Growth hormone treatment improves vitality and behavioural issues in children with Prader-Willi syndrome

Area of Science:

• Neurogenetics
• Pediatric Endocrinology
• Behavioral Science

Background:

• Prader-Willi syndrome (PWS) is a complex neurogenetic disorder characterized by obesity, short stature, intellectual deficits, and behavioral challenges.
• Growth hormone (GH) therapy is often used in PWS management, but its long-term effects on cognition and behavior require further investigation.

Purpose of the Study:

• To evaluate the effects of growth hormone (GH) treatment on cognitive function and behavior in children with Prader-Willi syndrome (PWS).
• To assess the lasting impact of GH therapy and the consequences of treatment cessation on PWS patients.

Main Methods:

• A randomized controlled trial involving 19 children (6 girls, 13 boys) with PWS.
• Treatment group received GH (0.033 mg/kg/day) for 2 years; control group had delayed/intermittent treatment.
• Treatment was discontinued for 6 months in both groups post-intervention.

Main Results:

• No significant differences in intellectual disabilities were observed between groups during or after treatment.
• Parents reported increased vitality in children during GH treatment.
• Abrupt cessation of GH treatment led to worsening behavioral problems, increased body fat, and decreased IGF-1 levels.

Conclusions:

• This study provides the first evidence that abruptly discontinuing growth hormone treatment exacerbates behavioral problems in children with Prader-Willi syndrome.
• GH therapy may influence behavioral aspects in PWS, necessitating careful consideration of treatment withdrawal protocols.