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We report the compassionate use of intravenous cidofovir for the treatment of cytomegalovirus retinitis in 51 patients with AIDS who were receiving highly active antiretroviral therapy (HAART). After a median of 9 doses, 49 patients showed no retinitis activity. However, treatment was stopped in 17 patients because of adverse reactions and in 5 patients for other reasons. Two deaths were considered related to the drug. Iritis developed in 21 patients (41.2%), a frequency higher than that reported during the pre-HAART era. Patients with iritis had median CD4 cell counts—both at nadir and at the initiation of cidofovir therapy—approximately 3 times higher than those for patients without iritis (P=.003 andP=.05, respectively). Our study confirms the efficacy of cidofovir therapy for AIDS-associated cytomegalovirus retinitis. Our data suggest that the risk of iritis may be higher for patients with better immunological status, probably because of their enhanced ability to mount an inflammatory response.