Clinical trial shows dexamethasone reduces treatment need for premature infants with ROP — Evidence Review

A new randomized trial suggests that dexamethasone eye drops may reduce the risk of severe retinopathy of prematurity (ROP) in extremely premature infants. Most related studies support the general safety and efficacy of dexamethasone in neonatal populations, though concerns about long-term outcomes remain; see the full study at the original source.

• The new trial’s finding of reduced need for invasive ROP treatment aligns with prior evidence showing dexamethasone can positively impact certain neonatal outcomes, but previous studies have also identified potential risks, including neuromotor impairments with systemic use, especially in male infants 1 2 4.
• Large-scale research on antenatal and postnatal dexamethasone suggests beneficial effects on neonatal morbidity and survival, but highlights the importance of dosage, timing, and route of administration in determining safety and efficacy 3 5.
• Existing literature on ROP emphasizes the need for less invasive, more accessible treatments, particularly in low-resource settings, and the potential for simple interventions like eye drops to address global disparities in ROP care 13 14.

Study Overview and Key Findings

Retinopathy of prematurity remains a leading cause of childhood blindness worldwide, particularly affecting infants born extremely preterm. Current treatments, such as laser therapy and intraocular injections, are invasive and carry risks for further visual impairment. The new multicenter DROPROP trial in Sweden is the first randomized controlled study to evaluate whether topical dexamethasone eye drops can slow disease progression and reduce the need for invasive ROP interventions. The study's significance lies in its focus on a simple, low-cost, and non-invasive intervention—one that could have broad global applicability, especially in regions where access to advanced ophthalmologic care is limited.

Property	Value
Study Year	2026
Organization	University of Gothenburg, Sahlgrenska University Hospital
Journal Name	JAMA Pediatrics
Authors	Ann Hellström, Mariya Petrishka-Lozenska, Ulrika Sjöbom, Jenny Wallander, Anders K. Nilsson, Chatarina Löfqvist, David Ley, Lotta Gränse, Hanna Maria Öhnell, Anna-Lena Hård, Gunnar Jakobsson, Karin Sävman, Magnus Domellöf, Stefan Löfgren, Eva Larsson, Lois E.H. Smith, Aldina Pivodic, Pia Lundgren, DROPROP Collaboration Group, Liv Vallin, Ingrid Hansen-Pupp, Kristina Teär Fahnehjelm, Eva Albinsson, Erik Normann, Afsaneh Alibakhshi, Nikica Tomašić, Fredrik Ingemansson, Pierfrancesco Mirabelli, Per Odelberg, Despina Tsamadou, Evangelos Tsigkoulis, Johanna Kopparström, Sofie Eriksson, Karin Hochard Sandgren, Aikaterni Massou, Ylva Friberg-Riad, Athanasia Skriapa-Manta, Liv Lind, Ulrika Lidén, Linnéa Holmberg
Population	Infants born extremely prematurely with ROP
Sample Size	n=100
Methods	Randomized Controlled Trial (RCT)
Outcome	Progression of ROP to treatment stage
Results	Dexamethasone reduced treatment need by 47% relative risk

Literature Review: Related Studies

To contextualize these findings, we searched the Consensus paper database, which contains over 200 million research papers. The following search queries were used to identify relevant studies:

1. dexamethasone premature babies blindness
2. treatment efficacy dexamethasone outcomes
3. relative risk blindness prevention infants

Topic	Key Findings
What are the long-term and short-term risks of dexamethasone in preterm infants?	- Early systemic dexamethasone reduces chronic lung disease but may increase neuromotor dysfunction and growth impairment in male infants 1 2 4. - Some long-term follow-up studies suggest no significant increase in combined death or major neurodevelopmental impairment 4.
How effective is dexamethasone for neonatal and perinatal outcomes?	- Antenatal dexamethasone reduces neonatal death and serious morbidity in low-resource settings without increasing maternal infection risk 3 5. - Both dexamethasone and betamethasone are effective for improving survival without neurosensory disability at age 2 years 5.
What are the current challenges and strategies in ROP prevention and management?	- Prediction models and registry studies help target ROP screening and identify infants at greatest risk, emphasizing early intervention 11 12 13. - There is increasing focus on less invasive and more accessible treatments, as well as on education and guideline adaptation 13 14.
What is the global and clinical context of ROP and corticosteroid use?	- ROP remains a major cause of preventable blindness, but incidence and treatment needs are changing with evolving screening criteria and treatment preferences 13. - Simple, scalable interventions are needed in low-resource settings to address disparities in outcomes 3 13 14.

What are the long-term and short-term risks of dexamethasone in preterm infants?

Research on systemic dexamethasone in preterm infants demonstrates efficacy in reducing chronic lung disease but also potential for adverse neuromotor and growth outcomes, particularly in male infants. While some studies indicate increased risk of cerebral palsy and abnormal neurological findings with prolonged or early systemic dexamethasone, others suggest the overall risk of death or major neurodevelopmental impairment may not be significantly increased over the long term. The new Swedish study differs in using topical eye drops rather than systemic administration, potentially mitigating systemic side effects.

• Systemic dexamethasone (especially early or prolonged courses) may increase neuromotor dysfunction and growth impairment in male infants 1 2.
• Long-term follow-up shows that overall risk of death or major impairment may not be significantly higher, but risk of cerebral palsy remains elevated 4.
• The mode and timing of dexamethasone administration are critical factors affecting safety and efficacy 1 2.
• The new study’s topical approach may offer a safer profile, though long-term follow-up is needed to confirm this.

How effective is dexamethasone for neonatal and perinatal outcomes?

Dexamethasone, both antenatally and postnatally, has been shown to reduce neonatal mortality and morbidity, particularly in low-resource settings. Studies comparing dexamethasone with other corticosteroids demonstrate similar efficacy for improving survival without neurosensory disability. These findings support the rationale for investigating dexamethasone in other neonatal complications, such as ROP, and for pursuing accessible interventions.

• Antenatal dexamethasone lowers risks of neonatal death and severe morbidity without increasing maternal infection 3.
• Both dexamethasone and betamethasone are effective for improving major neonatal outcomes, with no significant difference in survival without disability at age 2 5.
• The safety and benefit profile depends on context, population, and administration method 3 5.
• The new trial’s results add to growing evidence of dexamethasone’s potential for improving outcomes in high-risk neonates.

What are the current challenges and strategies in ROP prevention and management?

ROP management is evolving, with growing emphasis on individualized risk prediction, targeted screening, and less invasive treatments. Studies highlight the value of prediction models, registry data, and guideline revisions for optimizing screening and intervention strategies, as well as the importance of education to improve caregiver engagement. The Swedish trial’s focus on non-invasive eye drops aligns with these broader efforts to enhance ROP prevention and care.

• Risk prediction models (e.g., DIGIROP-Birth) optimize screening by identifying high-risk infants based on birth characteristics 11.
• Registry data show that changes in screening criteria can reduce unnecessary procedures without increasing severe ROP cases 13.
• There is a shift toward less invasive and more accessible treatments, a theme echoed in the new dexamethasone eye drop study 13 14.
• Caregiver education and guideline adaptation are crucial for improving ROP outcomes 14.

What is the global and clinical context of ROP and corticosteroid use?

ROP remains a significant cause of preventable childhood blindness, with global disparities in incidence and treatment access. Recent studies suggest that simple, scalable interventions are critical, especially in resource-limited settings. The new Swedish trial’s focus on eye drops represents a promising step toward accessible ROP prevention, consistent with calls for practical strategies to reduce the burden of blindness worldwide.

• ROP incidence and treatment needs are shifting due to evolving guidelines and practices 13.
• Infants with higher birth weight have a low risk for severe ROP, supporting more targeted screening 13.
• Low-resource settings require interventions that are simple, inexpensive, and easy to administer 3 13 14.
• The new study directly addresses the need for accessible treatments, which could have broad public health impact.

Future Research Questions

While the new study offers promising evidence for dexamethasone eye drops as a non-invasive ROP intervention, several important research questions remain. Further investigation is needed to clarify long-term safety, generalizability, and optimal protocols for this approach, especially considering the historical risks associated with systemic corticosteroid use in neonates.

Research Question	Relevance
What are the long-term visual and neurodevelopmental outcomes of dexamethasone eye drop therapy for ROP?	Long-term follow-up is essential to determine if topical dexamethasone avoids the neurodevelopmental risks seen with systemic administration and to assess sustained benefits or late-onset complications 1 2 4.
Does dexamethasone eye drop efficacy vary with gestational age or other risk factors for ROP?	Variations in ROP risk and progression may influence treatment effectiveness; understanding these factors can help tailor interventions for the highest-risk groups 11 12 13.
How does dexamethasone eye drop treatment compare directly with laser therapy or anti-VEGF agents for ROP?	Direct comparative trials are needed to assess whether dexamethasone eye drops can achieve similar or superior outcomes to current standard-of-care treatments in terms of efficacy, safety, and cost 13.
What are the optimal timing, dose, and duration of dexamethasone eye drop therapy for ROP prevention?	Determining the best regimen is critical for maximizing benefit and minimizing risk, given past findings that suggest timing and dosage are key determinants of safety and efficacy in corticosteroid therapy 1 2 3.
Can dexamethasone eye drops be safely and effectively implemented in low-resource settings to reduce ROP-related blindness?	Given the global burden of ROP and disparities in care, implementation studies are needed to evaluate the feasibility, safety, and impact of this approach in diverse clinical and resource environments 3 13 14.