On Dec. 17, 2004, the FDA approved pegaptanib sodium (Macugen, Eyetech Pharmaceuticals) for the treatment of choroidal neovascularization (CNV) associated with age-related macular degeneration (AMD).

Retinal specialists who have only several months of real-world experience using this first available anti-VEGF agent in clinical practice note it is too early to comment on efficacy in their personal series of patients. However, they agree that with its broad-based indication, pegaptanib sodium has been a welcome addition for allowing them to offer more patients with exudative AMD some treatment for their sight-threatening condition.

"Pegaptanib sodium is not a panacea, but it has been a real benefit in my practice in terms of being able to offer treatment to patients with large subfoveal lesions that are 100% occult, minimally classic, or even predominantly classic if the patient is not a candidate for verteporfin photodynamic therapy based on lesion size," said Mithlesh C. Sharma, MD, clinical assistant professor of ophthalmology, Northeastern University of Ohio College of Medicine, Rootstown. "As we move forward and accumulate results from treating more patients followed for longer periods, we will be able to make better judgments about the results and how pegaptanib sodium fits into the management algorithm."

While he too stresses that it would be premature to make any outcome judgments, Dr. Blinder has not encountered any safety problems and has been satisfied with the results to date.

"As with any treatment, some patients fare better than others," Dr. Blinder said. "On the positive side, however, the majority have at least maintained their vision postinjection and a small minority even showed significant improvement."

Dr. Sharma began using pegaptanib sodium when Medicare reimbursement became available. Over a period of about 2 months, he has treated about 25 patients. Encouragingly, a few of his patients have also demonstrated early vision improvement of at least 2 lines.

"I was surprised by that benefit, which developed within just about a week after injection, but I have my fingers crossed that these patients will continue to do well as I follow them over the long term," Dr. Sharma said.

He added that in addition to patients with AMD-related subfoveal CNV, he is using pegaptanib sodium in eyes with serous pigment epithelial detachment when there is an associated CNV membrane.

"These patients were not studied in the premarketing trials," Dr. Sharma said. "However, there is no other good treatment for this pathology, and so off-label use of pegaptanib sodium seems justifiable based on its favorable safety record in the premarketing trials."

Patient selection Since pegaptanib sodium can be used regardless of the lesion's angiographic subtype, the decision about who is a potential candidate for this treatment is simplified relative to choosing patients for verteporfin PDT. Nevertheless, embarking on pegaptanib sodium requires a strong commitment on the part of the physician and the patient.

"It is important to spend time counseling patients thoroughly about their options and the associated potential benefits and risks," Dr. Blinder said. "Importantly, they must understand that if they choose to receive pegaptanib sodium, there is a need for frequent follow-up visits and reinjection every 6 weeks.