Intravitreal ganciclovir salvage therapy for cytomegalovirus retinitis in AIDS: AIDS clinical trials group protocol 085

AbstractObjective: To determine the efficacy and safety of intravitreal injection of ganciclovir for active cytomegalovirus (CMV) retinitis in AIDS patients who are intolerant of systemic ganciclovir therapy.Methods: An open-labeled trial of intravitreal ganciclovir induction and maintenance therapy was conducted in outpatient clinics of five AIDS Clinical Trials Units. Sixteen eyes of 11 patients were treated; 11 eyes of eight patients were included in the analysis. Patients were treated with a series of two intravitreal injections per week for the first 3 weeks (induction period), followed by one injection per week until an endpoint was reached or for a total of 24 weeks. Each injection contained 200 μg ganciclovir in a total volume of 0.1 mL, administered through a 30-gauge needle. The primary endpoint measures were the time to retinitis progression as evidenced by the development of a new lesion or progression by more than 750 μm of a preexisting lesion, and progression of CMV as evidenced by development of active disease in the untreated eye or at an extraocular site. Other outcome measures were changes in visual acuity and adverse events related to the intravitreal injections.Results: All of the treated eyes responded to induction therapy, and three eyes were successfully reinduced a total of four times. Median time to retinitis progression was 8.9 weeks (range, 6–20 wk). Cytomegalovirus disease of the untreated fellow eye or at an extraocular site occurred in four of seven patients (57%) for whom adequate follow-up data exist, at a mean of 11.7 weeks of study. Intravitreal injections were well-tolerated, and all complications were transient and reversible.Conclusions: Intravitreal ganciclovir therapy is effective for the treatment of active CMV retinitis, with median times to retinitis progression comparable to those achieved with systemic therapy, and may be administered with minimal complications. However, the frequent occurrence of CMV disease in the untreated fellow eye and at extraocular sites suggests that when used as sole therapy, intravitreal treatment should be given for short periods of time, when systemic therapy is not possible