We conducted two concurrent, prospective, randomized, double-blind, multicenter, dose-ranging, controlled clinical trials using broad entry criteria. Intravitreous injection into one eye per patient of
pegaptanib (at a dose of 0.3 mg, 1.0 mg, or 3.0 mg) or
sham injections were administered every 6 weeks over a period of 48 weeks. The primary end point was the proportion of patients who had lost fewer than 15 letters of visual acuity at 54 weeks.
RESULTS: In the combined analysis of the primary end point (for a total of 1186 patients), efficacy was demonstrated, without a dose-response relationship, for all three doses of
pegaptanib (P<0.001 for the comparison of 0.3 mg with
sham injection; P<0.001 for the comparison of 1.0 mg with
sham injection; and P=0.03 for the comparison of 3.0 mg with
sham injection). In the group given
pegaptanib at 0.3 mg, 70 percent of patients lost fewer than 15 letters of visual acuity, as compared with 55 percent among the controls (P<0.001). The risk of severe loss of visual acuity (loss of 30 letters or more) was reduced from 22 percent in the
sham-injection group to 10 percent in the group receiving 0.3 mg of
pegaptanib (P<0.001). More patients receiving
pegaptanib (0.3 mg), as compared with
sham injection, maintained their visual acuity or gained acuity (33 percent vs. 23 percent; P=0.003). As early as six weeks after beginning
therapy with the study
drug, and at all subsequent points, the mean visual acuity among patients receiving 0.3 mg of
pegaptanib was better than in those receiving
sham injections (P<0.002). Among the adverse events that occurred,
endophthalmitis (in 1.3 percent of patients), traumatic injury to the lens (in 0.7 percent), and
retinal detachment (in 0.6 percent) were the most serious and required vigilance. These events were associated with a severe loss of visual acuity in 0.1 percent of patients.
CONCLUSIONS: