Do patients with idiopathic short stature or partial growth hormone deficiency need to continue growth hormone...

Area of Science:

• Pediatric Endocrinology
• Growth Hormone Therapy
• Idiopathic Short Stature

Background:

• Growth hormone (GH) therapy is established for increasing adult height in children with idiopathic short stature (ISS) and partial GH deficiency.
• The efficacy and benefit of continuing GH therapy into and through puberty remain less clear.
• Assessing the impact on predicted adult height (PAH) at puberty onset is crucial for treatment decisions.

Purpose of the Study:

• To evaluate whether continued GH therapy during puberty improves adult height compared to predicted adult height (PAH) at puberty onset.
• To identify factors influencing height gain during pubertal GH therapy.
• To analyze the cost-effectiveness of prolonged GH treatment.

Main Methods:

• Multicenter retrospective chart review of patients with ISS and partial GH deficiency.
• GH therapy initiated before or at puberty onset and continued until height velocity decreased (<2.5 cm/year).
• Collection of bone age, PAH, height standard deviation score (SDS), and Insulin-like Growth Factor 1 (IGF-1) SDS at various treatment points.

Main Results:

• GH therapy before puberty increased PAH significantly in both boys and girls.
• Continuing GH therapy through puberty did not yield a significant average increase in height over PAH at puberty onset (boys: -0.62 cm, girls: 1.82 cm).
• Factors like bone age, BMI SDS, mid-parental height, and duration of pubertal therapy influenced height gain, while peak GH and sex did not predict outcomes. The average cost for the final year of GH therapy was $122,000.

Conclusions:

• GH therapy initiated before puberty effectively increases predicted adult height (PAH).
• Continuation of GH therapy through puberty does not consistently provide additional significant height benefits and is associated with high costs.
• Clinicians should carefully consider the potential benefits versus the substantial costs when deciding on the continuation of GH therapy once adequate PAH is achieved.