Effectiveness and Safety of rhIGF-1 Therapy in Children: The European Increlex® Growth Forum Database Experience

Area of Science:

• Pediatric Endocrinology
• Growth Hormone Therapy
• Pharmacovigilance

Background:

• The EU Increlex® Growth Forum Database (IGFD) Registry monitors recombinant human insulin-like growth factor-1 (rhIGF-1) therapy in pediatric patients.
• Observational data collection focuses on clinical practice use of rhIGF-1 for growth failure.

Purpose of the Study:

• To assess the safety and effectiveness of rhIGF-1 therapy in children with growth failure within a real-world clinical setting.
• To identify predictors of treatment response and adverse events associated with rhIGF-1 therapy.

Main Methods:

• An open-label, observational study involving 195 children treated with rhIGF-1 from December 2008 to September 2013.
• Collection of safety and effectiveness data, including height velocity, height standard deviation score (SDS) changes, and adverse events.

Main Results:

• Mean height velocity in the first year was 6.9 cm/year, with higher response in treatment-naïve prepubertal patients (7.3 cm/year).
• Predictors of improved height SDS included female sex, younger age at therapy initiation, and lower baseline height SDS.
• Common adverse events included hypoglycemia (17.6%), lipohypertrophy (10.6%), and tonsillar hypertrophy (7.4%). Serious adverse events occurred in 16.5% of patients.

Conclusions:

• Real-world safety and effectiveness of rhIGF-1 align with findings from controlled trials.
• Severe growth phenotype and early rhIGF-1 initiation enhance height response and predict hypoglycemia risk.