Related Experiment Video
Updated: May 2, 2026
Oxygen-Induced Retinopathy Model for Ischemic Retinal Diseases in Rodents
Published on: September 16, 2020
Effects of Myo-inositol on Type 1 Retinopathy of Prematurity Among Preterm Infants <28 Weeks' Gestational Age: A
Dale L Phelps1, Kristi L Watterberg2, Tracy L Nolen3
1School of Medicine and Dentistry, University of Rochester, Rochester, New York.
Insights
Myo-inositol did not reduce retinopathy of prematurity (ROP) or death in preterm infants. The study was stopped early due to increased mortality in the myo-inositol group, limiting definitive conclusions.
Area of Science:
- Neonatalogy
- Perinatal Medicine
- Clinical Trials
Background:
- Previous studies suggested myo-inositol may reduce retinopathy of prematurity (ROP) severity and mortality in preterm infants.
- However, large-scale trials evaluating its efficacy and safety were lacking.
Purpose of the Study:
- To assess the efficacy and safety of myo-inositol in preventing type 1 ROP in infants born before 28 weeks' gestational age.
- To evaluate adverse events associated with myo-inositol treatment.
Main Methods:
- A randomized clinical trial involving 638 infants younger than 28 weeks' gestational age.
- Infants received either myo-inositol (40 mg/kg every 12 hours) or placebo for up to 10 weeks.
- The trial was terminated early due to observed higher mortality in the myo-inositol group.
Main Results:
- Treatment with myo-inositol did not decrease the incidence of type 1 ROP or death compared to placebo (29% vs 21%).
- All-cause mortality before 55 weeks' postmenstrual age was higher in the myo-inositol group (18% vs 11%).
- Serious adverse events, including necrotizing enterocolitis and intraventricular hemorrhage, were noted in both groups, with some events more frequent in the myo-inositol group.
Conclusions:
- Myo-inositol treatment did not reduce the risk of type 1 ROP or death in very preterm infants.
- The findings do not support the use of myo-inositol for this population.
- Early trial termination due to increased mortality limits definitive conclusions, warranting caution.
Importance:
Previous studies of myo-inositol in preterm infants with respiratory distress found reduced severity of retinopathy of prematurity (ROP) and less frequent ROP, death, and intraventricular hemorrhage. However, no large trials have tested its efficacy or safety.
Objective:
To test the adverse events and efficacy of myo-inositol to reduce type 1 ROP among infants younger than 28 weeks' gestational age.
Design, Setting, And Participants:
Randomized clinical trial included 638 infants younger than 28 weeks' gestational age enrolled from 18 neonatal intensive care centers throughout the United States from April 17, 2014, to September 4, 2015; final date of follow-up was February 12, 2016. The planned enrollment of 1760 participants would permit detection of an absolute reduction in death or type 1 ROP of 7% with 90% power. The trial was terminated early due to a statistically significantly higher mortality rate in the myo-inositol group.
Interventions:
A 40-mg/kg dose of myo-inositol was given every 12 hours (initially intravenously, then enterally when feeding; n = 317) or placebo (n = 321) for up to 10 weeks.
Main Outcomes And Measures:
Type 1 ROP or death before determination of ROP outcome was designated as unfavorable. The designated favorable outcome was survival without type 1 ROP.
Results:
Among 638 infants (mean, 26 weeks' gestational age; 50% male), 632 (99%) received the trial drug or placebo and 589 (92%) had a study outcome. Death or type 1 ROP occurred more often in the myo-inositol group 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). All-cause death before 55 weeks' postmenstrual age occurred in 18% of the myo-inositol group and in 11% of the placebo group (adjusted risk difference, 6% [95% CI, 0%-11%]; adjusted relative risk, 1.66 [95% CI, 1.14-2.43], P = .007). The most common serious adverse events up to 7 days of receiving the ending dose 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%).
Conclusions And Relevance:
Among premature infants younger than 28 weeks' gestational age, treatment with myo-inositol for up to 10 weeks did not reduce the risk of type 1 ROP or death vs placebo. These findings do not support the use of myo-inositol among premature infants; however, the early termination of the trial limits definitive conclusions.
