TOPLINE

Topical dexamethasone eye drops were linked to a numerical reduction in the odds of progression from prethreshold retinopathy of prematurity (ROP) to treatment-requiring type 1 ROP in infants born before 30 weeks’ gestational age; this reduction was not statistically significant.

METHODOLOGY

• Researchers conducted a multicenter randomized clinical trial in Sweden from September 2022 to October 2025, enrolling 100 infants (42% girls; five twin pairs) who were born before 30 weeks’ gestational age and had prethreshold ROP, defined as stage 1 or 2 ROP without plus disease in zone I or stage 2 or 3 ROP without plus disease in posterior zone II.
• Infants were randomly assigned to receive dexamethasone eye drops (1 mg/mL) or placebo (preservative-free saline), with one drop per eye administered every other day for stage 1 or 2 ROP and daily for stage 3 ROP; treatment was continued for up to 12 weeks.
• The primary outcome was progression to type 1 ROP requiring invasive treatment.
• Secondary outcomes were time to type 1 ROP, time to type 1 ROP among infants without regression, and ROP recurrence after laser or anti-VEGF therapy.
• Adverse events were monitored as safety outcomes, comprising elevated intraocular pressure, systemic growth, changes in plasma glucose levels, and pulmonary, cardiovascular, infectious, gastrointestinal, and neurologic complications.

TAKEAWAY

• Overall, type 1 ROP occurred in 20% of infants in the dexamethasone group vs 38% in the placebo group (adjusted odds ratio [aOR], 0.44; P = .08). 
• Subgroup analyses revealed numerically greater effects of dexamethasone in infants with a birth weight > 660 g (OR, 0.23; 95% CI, 0.06-0.94) and in those receiving parenteral nutrition for less than 14 days (OR, 0.04; 95% CI, 0.00-0.84). 
• ROP recurred in four infants in the dexamethasone group vs five in the placebo group.
• No clinically significant differences were observed between the groups for intraocular pressure, with 25.5% of infants in the dexamethasone group and 20.4% of those in the placebo group having at least one intraocular pressure measurement > 20 mm Hg.

IN PRACTICE

“Although not statistically significant, the findings from this RCT [randomized controlled trial] suggest a potential benefit of topical dexamethasone in preventing the need for invasive ROP treatment,” the authors of the study wrote. “[T]he study highlights the potential to reduce the risk of visual impairment in preterm infants, with meaningful implications for long-term quality of life,” they added.

“Dexamethasone eye drops appear to work. Existing treatments for ROP remain effective and available should progression occur. Certainly, additional studies may help refine timing, duration, and dosing, and ongoing monitoring for any new safety concerns is paramount,” an expert wrote in an accompanying editorial.

SOURCE

This study was led by Ann Hellström, MD, PhD, of the Institute of Neuroscience and Physiology in the Sahlgrenska Academy at the University of Gothenburg, Gothenburg, Sweden. The editorial was written by Gil Binenbaum, MD, MSCE, Children’s Hospital of Philadelphia, Philadelphia. The study and editorial were published online on August 3, 2026, in JAMA Pediatrics.

LIMITATIONS

The expected treatment effect of dexamethasone was overestimated, likely due to the effect observed in the pilot study with a higher dose. The event rate in the placebo group was also lower than expected, which may have reduced the study’s statistical power to detect significant differences. Seven infants were treated without verifying type 1 ROP, and if included in the analysis, may have diminished the observed treatment effect.

DISCLOSURES

Grants from the Swedish Research Council, the Swedish state under the ALF agreements, the Wallenberg Clinical Scholars, the National Institutes of Health, and Skåne University Hospital supported this work. The product Dexafree was provided by Théa Nordic AB at no cost. One author disclosed that her sister serves as CEO of ADDI Medical AB, though she holds no financial interest in the company. Another author disclosed receiving grants from the Swedish state under the ALF agreement during the conduct of the study. 

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.