Growth Hormone Therapy in Short Stature Disorders

Growth hormone therapy has become a cornerstone in the management of paediatric and adolescent short stature arising from a variety of underlying causes. Originally approved for classical growth hormone deficiency, its indications have expanded to include idiopathic short stature, Turner syndrome, chronic renal insufficiency and small-for-gestational-age birth status. Treatment relies on recombinant human growth hormone administered by daily subcutaneous injections, with dosing calibrated to body weight or surface area. Clinical monitoring encompasses auxological measurements, bone age assessment and serum levels of insulin-like growth factor-I to ensure both efficacy and safety. Early initiation, ideally during prepubertal years, maximises potential for near-adult height gains and mitigates psychosocial burden. Advances in genetic screening have elucidated monogenic causes—such as ACAN mutations and SHOX haploinsufficiency—permitting a more personalised approach. Long-term follow-up addresses metabolic effects, adherence challenges and quality-of-life outcomes, underscoring the global significance of hormone replacement strategies for children who otherwise remain substantially below their genetic height potential.

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Growth Hormone Therapy in Short Stature Disorders publication trend

The graph below shows the total number of articles in growth hormone therapy in short stature disorders across all publications each year (not limited to Nature Index journals).

Technical terms

Growth Hormone (GH): A pituitary-derived peptide hormone that stimulates linear growth, protein synthesis and cell proliferation.

Growth Hormone Deficiency (GHD): A clinical state characterised by inadequate endogenous GH secretion leading to impaired somatic growth.

Idiopathic Short Stature (ISS): Height below –2 standard deviation scores with no identifiable endocrine, genetic or systemic cause.

Insulin-Like Growth Factor-I (IGF-I): A liver-derived mediator of GH action in tissues, used as a biomarker for GH efficacy and safety.

Standard Deviation Score (SDS): A statistical measure indicating how far an individual’s height or laboratory value deviates from age- and sex-matched population norms.

Radiomics: A quantitative approach that extracts high-dimensional data from medical images to characterise tissue features beyond visual assessment.

References

1. Development and Validation of a Prediction Model Using Sella Magnetic Resonance Imaging–Based Radiomics and Clinical Parameters for the Diagnosis of Growth Hormone Deficiency and Idiopathic Short Stature: Cross-Sectional, Multicenter Study. Journal of Medical Internet Research (2024).
2. Transient Isolated, Idiopathic Growth Hormone Deficiency—A Self-Limiting Pediatric Disease with Male Predominance or a Diagnosis Based on Uncertain Criteria? Lesson from 20 Years’ Real-World Experience with Retesting at One Center. International Journal of Molecular Sciences (2024).
3. The growth hormone–insulin-like growth factor-I axis in the diagnosis and treatment of growth disorders. Endocrine Connections (2018).
4. Early growth hormone treatment start in childhood growth hormone deficiency improves near adult height: analysis from NordiNet® International Outcome Study. European Journal of Endocrinology (2017).
5. Genetic screening confirms heterozygous mutations in ACAN as a major cause of idiopathic short stature. Scientific Reports (2017).
6. Adherence and long-term growth outcomes: results from the easypod™ connect observational study (ECOS) in paediatric patients with growth disorders. Endocrine Connections (2018).