A comparison of different definitions of growth response in short prepubertal children treated with growth hormone

Area of Science:

• Pediatric Endocrinology
• Growth Hormone Therapy
• Short Stature Management

Background:

• Defining poor growth response in children treated with growth hormone (GH) is a significant clinical challenge.
• Current management strategies lack standardized criteria for identifying inadequate response to GH therapy.

Purpose of the Study:

• To assess and evaluate various established criteria for defining poor growth response in children undergoing GH treatment.
• To analyze the efficacy of different response metrics across diverse pediatric short stature populations.

Main Methods:

• Retrospective multicenter study of 456 short, GH-treated, prepubertal children (Height SDS ≤-2).
• Included diverse etiologies: idiopathic GH deficiency (IGHD), idiopathic short stature (ISS), small for gestational age (SGA), organic GHD, Turner syndrome (TS), skeletal dysplasia, and others.
• Analyzed first-year height SDS gain, height velocity (HtV), and Insulin-like Growth Factor-I (IGF-I) SDS response.

Main Results:

• Median first-year height SDS gain was 0.65, with a height velocity of 8.67 cm/year.
• Nearly 50% of IGHD children met criteria for poor responders, with 28% showing <0.5 SDS gain and lower IGF-I SDS increases.
• Children with TS, skeletal dysplasia, or born SGA showed a higher proportion with <0.5 SDS gain.

Conclusions:

• A substantial number of children demonstrate a poor response to growth hormone therapy.
• Establishing clear criteria for poor responders is essential for optimizing short stature management in pediatric patients.
• Further research is needed to refine response definitions and improve treatment outcomes.