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Progressive multifocal leukoencephalopathy: A 25-year retrospective cohort study
Pria Anand1, Gladia C Hotan2, Andre Vogel2
1From the Department of Neurology (P.A., A.V., N.V., F.J.M.), Massachusetts General Hospital, Boston; and Department of Brain and Cognitive Sciences (G.C.H.), Massachusetts Institute of Technology, Cambridge. pria.anand@gmail.com.
Neurology(R) Neuroimmunology & Neuroinflammation
|September 27, 2019
Summary
This study analyzed 91 progressive multifocal leukoencephalopathy (PML) cases over 25 years. HIV infection was the most common risk factor, and HIV-positive patients had significantly longer survival.
Area of Science:
- Neurology
- Infectious Diseases
- Epidemiology
Background:
- Progressive multifocal leukoencephalopathy (PML) is a rare, often fatal, demyelinating disease of the central nervous system.
- Understanding PML's risk factors, clinical course, and treatment is crucial for improving patient outcomes.
Purpose of the Study:
- To characterize the risk factors, clinical course, and treatment of patients diagnosed with progressive multifocal leukoencephalopathy (PML).
- To analyze outcomes in a large observational cohort over a 25-year period.
Main Methods:
- Retrospective analysis of 91 patients with confirmed PML (JC virus PCR or histopathology positive) between 1994 and 2019.
- Data collection included demographics, risk factors, clinical presentation, neuroimaging, and modified Rankin Scale (mRS) at follow-up.
Main Results:
- HIV infection was the most frequent risk factor (49%), followed by hematologic malignancies (31%) and immunosuppressive therapies (30% chemotherapy, 19% monoclonal antibodies).
- 30% of patients survived, with a median mRS of 2. Median survival was significantly longer in HIV-infected patients (1,992 days) vs. HIV-uninfected (101 days).
- 40 patients survived >1 year, and 13 survived >10 years, all of whom were HIV-infected.
Conclusions:
- This large cohort study provides valuable epidemiological data on PML.
- Findings highlight the significant impact of HIV status on PML survival and may inform future clinical trial design.
