Non-evidence-based and off-label growth therapies - observations from consultations and practice patterns

Ilja Dubinski; Kees Noordam; Urs Eiholzer

doi:10.1530/endoabs.110.P651

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Endocrine Abstracts (2025) 110 P651 | DOI: 10.1530/endoabs.110.P651

ECEESPE2025 Poster Presentations Growth Axis and Syndromes (91 abstracts)

Non-evidence-based and off-label growth therapies - observations from consultations and practice patterns

Ilja Dubinski ¹ , 2 , Kees Noordam ¹ , 3 & Urs Eiholzer ¹

Author affiliations

¹Pediatric Endocrinology Center Zurich (PEZZ), Zurich, Switzerland; ²Department of Pediatrics, Dr. von Hauner Children’s Hospital, University Hospital, LMU Munich, Munich, Germany; ³Department of Paediatrics, Radboud University Medical Centre, Nijmegen, Netherlands

JOINT740

Introduction: Growth assessments do not always lead to established diagnoses and respective therapeutic options. This can cause considerable stress for patients and families. Some ask for alternative (or additional) non-evidence-based or off-label methods (e. g. growth hormone) for growth stimulation. However, the possibility of such off-label treatment has led to a change in the way the diagnosis of ‘short stature’ has been approached in recent years. A trend from ‘growth medicine’ to ‘growth engineering’ is currently being observed. There is no reliable data on the frequency and content of such enquiries.

Methods: An anonymous international online survey was conducted with paediatric endocrinologists from the Young European Society for Paediatric Endocrinology (“YES-Group”). The response rate was 47. 8 % (total 65 participants). The relevance of this topic from the perspective of paediatric endocrinologists was surveyed. In addition, the frequency/thematic content of corresponding enquiries and the attitudes of colleagues were surveyed. Specific prescription frequencies of off-label use treatments with growth hormone (GH), aromatase inhibitors (AI)/Gonadotropin-Releasing-Hormone (GnRH) analogues or both were queried separately.

Results: In 44. 6 % of cases, non-evidence-based methods were requested in every fifth to second consultation by patients or parents. 96. 9 % of colleagues considered the topic to be relevant. Most clarify the ineffectiveness (76. 9%) or potential risks (47. 7%). Most people asked about ‘special diets/optimisation of nutrition’ and ‘food supplements’, among other methods. 29. 3% prescribed off-label GH, 50. 8 % refused (for AI 15. 4 % and 33. 9 % respectively/for GH plus AI or GnRH analogues 19. 9 % and 33. 9 % respectively).

Table 1: Which types of non-evidence-based methods have your patients or parents inquired about this year? Select all that apply (n = 65, multiple choice, below 5 % not shown).
Non-evidence based method	Ratio of requests
Food supplements	75. 0%
Special diets/optimisation of nutrition	59. 4%
Off-label use of Growth Hormone	40. 6%
Sports (e. g. swimming)	35. 9%
Medical drugs (other than GH and/or AI)	34. 4%
Physiotherapy (e. g. stretching exercises)/physical thrp./manual thrp.	26. 6%
Herbal remedies/phytotherapy	26. 6%
Sleep regulation	25. 0%
Zinc	18. 8%
Arginine suppelment	17. 2%
Strength/weight training	14. 1%
Homeopathy	7. 8%
Stress reduction	7. 8%
Aromatase inhibitors	6. 3%

Conclusion: Non-evidence-based and off-label methods for growth stimulation play a significant role in patient counselling. A proactive approach in counselling for non-evidence-based methods could prevent disappointments or even risks. The off-label use of GH or GH plus AI/GnRH is frequent and the subject of ongoing societal, ethical and medical discourse.