Treatment of non-AIDS progressive multifocal leukoencephalopathy with cytosine arabinoside

This open label study determined the outcome of non-AIDS progressive multif ocal leukoencephalopathy patients treated with a standard dose of intraveno us cytosine arabinoside. Nineteen patients with PML proven by brain biopsy or spinal fluid polymerase chain reaction were treated with intravenous cyt osine arabinoside 2 mg/kg per day for 5 days and followed for neurologic ou tcome by neurologic examination and MRI scanning. Seven of 19 PML patients treated with cytosine arabinoside intravenously improved neurologically. Th e range of follow-up for these patients was 2.0 to 4.5 years. All were left with neurologic deficits but were functionally improved, and 6 of 7 were a ble to independently carry out the activities of daily living. Twelve PML p atients showed no evidence of response and died rapidly of their disease af ter treatment (range, 8 days to 6 months). All who survived their neurologi c disease recovered from treatment-induced pancytopenia. Cytosine arabinosi de given intravenously to non-AIDS PML patients in this small study was ass ociated with a 36% chance of developing stabilization at 1 year. Treatment was associated with significant bone marrow toxicity. The improvement in NE U scan changes in those patients who responded took 6 weeks or longer.