Effectiveness of Recombinant Human Growth Hormone Therapy in Small-for-Gestational-Age Children With Short Stature: A

Sanghee Park1, Yena Lee2, Hwal Rim Jeong3

  • 1Department of Pediatrics, Hallym University Kangnam Sacred Heart Hospital, Seoul, Republic of Korea.

Clinical Endocrinology
|August 2, 2026
PubMed

Insights

Pathogenic genetic variants impact growth outcomes in children with persistent short stature. While recombinant human growth hormone (rhGH) therapy helps, those with variants show a slower growth response over time, emphasizing genetic evaluation for personalized treatment.

Area of Science:

  • Pediatric Endocrinology
  • Genetics
  • Growth Disorders

Background:

  • Children born small for gestational age with persistent short stature (SGA-SS) often exhibit growth challenges.
  • Genetic factors can significantly influence growth patterns and response to therapy.

Purpose of the Study:

  • To assess the influence of pathogenic genetic variants on growth outcomes in SGA-SS children treated with recombinant human growth hormone (rhGH) for three years.
  • To compare growth trajectories between genetically characterized SGA-SS patient groups.

Main Methods:

  • Retrospective cohort study of 179 SGA-SS children.
  • Classification into variant-positive (n=30) and variant-negative (n=149) groups based on genetic evaluation.
  • Longitudinal assessment of clinical characteristics and growth outcomes over 3 years of rhGH therapy using linear mixed models.

Main Results:

  • Variant-positive children had lower baseline height SDS and higher rates of intellectual disability and congenital anomalies.
  • rhGH therapy improved height SDS in both groups after 3 years.
  • A progressive attenuation of height SDS gain was observed in the variant-positive group over the treatment period.

Conclusions:

  • rhGH therapy is effective for linear growth in SGA-SS children.
  • Genetic evaluation is crucial as pathogenic variants are associated with lower baseline stature and reduced growth response to rhGH.
  • Personalized treatment strategies informed by genetic findings are important for optimizing growth outcomes in SGA-SS patients.
Abstract

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