Phelps DL, et al. - Researchers tested the safety and effectiveness of myo-inositol in reducing type 1 retinopathy of prematurity (ROP) in infants younger than 28 weeks’ gestational age in this randomized clinical trial. As per outcomes, treatment with myo-inositol for up to 10 weeks did not reduce the risk of type 1 ROP or death vs placebo among these infants. Hence the use of myo-inositol among premature infants is not supported; however, the early termination of the trial does not allow for definitive conclusions.

Methods

• Researchers conducted a randomized clinical trial with 638 infants younger than 28 weeks’ gestational age enrolled from 18 neonatal intensive care centers throughout the US from April 17, 2014 to September 4, 2015.
• They performed a follow-up finally on February 12, 2016.
• With the planned enrollment of 1,760 participants, an absolute reduction in death or type 1 ROP of 7% with 90% power is expected.
• Owing to a statistically significantly higher mortality rate in the myo-inositol group, they terminated the trial early.
• For up to 10 weeks, they provided a 40-mg/kg dose of myo-inositol every 12 hours (initially intravenously, then enterally when feeding; n = 317) or placebo (n = 321).
• Unfavorable outcomes included type 1 ROP or death before determination of ROP outcome; survival without type 1 ROP was designated as favorable outcome.

Results

• Trial drug or placebo was given to 632 (99%) of enrolled patients; among these, 589 (92%) had a study outcome.
• In the myo-inositol group, researchers encountered death or type 1 ROP more frequently vs the placebo group (29% vs 21%, respectively; adjusted risk difference, 7% [95% CI, 0%-13%]; adjusted relative risk, 1.41 [95% CI, 1.08-1.83], P=.01).
• In the myo-inositol group, all-cause death before 55 weeks’ postmenstrual age occurred in 18% compared to 11% in the placebo group (adjusted risk difference, 6% [95% CI, 0%-11%]; adjusted relative risk, 1.66 [95% CI, 1.14-2.43], P=.007).
• Up to 7 days of receiving the ending dose, the most common serious adverse events encountered were necrotizing enterocolitis (6% for myo-inositol vs 4% for placebo), poor perfusion or hypotension (7% vs 4%, respectively), intraventricular hemorrhage (10% vs 9%), systemic infection (16% vs 11%), and respiratory distress (15% vs 13%).