Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Clinical Research Article
  • Published:

Linear growth and metabolic outcomes of growth hormone treatment in childhood-onset craniopharyngioma: the OUTSPREAD study

Pediatric Research (2026) Cite this article

Abstract

Background

Growth hormone (GH) deficiency is common following craniopharyngioma resection. This study aimed to compare growth status and metabolic parameters before and after GH treatment for patients with craniopharyngioma.

Methods

This retrospective study included 134 patients with multicenter study for Ideal Outcomes of Pediatric Rare Endocrine and Metabolic Diseases in Korea (OUTSPREAD study), who received GH treatment for ≥1 year. Data on growth and metabolic parameters before and after GH treatment (at 1, 2, and 3 years, and at the end of treatment) were collected.

Results

The mean age at diagnosis was 7.6 years, with GH treatment initiated at 9.4 years and concluded at 15.7 years. Before GH treatment, height and weight were below average, while body mass index (BMI) was above average. Following GH treatment, height and weight significantly increased while BMI decreased. Analysis using a sex-, age- and steroid dose- adjusted linear mixed model showed significantly decreased aspartate aminotransferase, alanine aminotransferase and total cholesterol levels after treatment. Random glucose levels increased slightly but remained within the normal range, and glycosylated hemoglobin levels remained stable.

Conclusion

This is the first longitudinal report showing the positive effects of GH treatment for craniopharyngioma in Korean patients.

Impact

  • After surgery for craniopharyngioma, GH treatement is essential for patients with GH deficiency. In this first multicenter Korean cohort evaluating long-term growth and metabolic outcomes, GH treatment significantly improved height, weight, and BMI SDS. Hepatic indices (AST/ALT) and lipid parameters (total cholesterol) decreased during treatment. Random glucose increased slightly but remained within the normal range, and HbA1c showed no significant change. These findings support GH treatment as a key component of long-term management with benefits beyond linear growth.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on SpringerLink
  • Instant access to the full article PDF.

USD 39.95

Prices may be subject to local taxes which are calculated during checkout

Fig. 1: Study selection and baseline population.
The alternative text for this image may have been generated using AI.
Fig. 2: Auxological parameter changes from baseline to the completion of GH treatment.
The alternative text for this image may have been generated using AI.
Fig. 3: Metabolic parameter changes from baseline to the end of GH treatment.
The alternative text for this image may have been generated using AI.

Similar content being viewed by others

Data availability

Data are available from the authors on reasonable request as part of a collaboration project.

References

  1. Olsson, D. S., Andersson, E., Bryngelsson, I. L., Nilsson, A. G. & Johannsson, G. Excess mortality and morbidity in patients with craniopharyngioma, especially in patients with childhood onset: a population-based study in Sweden. J. Clin. Endocrinol. Metab. 100, 467–474 (2015).

    Article  CAS  PubMed  Google Scholar 

  2. Müller, H. L. Childhood craniopharyngioma. Pituitary 16, 56–67 (2013).

    Article  PubMed  Google Scholar 

  3. Wijnen, M. et al. Very long-term sequelae of craniopharyngioma. Eur. J. Endocrinol. 176, 755–767 (2017).

    Article  CAS  PubMed  Google Scholar 

  4. Jazbinšek, S. et al. Prevalence of endocrine and metabolic comorbidities in a national cohort of patients with craniopharyngioma. Horm. Res. Paediatr. 93, 46–57 (2020).

    Article  PubMed  Google Scholar 

  5. Erfurth, E. M. Craniopharyngioma-an update on metabolic and cognitive complications and new therapy. J. Intern Med. 294, 269–280 (2023).

    Article  CAS  PubMed  Google Scholar 

  6. Roth, C., Wilken, B., Hanefeld, F., Schröter, W. & Leonhardt, U. Hyperphagia in children with craniopharyngioma is associated with hyperleptinaemia and a failure in the downregulation of appetite. Eur. J. Endocrinol. 138, 89–91 (1998).

    Article  CAS  PubMed  Google Scholar 

  7. Shaikh, M. G., Grundy, R. G. & Kirk, J. M. Reductions in basal metabolic rate and physical activity contribute to hypothalamic obesity. J. Clin. Endocrinol. Metab. 93, 2588–2593 (2008).

    Article  CAS  PubMed  Google Scholar 

  8. Srinivasan, S. et al. Features of the metabolic syndrome after childhood craniopharyngioma. J. Clin. Endocrinol. Metab. 89, 81–86 (2004).

    Article  CAS  PubMed  Google Scholar 

  9. Li, S. et al. Metabolic effects of recombinant human growth hormone replacement therapy on juvenile patients after craniopharyngioma resection. Int J. Endocrinol. 2022, 7154907 (2022).

    Article  PubMed  PubMed Central  Google Scholar 

  10. Pei, L. L., Guo, Y., Chen, H. & Zhong, L. Y. Benefits and risks evaluation of recombinant human growth hormone replacement therapy in children with GHD after craniopharyngioma surgery. J. Pediatr. Endocrinol. Metab. 36, 484–491 (2023).

    Article  PubMed  Google Scholar 

  11. Lee, Y. J. et al. Cohort profile: multicenter networks for ideal outcomes of rare pediatric endocrine and metabolic diseases in Korea (OUTSPREAD study). Ann. Pediatr. Endocrinol. Metab. 29, 349–355 (2024).

    Article  PubMed  PubMed Central  Google Scholar 

  12. Kim, J. H. et al. The 2017 Korean National Growth Charts for children and adolescents: development, improvement, and prospects. Korean J. Pediatr. 61, 135–149 (2018).

    Article  PubMed  PubMed Central  Google Scholar 

  13. Olsson, D. S. et al. Tumour recurrence and enlargement in patients with craniopharyngioma with and without GH replacement therapy during more than 10 years of follow-up. Eur. J. Endocrinol. 166, 1061–1068 (2012).

    Article  CAS  PubMed  Google Scholar 

  14. Jiang, Z. et al. Short-term efficacy and safety of a lower dose of polyethylene glycol recombinant human growth hormone in children with growth hormone deficiency: a randomized, dose-comparison study. Front. Pharm. 13, 955809 (2022).

    Article  CAS  Google Scholar 

  15. Geffner, M. E., Ranke, M. B. & Wajnrajch, M. P. An overview of growth hormone therapy in pediatric cases documented in the Kabi International Growth Study (Pfizer International Growth Database). Ann. Pediatr. Endocrinol. Metab. 29, 3–11 (2024).

    Article  PubMed  PubMed Central  Google Scholar 

  16. Okońska, M. et al. The evaluation of the influence of growth hormone therapy on growing process and metabolic functions in patients after treatment of craniopharyngioma. Pediatr. Endocrinol. Diab. Metab. 16, 19–24 (2010).

    Google Scholar 

  17. Rodari, G., Cattoni, A. & Albanese, A. Final height in growth hormone-deficient childhood cancer survivors after growth hormone therapy. J. Endocrinol. Invest 43, 209–217 (2020).

    Article  CAS  PubMed  Google Scholar 

  18. Boot, A. M., Engels, M. A., Boerma, G. J., Krenning, E. P. & De Muinck Keizer-Schrama, S. M. Changes in bone mineral density, body composition, and lipid metabolism during growth hormone (GH) treatment in children with GH deficiency. J. Clin. Endocrinol. Metab. 82, 2423–2428 (1997).

    CAS  PubMed  Google Scholar 

  19. Boekhoff, S. et al. Long-term effects of growth hormone replacement therapy in childhood-onset craniopharyngioma: results of the German Craniopharyngioma Registry (HIT-Endo). Eur. J. Endocrinol. 179, 331–341 (2018).

    Article  CAS  PubMed  Google Scholar 

  20. Heinks, K. et al. Quality of life and growth after childhood craniopharyngioma: results of the multinational trial KRANIOPHARYNGEOM 2007. Endocrine 59, 364–372 (2018).

    Article  CAS  PubMed  Google Scholar 

  21. Xue, J., Liang, S., Ma, J. & Xiao, Y. Effect of growth hormone therapy on liver enzyme and other cardiometabolic risk factors in boys with obesity and nonalcoholic fatty liver disease. BMC Endocr. Disord. 22, 49 (2022).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  22. Doycheva, I., Erickson, D. & Watt, K. D. Growth hormone deficiency and NAFLD: an overlooked and underrecognized link. Hepatol. Commun. 6, 2227–2237 (2022).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  23. Takahashi, Y. Nonalcoholic fatty liver disease and adult growth hormone deficiency: an under-recognized association? Best. Pr. Res Clin. Endocrinol. Metab. 37, 101816 (2023).

    Article  CAS  Google Scholar 

  24. Møller, N. & Jørgensen, J. O. Effects of growth hormone on glucose, lipid, and protein metabolism in human subjects. Endocr. Rev. 30, 152–177 (2009).

    Article  PubMed  Google Scholar 

  25. Seminara, S. et al. Effect of long-term growth hormone treatment on carbohydrate metabolism in children with growth hormone deficiency. Clin. Endocrinol. 49, 125–130 (1998).

    Article  CAS  Google Scholar 

  26. Saenger, P. Metabolic consequences of growth hormone treatment in paediatric practice. Horm. Res. 53, 60–69 (2000).

    CAS  PubMed  Google Scholar 

  27. ElSayed, N. A. et al. 2. Classification and diagnosis of diabetes: standards of care in diabetes-2023. Diab. Care 46, S19–s40 (2023).

    Article  CAS  Google Scholar 

  28. Lee, D. H., Kim, H. Y., Park, J. Y., Kim, J. & Park, J. H. New-onset type 1 and type 2 diabetes among Korean youths during the COVID-19 Pandemic. JAMA Pediatr. 179, 155–162 (2025).

    Article  PubMed  PubMed Central  Google Scholar 

  29. Nguyen Quoc, A. et al. GH and childhood-onset craniopharyngioma: when to initiate GH replacement therapy? J. Clin. Endocrinol. Metab. 108, 1929–1936 (2023).

    Article  CAS  PubMed  Google Scholar 

Download references

Acknowledgements

The study analyzed the data collected from multi-center cohorts. The authors express their gratitude for all participants of this cohort-study, without whom the study would not have been possible. The content is solely the responsibility of the authors. Also, we would like to thank Editage (www.editage.co.kr) for English language editing.

Funding

This research was supported and funded by SNUH Lee Kun-hee Child Cancer & Rare Disease Project, Republic of Korea (grant number: 26C-047-0000).

Author information

Author notes

  1. These authors contributed equally: Moon Bae Ahn, Junghwan Suh.

Authors and Affiliations

  1. Department of Pediatrics, Severance Children’s Hospital, Yonsei University College of Medicine, Seoul, Republic of Korea

    Sujin Kim & Junghwan Suh

  2. Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea

    Sung Yoon Cho

  3. Department of Pediatrics, Hallym University Sacred Heart Hospital, Hallym University College of Medicine, Anyang, Republic of Korea

    Yena Lee

  4. Department of Pediatrics, Chonnam National University Hospital, Gwangju, Republic of Korea

    Seong Hwan Chang

  5. Department of Pediatrics, Kyungpook National University Chilgok Hospital, Daegu, Republic of Korea

    Jung-Eun Moon

  6. Department of Pediatrics, Seoul National University Bundang Hospital, Seongnam, Republic of Korea

    Jaehyun Kim

  7. Department of Pediatrics, Seoul National University College of Medicine, Seoul, Republic of Korea

    Jaehyun Kim, Young Ah Lee & Yun Jeong Lee

  8. Department of Pediatrics, Seoul National University Children’s Hospital, Seoul, Republic of Korea

    Young Ah Lee & Yun Jeong Lee

  9. Department of Pediatrics, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea

    Moon Bae Ahn

Authors
  1. Sujin Kim
    View author publications

    Search author on:PubMed Google Scholar

  2. Sung Yoon Cho
    View author publications

    Search author on:PubMed Google Scholar

  3. Yena Lee
    View author publications

    Search author on:PubMed Google Scholar

  4. Seong Hwan Chang
    View author publications

    Search author on:PubMed Google Scholar

  5. Jung-Eun Moon
    View author publications

    Search author on:PubMed Google Scholar

  6. Jaehyun Kim
    View author publications

    Search author on:PubMed Google Scholar

  7. Young Ah Lee
    View author publications

    Search author on:PubMed Google Scholar

  8. Yun Jeong Lee
    View author publications

    Search author on:PubMed Google Scholar

  9. Moon Bae Ahn
    View author publications

    Search author on:PubMed Google Scholar

  10. Junghwan Suh
    View author publications

    Search author on:PubMed Google Scholar

Contributions

S.K. and H.J.S. made substantial contributions to the conception and design, interpretation of data, and drafting of the manuscript. S.Y.C. and Y.L. contributed to the conception and execution of the study. J.S. and M.A. made contributions to the conception and design of the study. C.J.K. and J.E.M. contributed to the preparation of data and figures. J.K., H.C.S. and Y.A.L. contributed to the analysis and interpretation of data. Y.A.L. contributed to the critical revision of important intellectual content.

Corresponding authors

Correspondence to Moon Bae Ahn or Junghwan Suh.

Ethics declarations

Competing interests

The authors declare no competing interests.

Ethics approval

This study was approved by the Institutional Review Board of the Yonsei University College of Medicine (approval number: 4-2024-0636).

Additional information

Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Rights and permissions

Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Kim, S., Cho, S.Y., Lee, Y. et al. Linear growth and metabolic outcomes of growth hormone treatment in childhood-onset craniopharyngioma: the OUTSPREAD study. Pediatr Res (2026). https://doi.org/10.1038/s41390-026-04971-z

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Version of record:

  • DOI: https://doi.org/10.1038/s41390-026-04971-z

Search

Advanced search

Quick links