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New Approach to Retinoblastoma Saves Eyes and Vision
Zosia Chustecka
March 25, 2008 — A new approach to the treatment of retinoblastoma, which involves the administration of chemotherapy directly into the ophthalmic artery, can save children from having an eye removed, and in some cases can save vision. The technique is very new — results from the first patients are just being reported — but it is already being hailed as an advance in the treatment of retinoblastoma by those involved.
Results from 18 patients were reported last week at the Society of Interventional Radiology 33rd Annual Scientific Meeting, in Washington, DC, and results from the first 10 of these patients were published online March 14 in Ophthalmology. At present, the new technique is being carried out only at the New York-Presbyterian Hospital/Weill Cornell Medical Center and Memorial Sloan Kettering Cancer Center, in New York, where it was developed.
Retinoblastoma is a common pediatric cancer, usually diagnosed in children younger than 3 years old, Pierre Gobin, MD, from the Weill Cornell Medical Center, explained at a press conference during the meeting. The presenting sign is usually leukocoria (white papillary reflection), but by the time this is spotted, the tumor has already filled the eye, he said. Treatment options include cryotherapy, photocoagulation, radiation, and intravenous chemotherapy, but most children present with such advanced disease that often the only option left is to remove the eye (enucleation), which is life-saving.
The new technique involves delivering the chemotherapeutic drug melphalan (Alkeran, GlaxoSmithKline) through a catheter inserted at the groin, which travels up to the carotid artery and then on to the ophthalmic artery before reaching the retina. The procedure is carried out under general anesthetic but on an outpatient basis, he explained. The new technique is a "tremendous advance in treating retinoblastoma," he said. "It allows many children to keep their eyes and, in some cases, restores vision."
Coauthor David Abramson, MD, from New York-Presbyterian Hospital/Weill Cornell Medical Center and Memorial Sloan Kettering Cancer Center, told Medscape Oncology that "everyone in the retinoblastoma community" agrees that the new technique offers an advance in treatment. "It will not only save many eyes that are currently enucleated, but it will also allow us to deliver more chemotherapy to the eye and eliminate the need for systemic chemotherapy for intraocular disease." Retinoblastoma is an unusual case in pediatric cancer, because the disease is limited to the eye; other cancers have often already metastasized, so there is a need for systemic chemotherapy, which is toxic.
At the meeting, Dr. Gobin reported results from 18 patients; another 2 are still completing the treatment. There were 2 treatment failures, but 16 patients had their tumor "cured" and 14 kept their eye (75%). In 2 cases, the eye was removed because it was already badly damaged, but pathology showed that there was no tumor remaining. Of the 14 eyes that were saved, 9 had vision (50%), and in 4 of these cases the vision improved. The other 5 eyes had no vision, but even this is a good result; "it is better to have an eye that is blind but your own than to have a prosthesis," he commented.
The small size of the blood vessels in very young children can present a problem. In 2 infants (of 20 in total), Dr. Gobin said he was unable to reach the ophthalmic artery. In 4 cases, there was some retinopathy and in 2 cases some transient skin discoloration, both caused by too high a dose of the drug in the first few instances, he said. Otherwise, there were no complications, and in particular no adverse effects, such as infection or neuropenia, which are seen when the same chemotherapy is administered intravenously.
"We are hopeful that this interventional treatment will prevent removal of the eye in advanced tumors and even replace conventional (whole-body) chemotherapy and radiation in less advanced forms of retinoblastoma," Dr. Gobin commented in statement. "Our work comes from a collaboration among an interventional radiologist, an ophthalmologist specialist in cancer, and a pediatric oncologist. Modern medicine is so complex that new treatments are often discovered by physicians working in a team."
Society of Interventional Radiology (SIR) 33rd Annual Scientific Meeting:
Abstract 60. Presented March 17, 2008
Ophthalmology. Published online March 14, 2008. Abstract
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Zosia Chustecka is news editor for Medscape Hematology-Oncology and prior
news editor of jointandbone.org, a website acquired by WebMD. A veteran medical journalist based in London, UK, she has won a prize from the British Medical Journalists Association and is a pharmacology graduate. She has written for a wide variety of publications aimed at the medical and related health professions. She can be contacted at zchustecka@medscape.net.
Medscape Medical News 2008. © 2008 Medscape
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