Changes with growth hormone treatment in growth hormone deficient children

Area of Science:

• Pediatric Endocrinology
• Growth Hormone Therapy
• Childhood Growth Disorders

Background:

• Idiopathic growth hormone deficiency (IGHD) causes poor growth in children.
• Patients with IGHD often have significantly below-average height and blunted growth hormone secretion.
• Effective long-term treatment strategies are crucial for improving growth outcomes.

Purpose of the Study:

• To evaluate the efficacy and safety of continuous human growth hormone (hGH) therapy in children with IGHD.
• To assess changes in height velocity (HV) and height standard deviation score (SDS) over 4 years of treatment.
• To identify factors influencing treatment outcomes, such as initial height deficit.

Main Methods:

• A cohort of 54 treatment-naïve children with IGHD received continuous hGH therapy for up to 4 years.
• Baseline characteristics included peak hGH < 10 ng/mL and height SDS < -2.5.
• Height velocity and height SDS were measured annually; adverse effects were monitored.

Main Results:

• Height velocity significantly increased in the first year (P < 0.001) and remained elevated compared to baseline throughout therapy.
• Patients achieved a mean height SDS gain of +1.72, improving from -4.11 to -2.21.
• Despite improvements, final height after 4 years remained below -2 SDS; greater initial height deficits correlated with less height gain.

Conclusions:

• Continuous hGH therapy effectively increases height velocity in children with IGHD.
• While treatment improves growth, final height may not reach the average range, especially in those with severe initial growth deficits.
• hGH therapy was well-tolerated, with no adverse effects or bone age acceleration observed.