Trial reportJournal of neurovirology2013
A study of mefloquine treatment for progressive multifocal leukoencephalopathy: results and exploration of predictors of PML outcomes.
Trial report in Journal of neurovirology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00746941 (A Randomized, Rater-Blinded Study to Explore the Effect of Mefloquine in Subjects With Progressive Multifocal Leukoencephalopathy), which is not on this map. Cited by 73 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Randomized, Rater-Blinded Study to Explore the Effect of Mefloquine in Subjects With Progressive Multifocal Leukoencephalopathy (PML)
Who cites it
73 citing papers in PubMed, 1 synthesis or guideline pooled it, 162 citations in OpenAlex.
- From hydroxychloroquine to ivermectin: what are the anti-viral properties of anti-parasitic drugs to combat SARS-CoV-2?Journal of travel medicine · 2021Pooled it
- Characteristics of progressive multifocal leukoencephalopathy in hematological malignancies, With reference to HIV-infected patients.International journal of hematology · 2026Article
- Current Perspectives on JC Polyomavirus Transmission and Associated Diseases: Implications for Prevention in Risk Populations.Viruses · 2026Review
- Progressive Multifocal Leukoencephalopathy Initially Considered as Central Nervous System Involvement of Post-Transplant Lymphoproliferative Disorder: The Critical Role of Contrast Enhancement on Magnetic Resonance Imaging.Internal medicine (Tokyo, Japan) · 2026Article
- Article
- JC Polyomavirus Infection: A Narrative Review.Infectious diseases and therapy · 2025Review
- Article
- Review
- Natalizumab-associated progressive multifocal leukoencephalopathy.Frontiers in neurology · 2025Review
- Article
- Upregulation of the NKG2D Ligand ULBP2 by JC Polyomavirus Infection Promotes Immune Recognition by Natural Killer Cells.The Journal of infectious diseases · 2024Article
- Biomarkers for progressive multifocal leukoencephalopathy: emerging data for use of JC virus DNA copy number in clinical trials.The Lancet. Neurology · 2024Review
- Progressive Multifocal Leukoencephalopathy: Pathogenesis, Diagnostic Tools, and Potential Biomarkers of Response to Therapy.Neurology · 2023Review
- Long-term survival from progressive multifocal leukoencephalopathy in living-donor liver transplant recipient with preformed donor-specific antibody.Journal of neurovirology · 2023Article
- Long-term prognosis and overall mortality in patients with progressive multifocal leukoencephalopathy.Scientific reports · 2023Article
- Cerebellar Progressive Multifocal Leukoencephalopathy Mimicking Anti-Yo-Antibody-Associated Rapidly Progressive Cerebellar Syndrome.Neurology international · 2023Article
- An Elusive Target: Inhibitors of JC Polyomavirus Infection and Their Development as Therapeutics for the Treatment of Progressive Multifocal Leukoencephalopathy.International journal of molecular sciences · 2023Review
- Successful treatment of HIV-associated progressive multifocal leukoencephalopathy (PML) with mirtazapine, mefloquine, and IVIG combination therapy: a case report.Journal of neurovirology · 2023Article
- Progressive multifocal leukoencephalopathy 10 years following transplant: 5HT receptor antagonism as an adjunct to immune reconstitution.BMJ case reports · 2022Article
- [Immune checkpoint inhibitors in the treatment of progressive multifocal leukoencephalopathy].Der Nervenarzt · 2022Article
13 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 7 institutions in 3 countries.
Funding
Abstract
Immune reconstitution has improved outcomes for progressive multifocal leukoencephalopathy (PML), a potentially lethal brain disease caused by JC virus (JCV). However, an antiviral treatment to control JCV is needed when immune reconstitution is delayed or not possible. On the basis of in vitro efficacy, this study evaluated the effect of mefloquine on PML and factors that may predict PML outcomes. This 38-week, open-label, randomized, parallel-group, proof-of-concept study compared patients with PML who received standard of care (SOC) with those who received SOC plus mefloquine (250 mg for 3 days, then 250 mg weekly). Patients randomized to SOC could add mefloquine treatment at week 4. The primary endpoint was change from baseline to weeks 4 and 8 in JCV DNA copy number (load) in cerebrospinal fluid (CSF). Exploratory analyses evaluated factors that might correlate with clinical outcome. The majority of enrolled patients were HIV positive. Preplanned interim data analyses suggested that the study was unlikely to successfully demonstrate a significant difference between groups; therefore, the study was terminated prematurely. There was no significant difference between groups in CSF JCV DNA loads or clinical/MRI findings. Decrease in CSF JCV DNA load from baseline to week 4 was associated with a better clinical outcome at 16 weeks, as measured by Karnofsky scores. This study found no evidence of anti-JCV activity by mefloquine. An early decrease of CSF JCV DNA load appears to be associated with a better clinical outcome.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.