Premature babies are to be offered treatment with ranibizumab routinely as an option for some forms of retinopathy of prematurity (ROP), as an alternative to laser treatment. Use of the drug on the NHS was heralded as potentially "life-changing" in saving vision and preventing blindness in premature babies.
Ranibizumab (Lucentis, Novartis) is a humanised recombinant monoclonal antibody fragment targeted against human vascular endothelial growth factor (VEGF)-A. It is indicated in preterm infants for the treatment of ROP with zone I (stage 1+, 2+, 3 or 3+), zone II (stage 3+), or aggressive posterior ROP (AP-ROP) disease. The off-patent drug reduces or reverses retinal neovascularisation and has been found to be effective compared with laser treatment in children.
Currently, laser treatment is the standard of care for ROP. However, some premature babies are too unwell and fragile to receive laser treatment, which also carries a risk of leaving scarring on the retina and permanently damaging vision. Ranibizumab is administered by intraocular injection and does not cause scarring.
It is also used routinely in adults with wet age-related macular degeneration, as well as those with diabetic macular oedema, proliferative diabetic retinopathy, retinal vein occlusion, and choroidal neovascularisation.
'Life-Changing Option' to Save a Child's Sight
Announcing that the drug would be made available for ROP routinely on the NHS for the first time, following the publication of new national guidance, NHS chief executive Amanda Pritchard said: "The impacts of vision loss can be absolutely devastating, particularly for children and young people, so it's fantastic that this treatment will now give families across the country another life-changing option to help save their child's precious sight.
"The national roll-out of this lifeline treatment for babies who are too poorly to undergo laser therapy is a vital step forward in preventing avoidable vision loss."
According to Great Ormond Street Hospital for Children, ROP affects around 20% of premature babies, mainly those born before week 32 or weighing less than 1500 g. When birth occurs before retinal vasculature is fully developed, blood vessels continue to grow but are fragile and can leak blood, causing scarring. This can lead to retinal detachment, compromising vision and, at its most extreme, causing irreversible sight loss and blindness.
Clinical guidelines published in 2022 by the Royal College of Ophthalmologists and endorsed by the Royal College of Paediatrics and Child Health stated that "timely intervention will prevent blindness in most cases", but that late treatment imparted a much higher risk of an unfavourable outcome.
Comparisons had suggested that for Zone I and Zone II ROP, ranibizumab gave results similar to laser, with no significant difference in disease regression, vision outcomes or safety measures. Fewer eyes developed retinal detachment following anti-VEGF (1/164, 0.6%) compared with laser treatment (49/1003, 4.9%), and fewer eyes (5% compared with 20%) developed high myopia.
Drug Therapy 'Simpler' but Requires 'More Intense Follow Up'
However, while drug treatment was "simpler to administer" than laser therapy, it did require "much more intense follow-up", including early examinations for adverse effects, including endophthalmitis, and to monitor disease regression, as well as more prolonged follow-up to detect and treat disease reactivation. Anti-VEGF therapy carried a higher rate of retreatment and, in addition to the risk of both short- and long-term ophthalmic morbidities, there were "some concerns related to systemic morbidity" following anti-VEGF treatment.
NHS England (NHSE) highlighted that as ROP was "preventable", all preterm or low birth weight babies are screened for it on the NHS. Most (>90%) screened infants do not require treatment. However: "If severe ROP is diagnosed, treatment with ranibizumab could begin within 2 or 3 days," the announcement of the new drug said.
NHSE noted that up to 1 in 3 (31%) babies would need a second treatment within 4 months. Treated infants would have regular follow up in the first 6 months, followed by annual follow up to age 5, it said.