Three years of growth hormone therapy in children born small for gestational age: results from the ANSWER Program

Area of Science:

• Pediatric Endocrinology
• Growth Hormone Therapy
• Child Development

Background:

• Growth hormone (GH) therapy is vital for treating short stature and growth failure.
• Individual responses to GH therapy are influenced by birth size and GH status.
• Optimizing GH therapy requires understanding these modulating factors.

Purpose of the Study:

• To investigate the effect of birth size on GH therapy response.
• To assess how GH status impacts GH therapy outcomes in small for gestational age (SGA) children.
• To refine recommendations for GH therapy in SGA populations.

Main Methods:

• Analysis of data from the prospective, non-interventional American Norditropin Studies: Web-Enabled Research (ANSWER) Program.
• Inclusion of GH-naïve children categorized by birth size (SGA or not) and GH status (peak GH levels, IGHD, idiopathic short stature).
• Evaluation of growth outcomes, including height Standard Deviation Score (SDS) and Body Mass Index SDS, over 3 years.

Main Results:

• In patients with isolated growth hormone deficiency (IGHD), those not born SGA showed significantly greater improvements in height SDS and BMI SDS compared to IGHD patients born SGA.
• For children born SGA, the response to GH therapy demonstrated variability based on their specific GH status.
• GH status was identified as a key determinant of GH therapy effectiveness in SGA children.

Conclusions:

• GH status should be evaluated in children born SGA to personalize and optimize GH therapy.
• Current guidelines may need revision to incorporate GH status assessment for SGA patients.
• Tailoring GH therapy based on both birth size and GH status can lead to improved growth outcomes.