Long-term side effects of growth hormone treatment in children with Prader-Willi syndrome

Area of Science:

• Pediatric endocrinology
• Genetics and rare diseases
• Clinical pharmacology

Background:

• Prader-Willi syndrome (PWS) is a complex genetic disorder affecting growth, metabolism, and development.
• Growth hormone (GH) therapy is used in PWS to improve linear growth and body composition.
• Potential adverse events associated with GH treatment in PWS require thorough investigation.

Purpose of the Study:

• To review and discuss the latest data on side effects of long-term GH treatment in children with PWS.
• To evaluate the safety profile of GH therapy in the context of PWS management.
• To inform clinical practice regarding potential risks and benefits of GH in PWS.

Main Methods:

• Literature review of studies on GH treatment in Prader-Willi syndrome.
• Analysis of reported adverse events, including growth, behavior, and specific complications.
• Discussion of the latest findings on safety and efficacy.

Main Results:

• GH treatment in PWS primarily aims to stimulate growth and increase lean muscle mass.
• Favorable effects on behavior and cognitive performance have been observed with GH therapy.
• Significant adverse events include scoliosis and breathing disorders, with potential links to mortality, especially early in treatment. Obstructive sleep apnea is also noted.

Conclusions:

• GH therapy offers benefits for growth and potentially behavior in PWS children.
• Careful monitoring for adverse events, particularly respiratory issues and scoliosis, is crucial during long-term GH treatment.
• The risk-benefit profile of GH in PWS necessitates ongoing evaluation and individualized patient management.