GH-IGF-1 axis and rhGH outcomes in children with GHD, ISS and SGA: a systematic review and meta-analysis

Area of Science:

• Pediatric Endocrinology
• Growth Hormone Therapy
• Metabolic Disorders

Background:

• Growth hormone deficiency (GHD), idiopathic short stature (ISS), and small for gestational age (SGA) are key indications for recombinant human growth hormone (rhGH) therapy in children.
• Limited direct comparisons exist for GH-IGF-1 axis profiles and treatment outcomes across these pediatric conditions.

Purpose of the Study:

• To systematically compare GH-IGF-1 axis profiles and rhGH treatment outcomes in pediatric GHD, ISS, and SGA populations.
• To identify factors predicting treatment response to rhGH therapy.

Main Methods:

• A systematic literature search of PubMed/MEDLINE (January 2005-December 2024) identified 47 studies with 18,642 children.
• Study quality was assessed using Newcastle-Ottawa Scale, Cochrane RoB 2.0, and AMSTAR-2.
• Random-effects meta-analyses were employed to compare outcomes, including height velocity and IGF-1 standard deviation scores (SDS).

Main Results:

• Baseline IGF-1 SDS was significantly lower in GHD (-2.9) compared to ISS (-1.5) and SGA (-1.3).
• Children with complete GHD exhibited greater first-year height velocity and height SDS gain compared to ISS and SGA.
• Younger age at treatment initiation and lower baseline IGF-1 SDS were significant predictors of a more robust rhGH treatment response.

Conclusions:

• rhGH therapy demonstrates efficacy in improving growth and IGF-1 outcomes across GHD, ISS, and SGA pediatric populations.
• Complete GHD shows the most pronounced response to rhGH treatment.
• Early initiation of rhGH therapy and utilizing IGF-1 levels to guide treatment strategies are recommended.