The DROPROP trial found fewer preterm infants progressed to treatment-requiring retinopathy of prematurity with dexamethasone eye drops, but the result was not statistically significant.
Written by: Mayuresh Salvi, PharmD
Reviewed by: Pharmacally Editorial Team
A New Finding for a Long-Established Medicine
Dexamethasone is one of the most widely used corticosteroids in modern medicine, with established roles across a broad range of inflammatory and supportive-care settings. Its clinical history, however, does not mean that its therapeutic potential is fully understood.
New findings from the DROPROP randomized clinical trial suggest another possible application for the long-established drug: preventing progression of retinopathy of prematurity (ROP) in extremely preterm infants.
The study, published online August 3, 2026, in JAMA Pediatrics, evaluated whether dexamethasone eye drops could reduce progression from prethreshold ROP to type 1 ROP requiring invasive treatment.
The findings were encouraging but did not establish a statistically significant treatment benefit.
DROPROP Evaluated Topical Dexamethasone in Preterm Infants
ROP is a potentially blinding complication of prematurity caused by abnormal development of the retinal blood vessels. Severe disease can require interventions such as laser photocoagulation or intravitreal anti-VEGF therapy. The invasive nature of these treatments has created interest in approaches that could slow disease progression before treatment becomes necessary.
The double-masked DROPROP trial was conducted across six university hospitals and eight county hospitals in Sweden between September 2022 and October 2025. It included 100 infants born before 30 weeks of gestational age who had prethreshold ROP.
Infants received either dexamethasone eye drops at a concentration of 1 mg/mL or preservative-free saline placebo. The dosing schedule depended on disease stage, with treatment given every other day for stage 1 or 2 ROP and daily for stage 3 ROP, for up to 12 weeks.
The primary question was straightforward: could topical dexamethasone prevent prethreshold ROP from progressing to type 1 disease requiring invasive treatment?
Fewer Infants Progressed to Treatment-Requiring ROP
Among the 100 infants, treatment-requiring type 1 ROP developed in 10 of 50 infants receiving dexamethasone, compared with 19 of 50 receiving placebo.
In other words, 20% of infants in the dexamethasone group progressed to treatment-requiring disease compared with 38% in the placebo group. The investigators described this as a 47% numerical relative reduction in risk.
However, the difference did not meet the study’s prespecified threshold for statistical significance. The adjusted analysis produced an odds ratio of 0.44, with a 95% confidence interval of 0.17 to 1.12 and a P value of .08. Because the confidence interval included 1 and the P value was above .05, the trial did not confirm a statistically significant efficacy benefit.
This distinction is important. The study provides a clinically interesting signal, but it does not establish dexamethasone eye drops as a proven preventive treatment for ROP.
Safety Findings Were Reassuring During the Trial
Safety was an important part of the study because topical corticosteroids can potentially be absorbed systemically, particularly in premature infants.
The investigators found no clinically significant difference in overall adverse events between the dexamethasone and placebo groups. They also found no clinically significant difference in intraocular pressure during the study. Overall adverse events occurred in 70.6% of infants in the dexamethasone group and 73.5% in the placebo group.
The study did identify some findings requiring continued observation. Salivary cortisol concentrations decreased during dexamethasone treatment but returned toward baseline after treatment stopped. The investigators did not observe clinical evidence of adrenal suppression during the trial.
Long-term follow-up is planned to assess ocular and visual outcomes and potential late adverse effects, making continued safety evaluation particularly important in this vulnerable population.
Why the Findings Matter for Dexamethasone
The DROPROP study highlights how established medicines can continue to generate new therapeutic possibilities decades after their introduction. Dexamethasone has long been used across medicine, but earlier experimental and clinical observations suggested that its anti-inflammatory and other biological effects might also influence ROP progression.
The findings therefore add another example of how an existing drug can be evaluated in a new disease setting, population, or treatment approach.
The Study Does Not Yet Change ROP Treatment
Although fewer infants progressed to treatment-requiring ROP with dexamethasone, the primary analysis did not reach statistical significance. The investigators also noted that the observed treatment effect and placebo event rate differed from those anticipated during study planning, potentially limiting statistical power. Larger randomized trials are needed before routine clinical implementation or guideline adoption.
Dexamethasone’s Potential Beyond Established Uses
DROPROP does not establish dexamethasone eye drops as a proven preventive treatment for ROP. Instead, it provides a clinically meaningful signal that warrants further investigation.
For dexamethasone, the study illustrates an important principle in drug development: the clinical potential of a medicine can continue to evolve long after its initial introduction. A larger evidence base will determine whether this long-established drug can eventually offer a noninvasive way to reduce the need for invasive ROP treatment.
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About the Writer
Mayuresh Sunil Salvi (Linkedin) is a PharmD professional and healthcare writer with a strong interest in pharmacovigilance, drug safety, and emerging medical research. He is passionate about exploring new drug discoveries, clinical research, and advances in evidence-based medicine. His interests also include ward rounds, prescription audits, and treatment analysis to support rational pharmacotherapy and improved patient care.