Early-onset growth hormone treatment in Prader-Willi syndrome attenuates transition to severe obesity

Area of Science:

• Pediatric Endocrinology
• Genetics and Genetic Diseases
• Metabolic Disorders

Background:

• Prader-Willi syndrome (PWS) is a complex genetic disorder characterized by early feeding issues followed by hyperphagia and severe obesity.
• Growth hormone (GH) therapy is an established treatment for PWS, aimed at improving growth, body composition, and body mass index (BMI).

Purpose of the Study:

• To investigate the impact of the age of initiating GH therapy on the growth and BMI trajectories in children with PWS.
• To compare the long-term effects of early-onset versus late-onset GH therapy on height and BMI in PWS patients.

Main Methods:

• Analysis of height and BMI data from 114 children (58 boys) in the REPAR - Czech national GH registry.
• Patients were divided into two groups: early-onset (EO) group (n=69, started GH before 2 years) and late-onset (LO) group (n=45, started GH at 7.1 years).

Main Results:

• Height standard deviation scores (SDS) normalized in both groups after 5 years of GH therapy (EO: -0.1 ± 1.1 SDS).
• While LO children were obese at treatment initiation (BMI-SDS: 2.9 ± 2.2), their BMI-SDS decreased after 1 year.
• EO children, initially underweight (BMI-SDS: -0.9 ± 1.2), became overweight within 3 years, but their highest lifetime BMI-SDS was significantly lower than in the LO group (2.2 ± 2.6 vs 3.7 ± 2.2).

Conclusions:

• GH therapy effectively normalizes body height in children with PWS.
• Although BMI-SDS becomes similar in both early and late-onset groups after 5 years, early initiation of GH therapy is associated with lower peak BMI-SDS, suggesting a better long-term weight management profile.