Objective:

To evaluate the effect of topical dexamethasone on the progression to treatment-requiring retinopathy of prematurity (ROP) in extremely preterm infants.

Approach:

• Study Design: A double-masked randomized clinical trial (DROPROP) enrolling 100 infants born before 30 weeks' gestational age across six university hospitals and eight county hospitals in Sweden from 2022 to 2025.
• Intervention: Infants with prethreshold ROP were assigned to receive either topical dexamethasone (1 mg/mL) or placebo for up to 12 weeks.
• Primary Outcome: Progression to type 1 ROP requiring invasive treatment, analyzed by intention-to-treat and adjusted for gestational age and study site.
• Secondary Outcomes: Included time to treatment-requiring disease, recurrence following treatment, and safety outcomes.

Key Findings:

• 20% of infants receiving dexamethasone developed treatment-requiring type 1 ROP compared to 38% in the placebo group, indicating a 47% relative risk reduction, although the difference did not reach statistical significance.
• No significant differences were observed in time to progression to type 1 ROP or recurrence following treatment between groups.
• Adverse event rates were similar between dexamethasone and placebo groups, with no clinically significant difference in intraocular pressure.

Interpretation:

The trial did not meet its primary endpoint, and the findings were not statistically significant.

Limitations:

• The trial may have been underpowered due to lower than anticipated treatment effect and placebo event rate.
• Seven infants received invasive treatment despite not meeting criteria for type 1 ROP, which may have influenced treatment estimates.

Conclusion:

The findings suggest the need for further investigation to confirm results and identify potential subgroups of infants who may benefit from dexamethasone.

Sources:

• JAMA Pediatrics