A safety review of drugs used for the treatment of retinopathy of prematurity

ABSTRACT

Introduction

Retinopathy of Prematurity (ROP) is a sight-threatening disease representing one of the main disabling diseases affecting premature newborns. Presently, ROP is treated by surgical interventions and drug therapies are limited to the off-label use of a little amount of molecules approved for other pathologies.

Areas covered

Many drugs that may potentially be used in treating ROP are recently proposed, in many cases after the demonstration of their effectiveness in preclinical studies. In this review, the authors discuss safety and effectiveness of the main proposed approaches in the pharmacologic treatment of the disease, including approaches based on oxygen therapy and nutritional interventions.

Expert opinion

Surgical approaches to ROP are not without side effects. However, most of the proposed pharmacologic interventions can also raise specific concerns. In particular, these approaches follow a curative paradigm and are proposed in patients once the disease has progressed, with an effectiveness that is often smaller than expected. A goal in the treatment of ROP would be moving the paradigm toward a preventive approach that could be potentially effective in treating extremely low birth weight preterm infants before ROP becomes manifest.

KEYWORDS:

• Antioxidants
• anti-VEGF
• hyperoxia/hypoxia
• IGF-1
• NSAIDs
• propranolol
• steroids
• VEGF

Article highlights

• ROP is a preventable neovascular retinal disease with a great impact on vision. Surgical therapies are the approved interventions to treat this disease.

• Surgical interventions show several adverse side effects that underlie the urgent need for pharmacologic therapies to treat ROP.

• Several drugs that may potentially be used in treating ROP have been proposed based on studies in preclinical models.

• Among pharmacologic interventions, anti-VEGF drugs are used off-label to treat newborns suffering from severe ROP. However, also anti-VEGF drugs raise several concerns. For instance, the neuroprotective role exerted by VEGF may be abrogated by sequestering VEGF.

• Propranolol may represent a valid approach to treat ROP although its safe profile needs to be better evaluated in appropriate clinical trials.

• Presently, pharmacologic approaches to ROP are mainly intended to treat ROP after the disease has progressed. A new goal would be the introduction of preventive approaches effective in treating extremely low birth weight preterm infants before ROP becomes manifest.

This box summarizes key points contained in the article.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Additional information

Funding

This paper was funded by the University of Pisa.