Current and Emerging Treatment

While the majority of cases of retinopathy of prematurity (ROP) improve without treatment, the presence of plus disease, Type 1 ROP or aggressive ROP (A-ROP) requires transition from observation to treatment for prevention of vision loss.^1-3 Intervention should commence within 72 hours of detection of treatable disease to reduce the risk for retinal detachment.³ Treatment is most often required between 34-38 weeks of gestation.⁴

The treatment of ROP is a collaboration among ophthalmologists, pediatricians, neonatologists, and other health professionals.^1,3 Potential modalities include laser therapy, cryotherapy, surgery, or the use of intravitreal anti-vascular endothelial growth factor (anti-VEGF) agents.^1,5 Other supportive treatments may be needed, including the use of eyeglasses, the use of an eye patch, or future eye surgeries.¹

Cryotherapy and laser photocoagulation

Treatments aimed at ablation of the avascular, peripheral retina include cryotherapy as well as argon and diode laser photocoagulation.^1,2,5 Post-treatment followup is recommended within 3-7 days for assessment of regression and reactivation.⁵ Although cryotherapy was the first treatment for ROP, laser is preferred due to portability and better tolerability.⁵Treatment with either modality can prevent or reverse abnormal vessel proliferation, but may result in in loss of some peripheral vision.¹

Intravitreal anti-VEGF

An emerging treatment to suppress abnormal vessel growth, which can be used as monotherapy or in addition to laser treatment.^4,8 Post-injection follow-up includes monitoring for endophthalmitis and reexamination ≤7 days to ensure reduction in ROP features.⁵ Studies have shown promise for treatment of zone I, stage 3 ROP with plus disease, as well as type 1 ROP; bevacizumab and ranibizumab, and aflibercept have all demonstrated efficacy in treating ROP.^5,9-13 In fact, aflibercept received US Food and Drug Administration (FDA) approval in early 2023 (FIREFLEYE and BUTTERFLEYE studies), making it the first anti-VEGF therapy to receive an approved indication for the pharmacologic treatment of premature infants with ROP.¹⁴ Results of various studies are encouraging, with continued research underway to determine long-term safety, optimal dosing and rates of ROP recurrence.⁸

Vitrectomy and scleral buckle

Later stages of ROP with subsequent retinal detachment may require support with scleral buckling or repair via vitrectomy.¹ Scleral buckles may need to be divided within months to years to prevent myopia with continued growth of the eye.¹⁵ In newborns, treatment of a serious retinal detachment may not have much benefit in restoring vision.¹

Outcomes of laser or intravitreal injection treatment for ROP is usually favorable with disappearance of abnormal vasculature and resolution of plus disease.⁸ However, despite accurate diagnosis and timely treatment, ROP may sometimes continue to worsen and progress to retinal detachment, with subsequent poor visual prognosis.⁸ Among infants who weigh less than 750g at birth (micropreemie), 16% to 20% will require treatment and approximately 5% will be blind despite best intervention.^1,18

Pathophysiology

Diagnosis and Associated Risk Factors

Screening and Symptoms in ROP