rhIGF-1/rhIGFBP-3 in Preterm Infants: A Phase 2 Randomized Controlled Trial.

Area of Science:

• Neonatal Medicine
• Pediatric Endocrinology
• Critical Care Medicine

Background:

• Retinopathy of prematurity (ROP) and bronchopulmonary dysplasia (BPD) are significant morbidities in extremely preterm infants.
• Recombinant human insulin-like growth factor 1 complexed with its binding protein (rhIGF-1/rhIGFBP-3) has been investigated for its potential to mitigate these complications.

Purpose of the Study:

• To evaluate the efficacy of rhIGF-1/rhIGFBP-3 in preventing ROP and other prematurity-related complications in extremely preterm infants.

Main Methods:

• A phase 2 randomized trial involving infants born between 23 and 27 weeks gestational age.
• Infants received either rhIGF-1/rhIGFBP-3 infusion or standard neonatal care, with follow-up to 40 weeks postmenstrual age.
• Primary endpoint was ROP severity; secondary endpoints included BPD, intraventricular hemorrhage (IVH), and growth.

Main Results:

• rhIGF-1/rhIGFBP-3 did not reduce ROP severity or occurrence.
• A significant reduction in severe BPD was observed (53% decrease in the full analysis set, 89% in the evaluable set).
• A trend towards decreased severe IVH (grades 3-4) was noted, but no effect on discharge time or growth.

Conclusions:

• rhIGF-1/rhIGFBP-3 therapy did not impact ROP development.
• The treatment demonstrated a significant benefit in reducing severe bronchopulmonary dysplasia in extremely preterm infants.
• A potential, though not statistically significant, reduction in severe intraventricular hemorrhage was observed.