The age-related macular degeneration radiotherapy trial (AMDRT): One year results from a pilot study

PURPOSE: To assess the short-term safety and efficacy of treating subfoveal choroidal neovascularization (CNV) with external beam radiation delivered in 5 x 4 Gy fractions among patients having age-related macular degeneration (AMD). DESIGN: A multicenter prospective randomized controlled pilot study. METHODS: Eighty-eight patients were enrolled through 10 sites and were randomized to radiotherapy (20 Gy delivered in 5 daily fractions of 4 Gy each; 6 MV [N = 411) or no radiotherapy (sham radiotherapy EN = 22] or observation [N = 25]). Eligibility criteria included visual acuity of at least 20/320 and subfoveal CNV not ame- nable to treatment. Randomization was stratified by lesion type (new or recurrent CNV) and blood (<50% or greater than or equal to50% of the lesion [N = 13]). The primary outcome measure was loss of greater than or equal to3 lines of visual acuity. Secondary outcome measures were angiographic response and side effects. RESULTS: At baseline, patient and ocular characteristics were similar between treatment groups. At six months, 9 radiated eyes (26%) and 17 eyes not radiated (49%) lost greater than or equal to3 lines of visual acuity (P =.04; stratified X 2 test). At 12 months, 13 radiated eyes (42%) and 9 observed eyes (49%) lost greater than or equal to3 visual acuity lines (P =.60). The radiated group demonstrated smaller lesions and less fibrosis than the nonradiated group (P =.05 and .004, respectively) at 12 months. Radiation,induced complications were not observed except for one radiated eye with numerous cotton wool spots and possible radiation retinopathy. CONCLUSIONS: External beam radiation at 5 x 4 Gy may have a modest and short,lived (six month) benefit in preserving visual acuity. (C) 2004 by Elsevier Inc. All rights reserved.