Response to cidofovir after failure of antiretroviral therapy alone in AIDS-associated progressive multifocal leukoencephalopathy

Abstract

Progressive multifocal leukoencephalopathy (PML) is a demyelinating brain disorder caused by the polyoma JC virus (JCV). It occurs in approximately 4% of patients with AIDS, producing a rapidly progressive, usually fatal course. There is no proven effective therapy for this condition. Despite initially promising reports of cytosine arabinoside treatment, later studies showed no efficacy of this agent.1,2 Disease stabilization was reported in patients taking highly active antiretroviral therapy (HAART), but further observations did not improve survival.3,4 There is a single anecdotal report of stabilization after cidofovir and cytarabine.5 Cidofovir is the most potent inhibitor of human and murine polyomavirus replication in vitro, and short-term stabilization of PML has been described using this agent alone.6 We report a patient with AIDS-associated PML showing a marked clinical and neuroradiologic improvement after cidofovir was added to HAART. A 32-year-old HIV-positive woman, a former IV drug abuser, was diagnosed with PML after presenting with a paresis …