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Auxological and metabolic effects of long-term treatment with recombinant growth hormone in children born small for gestational age: a retrospective study

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Abstract

Rationale

Children born small for gestational age (SGA) not showing catch-up during the first two years of life reportedly show an impaired growth rate and adult height, as well as a worse metabolic outcome, mainly in terms of glycemic and lipid profile, compared to general population. In SGA children with short stature, treatment with recombinant growth hormone (GH) is currently recommended until adolescence; therefore, it may last long-term.

Study methods

The aim of the current study was to evaluate the auxological and metabolic effects and the safety of long-term recombinant GH treatment in SGA children. The study included 15 SGA children (5 F, 10 M; mean age: 6.78 yrs) treated with GH for at least 48 months. Growth and metabolic parameters, including glycemic and lipid profile, transaminases, and urycemia, were collected every six months.

Results

Compared to baseline, SGA children showed a significant improvement in height, weight, and growth rate after four yaers of treatment with GH (p ≤ 0.002), being already evident after six months of treatment (p < 0.001). Noteworthy, patients showed a constant, significant improvement in height throughout the treatment, as it was significantly higher at each follow-up compared to the previous one, until 42 months of treatment, except at 30 months of treatment (p < 0.001 T6VST12; p < 0.01 T12VST18, T18VST24; p < 0.05 T30VST36, T36VST42). Considering metabolic parameters, compared to baseline, a recurring increase in glycemia (p ≤ 0.028 vs T30, T36, and T48) and decrease in AST (p ≤ 0.035 vs T36, T42, and T48) and an occasional decrease in LDL cholesterol (p ≤ 0.04 vs T24 and T42) and triglycerides (p = 0.008 vs T18) and increase in urycemia (p = 0.034 vs T42). Considering safety profile, treatment was well tolerated, as the most frequently reported adverse event was poor compliance (20%); no hyperglycemia, hypercholesterolemia or hyperstransaminasemia occurred throughout the treatment,

Conclusions

Long-term GH treatment showed to be effective in improving height and growth rate in SGA children, with a positive impact of metabolic profile and a safety profile, although glycemia should be carefully monitored over time.

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Acknowledgements

We are deeply grateful to Professor Martin Savage for his kindness, availability, and precious support in advising the manuscript.

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Authors and Affiliations

  1. UOSD di Auxologia ed Endocrinologia, AORN Santobono-Pausilipon, Napoli, Italy

    Rosario Ferrigno, Maria Cristina Savanelli, Daniela Cioffi, Valeria Pellino & Antonella Klain

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  1. Rosario Ferrigno
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  2. Maria Cristina Savanelli
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  3. Daniela Cioffi
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  4. Valeria Pellino
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  5. Antonella Klain
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Correspondence to Rosario Ferrigno.

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Conflict of interest

The Authors hereby declare to acknowledge the ethical requirements of the Journal of Endocrinological Investigation and to respect the ethic rules of the journal. Moreover, the Authors report the following conflict of interest: R.F.: honoraria by Savio Pharma, sponsorships by Novartis, Recordati, Novo Nordisk, Savio Pharma, Corcept Therapeutics M.C.S.: sponsorship by Novo Nordisk, Savio Pharma D.C. : honoraria by Novo Nordisk; sponsorships by Merck Serono, Pfizer, Novo Nordisk V.P. : honoraria by Novo Nordisk, Merck Serono; sponsorship by Pfizer A.K. research grant by Pfizer Inc, Merck Serono; honoraria by Novo Nordisk, Merck Serono; sponsorships by Novo Nordisk, Merck Serono, Pfizer, Lilly, Buona. The present study was conducted thanks to the financial support of Pfizer. Pfizer was not involved in study design, study conduction, data analysis, and writing.

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Ferrigno, R., Savanelli, M.C., Cioffi, D. et al. Auxological and metabolic effects of long-term treatment with recombinant growth hormone in children born small for gestational age: a retrospective study. Endocrine 84, 213–222 (2024). https://doi.org/10.1007/s12020-023-03665-4

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