-acting TransCon GH versus daily GH in childhood growth hormone deficiency. J Clin Endocrinol Metab 2017; 102: 1673–1682. doi: 10.1210/jc.2016-3608.

11. Thornton PS, Maniatis AK, Aghajanova E, et al. Weekly lonapegsomatropin in treatment-naïve children with growth hormone deficiency: the phase 3 heiGHt trial. J Clin Endocrinol Metab 2021; 106: 3184–3195. doi: 10.1210/clinem/dgab529.

12. Maniatis AK, Casella SJ, Nadgir UM, et al. Safety and efficacy of lonapegsomatropin in children with growth hormone deficiency: enliGHten trial 2-year results. J Clin Endocrinol Metab 2022; 107: e2680–e2689. doi: 10.1210/clinem/dgac217.

13. Maniatis AK, Thornton PS, Nadgir UM, et al. Sustained height improvements up to 6 years in children with growth hormone deficiency treated with lonapegsomatropin: enliGHten final results. Horm Res Paediatr. 2025; 2025: 1–13. doi: 10.1159/000545064.

14. Sävendahl L, Battelino T, Rasmussen MH, et al. Weekly somapacitan in growth hormone deficiency: 4-year efficacy, safety, and treatment burden (REAL 3). J Clin Endocrinol Metab 2023; 108: 2569–2578. doi: 10.1210/clinem/dgad183.

15. Miller BS, Blair JC, Rasmussen MH, et al. Weekly somapacitan in children with growth hormone deficiency: the randomized phase 3 REAL4 trial. J Clin Endocrinol Metab 2022; 107: 3378–3388. doi: 10.1210/clinem/dgac513.

16. Brod M, Rasmussen MH, Alolga S, et al. Psychometric validation of the GHD-CTB and GHD-PTB instruments. Pharmacoecon Open 2023; 7: 121–138. doi: 10.1007/s41669-022-00373-z.

17. Miller BS, Blair JC, Rasmussen MH, et al. REAL4 2-year results and outcomes after switching from daily GH to somapacitan. J Clin Endocrinol Metab 2023; 108: 3090–3099. doi: 10.1210/clinem/dgad394.

18. Miller BS, Blair JC, Rasmussen MH, et al. Three-year efficacy, safety, and IGF-I outcomes of weekly somapacitan in children with GHD. Eur J Endocrinol 2025; 192: 651–661. doi: 10.1093/ejendo/lvaf096.

19. Zadik Z, Zelinska N, Iotova V, et al. Five-year results from an open-label extension of a phase 2 study of once-weekly somatrogon. J Pediatr Endocrinol Metab 2023; 36: 261–269. doi: 10.1515/jpem-2022-0359.

20. Deal CL, Steelman J, Vlachopapadopoulou E, et al. Weekly somatrogon versus daily somatropin in children with growth hormone deficiency: a phase 3 study. J Clin Endocrinol Metab 2022; 107: e2717–e2728. doi: 10.1210/clinem/dgac220.

21. Deal CL, Steelman J, Vlachopapadopoulou E, et al. Comparing weekly somatrogon with daily somatropin: a plain-language summary. Ther Adv Endocrinol Metab 2024; 15: 20420188241277404. doi: 10.1177/20420188241277404.

22. ClinicalTrials.gov. NCT03831880. Patient perception of treatment burden in weekly versus daily GH injections in children with GHD. Accessed May 17, 2025.

23. Loftus J, Quitmann J, Valluri SR. Health-related quality of life in prepubertal children treated with once-weekly somatrogon versus daily somatropin. Curr Med Res Opin 2024; 40: 175–184. doi: 10.1080/03007995.2023.2290623.

24. Choe J, Cara J, Stawerska R, et al. Results from the open-label extension of a global phase 3 study of once-weekly somatrogon in pediatric patients with growth hormone deficiency. 21st International Congress of Endocrinology 2024, Dubai, 01-03.03.2024. Abstract: 587.

25. Silverman L, Steelman J, Choe J, et al. Up to 5 years of once-weekly somatrogon treatment in pediatric patients with growth hormone deficiency: results from an open-label extension of a global Phase 3 study. 62nd Annual ESPE, Liverpool, 16-18.11.2024. Abstract: 8 P2-351.

26. Albers N, Cadarette S, Feakins B, et al. Long-acting growth hormone for pediatric growth hormone deficiency. J Endocr Soc 2025; 9: bvaf040. doi: 10.1210/jendso/bvaf040.

27. Horikawa R, Tanaka T, Hasegawa Y, et al. Once-weekly somatrogon versus daily somatropin in Japanese children with pediatric GHD. Horm Res Paediatr 2022; 95: 275–285. doi: 10.1159/000524600.

28. Khadilkar V, Radjuk KA, Bolshova E, et al. Twenty-four–month use of once-weekly GH (LB03002) in prepubertal children with GHD. J Clin Endocrinol Metab 2014; 99: 126–132.

29. Maniatis A, Cutfield W, Dattani M, et al. Long-acting growth hormone therapy in pediatric growth hormone deficiency: a consensus statement. J Clin Endocrinol Metab 2024; 110: e1232–e1240. doi: 10.1210/clinem/dgae834.

30. Cohen-Sela E, Oren A, Perl L, et al. Introduction of somatrogon in pediatric GHD: real-world insights from a national survey.

31. Endocr Pract 2025; 2025: S1530-891X(25)01272-8. doi: 10.1016/j.eprac.2025.11.008.

32. Büyükgebiz A, Demir A. Severe lipoatrophy in a toddler treated with long-acting growth hormone. Children (Basel) 2025; 12: 58. doi: 10.3390/children12010058.

33. Akhtar S, Berg B, Medina J, et al. Device preference for somapacitan versus somatrogon in patients and caregivers. Med Devices (Auckl) 2024; 17: 427–439. doi: 10.2147/MDER.S484354.

34. Medina J, Ter-Borch G, Kelepouris N, et al. Usability and preference of somapacitan pen versus lonapegsomatropin autoinjector. Patient Prefer Adherence 2025; 19: 1119–1131. doi: 10.2147/PPA.S505952.

35. Woelfle J, Kreitschmann-Andermahr I, Strasburger CJ, et al. Real-world evaluation of the first 100 patients receiving long-acting GH therapy (INSIGHTS-GHT). Orphanet J Rare Dis 2025; 20: 372. doi: 10.1186/s13023-025-03898-8.

36. Coyne E, Muthuvel G, Gutmark-Little I. Switching from daily somatropin to lonapegsomatropin in pediatric GHD. J Pediatr Endocrinol Metab 2025; 38: 248–253. doi: 10.1515/jpem-2024-0496.

37. Boller E, Raveendran S, Smith A, et al. Budget impact analysis of lonapegsomatropin in pediatric GHD. J Med Econ 2025; 28: 1733–1745. doi: 10.1080/13696998.2025.2561472.

38. Walpurgis K, Thomas A, Rauer A, et al. Detection of somatrogon in doping control urine samples. Sci Rep 2025; 15: 13160. doi: 10.1038/s41598-025-96361-4.

39. Johannsson G, Gordon MB, Rasmussen MH, et al. Once-weekly somapacitan in adults with growth hormone deficiency: a phase 3 trial. J Clin Endocrinol Metab 2020; 105: e1358–e1376. doi: 10.1210/clinem/dgaa049.

40. Lewiński A, Karbownik-Lewińska M, Wieczorek-Szukała K, et al. Contribution of ghrelin to the pathogenesis of growth hormone deficiency. Int J Mol Sci 2021; 22: 9066. doi: 10.3390/ijms22169066.

41. Dauber A, Wikiera B, Stawerska R, et al. OraGrowtH210 trial: oral LUM-201 in moderate pediatric GHD. Horm Res Paediatr 2024; 97 (suppl).

New directions in growth hormone treatment in children

Renata Stawerska 1, 2

Renata Stawerska

^{1, 2}