Beneficial Effects of Long-Term Growth Hormone Treatment on Adaptive Functioning in Infants With Prader-Willi...

Area of Science:

• Pediatrics
• Endocrinology
• Genetics

Background:

• Prader-Willi syndrome is a complex genetic disorder affecting multiple aspects of development.
• Adaptive functioning, crucial for daily living, is often significantly delayed in individuals with Prader-Willi syndrome.
• Growth hormone therapy is a common intervention, but its long-term impact on adaptive skills requires further investigation.

Purpose of the Study:

• To evaluate the effect of growth hormone (GH) treatment on adaptive functioning in children with Prader-Willi syndrome (PWS).
• To assess the long-term outcomes of GH treatment initiated during infancy versus later childhood.

Main Methods:

• A randomized controlled trial (RCT) initially included 75 children with PWS (42 infants, 33 prepubertal).
• 53 children received long-term GH treatment.
• Adaptive functioning was measured using the Vineland Adaptive Behavior Scale (VABS) during the RCT and after 7 years of treatment.

Main Results:

• Children with PWS exhibit a marked delay in adaptive functioning, correlated with older age and lower intelligence.
• Long-term follow-up revealed that earlier initiation of GH treatment during infancy resulted in significantly better adaptive skills.
• Repeated measurements confirmed a positive correlation between early GH intervention and improved long-term adaptive outcomes.

Conclusions:

• Early growth hormone treatment is crucial for enhancing adaptive functioning in infants and children with Prader-Willi syndrome.
• Initiating GH therapy in infancy provides the most substantial long-term benefits for adaptive skills in this population.
• This study underscores the importance of timely intervention in managing developmental delays associated with Prader-Willi syndrome.