Mefloquine Treatment in a Patient Suffering from Progressive Multifocal Leukoencephalopathy after Umbilical Cord Blood Transplant

Abstract

We report herein the case of a 37-year-old man who developed probable progressive multifocal leukoencephalopathy (PML) following an umbilical cord blood transplant. The patient showed favorable clinical, neuroradiological and virological responses after treatment with mefloquine, an anti-malarial drug. Mefloquine may offer some benefits as a treatment for PML in patients with or without human immunodeficiency virus type-1 infection. This report highlights the need to gather sufficient data to confirm the efficacy of mefloquine against this devastating viral disease of the central nervous system.

Mefloquine Improved Progressive Multifocal Leukoencephalopathy in a Patient with Systemic Lupus Erythematosus

“…With respect to PML in patients without HIV, there are currently no approved therapies, although cytosine arabinoside, α-interferon, and cidofovir have been reported effective in some cases. Mefloquine, a widely used antimalarial agent, was recently reported to inhibit the JC virus infection in vitro (2) and produce some clinical improvement (3)(4)(5). Here, we report the beneficial effect of mefloquine in a case of PML associated with systemic lupus erythematosus (SLE).…”

Molecular Biology, Epidemiology, and Pathogenesis of Progressive Multifocal Leukoencephalopathy, the JC Virus-Induced Demyelinating Disease of the Human Brain

“…Mefloquine is known to cross the BBB and accumulate in the brain, where JCV infection is pathological (221,388), but has been associated with neurotoxicity (493). Several independent case reports showed that mefloquine treatment of PML was successful in reducing the viral burden in the brain and was associated with improvement of clinical symptoms (168,244). However, as reported at the 2011 annual meeting of the American Academy of Neurology, a multicenter clinical trial supported by Biogen-IDEC Inc. and Elan Pharmaceuticals failed to show a reproducible reduction in the JCV DNA in PML patient CSF or reduced clinical progression of PML in response to mefloquine treatment (150).…”

Usefulness of 11C-methionine-positron emission tomography for the diagnosis of progressive multifocal leukoencephalopathy

“…Especially, neurologists are aware of the risk of PML when immunosuppressive therapy including natalizmab is used to treat multiple sclerosis [4]. Cases in which mefloquine therapy was effective have been reported [5,6].…”