Anti-VEGF Drug Effective for Treating ROP; Safety Issues Unresolved
Major Finding: Intravitreal bevacizumab monotherapy showed a significant benefit vs. conventional laser therapy for treating infants with stage 3+ retinopathy of prematurity, but only for zone I disease. However the safety of the treatment and the appropriate dosage require further study; five infants died with monotherapy compared with two who had conventional treatment.
Data Source: BEAT-ROP, a prospective, randomized, stratified, controlled multicenter clinical trial of 150 infants with stage 3+ ROP.
Disclosures: The study was supported by grants from Research to Prevent Blindness, the National Eye Institute, and the Hermann Eye Fund as well as research funds from the Alfred W. Lasher III Professorship and a grant from the center for clinical and translational research at the University of Texas Health at Houston. Helen A. Mintz-Hittner, the corresponding author, receives honoraria from the Bascom Palmer Eye Institute and Clarity Medical Systems, and from testifying as an expert witness in a retinopathy of prematurity suit.
FROM NEW ENGLAND JOURNAL OF MEDICINE
Also, intravitreal bevacizumab reaches the systemic circulation, which raises concern about untoward effects on an infant’s developing organs. Although there are no documented instances of this, a much larger sample size than that of this study is necessary to determine the statistical safety of the drug in premature infants. The dose of intravitreal bevacizumab is a fraction of the dose used for cancer treatment, and the amount of circulating bevacizumab is very small. "Bearing in mind the pharmacokinetics of bevacizumab, the extensive experience of adults taking this drug, and [the results of this study], it seems reasonable to assume that intravitreal bevacizumab is safe. However, continued vigilance will be important as use of the drug continues," Dr. Reynolds wrote.
"As our experience with bevacizumab grows, its indications and relative contraindications will be refined. In the meantime, intravitreal bevacizumab should become the treatment of choice in premature infants," Dr. Reynolds concluded.
The BEAT-ROP study was supported by grants from Research to Prevent Blindness, the U.S. National Eye Institute, and the Hermann Eye Fund as well as research funds from the Alfred W. Lasher III Professorship and a grant from the center for clinical and translational science at the University of Texas Health Science Center in Houston. Helen A. Mintz-Hittner, the corresponding author, receives honoraria from the Bascom Palmer Eye Institute and Clarity Medical Systems, as well as from testifying as an expert witness in a retinopathy of prematurity suit.
Dr. Reynolds received payment as an expert witness in trials concerning treatment of ROP from various legal firms and royalties for an up-to-date glaucoma chapter.
