Progressive multifocal leukoencephalopathy: Treatment and prognosis
- Igor J Koralnik, MD
Igor J Koralnik, MD
- Jean Schweppe Armour Professor of Neurology and Medicine
- Rush Medical College
- Chair, Department of Neurological Sciences
- Section Chief, Neuroinfectious Disease
- Rush University Medical Center
Progressive multifocal leukoencephalopathy (PML) is an often fatal demyelinating disease of the central nervous system that occurs almost exclusively in immunosuppressed individuals. This disease is caused by the polyomavirus JC (JCV). (See "Virology, epidemiology, and pathogenesis of JC polyomavirus, BK polyomavirus, and other human polyomaviruses" and "Progressive multifocal leukoencephalopathy: Epidemiology, clinical manifestations, and diagnosis".)
This topic will review the various approaches that have been employed to treat PML. Other aspects of JC viral infection and PML are discussed elsewhere. (See "Virology, epidemiology, and pathogenesis of JC polyomavirus, BK polyomavirus, and other human polyomaviruses" and "Progressive multifocal leukoencephalopathy: Epidemiology, clinical manifestations, and diagnosis".)
APPROACH TO TREATMENT
There is no specific treatment for PML, which has a high mortality rate. Therefore, the main approach is restoring the host adaptive immune response, a strategy that appears to prolong survival [1,2]. Implementation of this strategy differs according to the clinical setting:
●Initiating or optimizing effective antiretroviral therapy (ART) for patients with HIV infection
●Withdrawing immunosuppressive drugs (when possible) for patients without HIV infection
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- APPROACH TO TREATMENT
- HIV infection
- Immunosuppression without HIV infection
- Natalizumab-associated PML
- Inflammatory PML
- Pharmacologic agents
- - Cytarabine
- - Cidofovir
- - Topotecan
- - Mirtazapine
- - Maraviroc
- - Interleukin 7
- - Mefloquine
- NATURAL HISTORY AND PROGNOSIS
- Prognostic markers
- SUMMARY AND RECOMMENDATIONS