Early Growth Hormone Initiation Leads to Favorable Long-Term Growth Outcomes in Children Born Small for Gestational...

Area of Science:

• Pediatric Endocrinology
• Growth and Development
• Pharmacological Interventions

Background:

• Early initiation of growth hormone (GH) therapy is recommended for children born small for gestational age (SGA) but is often delayed in real-world practice.
• Short children born SGA represent a significant population where timely intervention is crucial for optimal growth outcomes.

Purpose of the Study:

• To evaluate the impact of patient age at the initiation of GH therapy on long-term growth outcomes and safety.
• To compare growth trajectories in short children born SGA based on different age groups at GH therapy start.

Main Methods:

• Pooled analysis of data from two large observational studies: NordiNet® International Outcome Study and the ANSWER Program.
• Categorization of 3318 treatment-naïve, prepubertal patients born SGA into three age groups at GH initiation: 2 to <4 years, 4 to <6 years, and ≥6 years.
• Evaluation of auxological and safety data following up to 8 years of GH therapy.

Main Results:

• Patients initiating GH therapy between 2 to <4 years showed a significantly greater mean height standard deviation score improvement after 8 years compared to those initiating therapy at 4 to <6 years (+2.5 vs +2.2) and ≥6 years (+2.5 vs +1.7).
• Statistical significance was observed for these comparisons (P = 0.0054 and P < 0.0001, respectively).
• No unexpected adverse safety events were reported during the study period.

Conclusions:

• Early initiation of GH therapy in short children born SGA is a key factor for achieving optimal height outcomes.
• Long-term GH therapy in this pediatric population is associated with a reassuring safety profile.