Primary response in GHD children treatment as a predictor for long-term therapy effectiveness therapy effectiveness

Area of Science:

• Pediatrics
• Endocrinology
• Growth Disorders

Background:

• Recombinant human growth hormone (rhGH) effectively treats short stature in growth hormone deficiency (GHD).
• However, a significant portion of patients do not achieve satisfactory outcomes with rhGH therapy.
• Predicting long-term response is crucial for optimizing GHD treatment.

Purpose of the Study:

• To assess the predictive value of the first-year height increase on long-term outcomes in children with GHD treated with rhGH.
• To identify early indicators of treatment success in rhGH therapy for short stature.

Main Methods:

• A cohort of 165 children with GHD and short stature were treated with rhGH for at least one year.
• Patients were categorized into good responders (GR) and poor responders (PR) based on height standard deviation score (SDS) change after the first year.
• Longitudinal data including height, weight, bone age, and insulin-like growth factor levels were collected and analyzed.

Main Results:

• The good responders (GR) group exhibited a significantly higher mean height velocity SDS (1.19 ±0.41/year) over five years compared to the poor responders (PR) group (0.59 ±0.38/year).
• Statistical analysis confirmed a significant difference (p < 0.05) in height velocity between the two groups.
• These findings highlight a clear distinction in growth response between early good and poor responders.

Conclusions:

• The initial response to rhGH treatment in children with GHD serves as a reliable predictor of long-term therapy outcomes.
• Early assessment of height velocity SDS after one year of rhGH therapy can guide treatment adjustments and predict final height achievement.
• This study underscores the importance of early treatment response in managing GHD and optimizing growth in affected children.