ArticleJournal of central nervous system disease2012
Vitamin k antagonist warfarin for palliative treatment of metachromatic leukodystrophy, a compassionate study of four subjects.
Article in Journal of central nervous system disease, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
6 citing papers in PubMed, 1 synthesis or guideline pooled it, 9 citations in OpenAlex.
- The natural history and burden of illness of metachromatic leukodystrophy: a systematic literature review.European journal of medical research · 2024Pooled it
- Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a long-term follow-up pilot study.Protein & cell · 2025Article
- Metachromatic Leukodystrophy: Diagnosis, Modeling, and Treatment Approaches.Frontiers in medicine · 2020Review
- Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective.Orphanet journal of rare diseases · 2019Review
- Multi-Voxel 1H-MRS in Metachromatic Leukodystrophy.Journal of central nervous system disease · 2013Article
- Developing therapeutic approaches for metachromatic leukodystrophy.Drug design, development and therapy · 2013Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
MLD is characterized by accumulation of sulfatides in the brain. Vitamin K regulates two enzymes in sphingolipid biosynthesis and warfarin is known to lower brain sulfatides in rats and mice. We hypothesized that warfarin may mitigate the MLD phenotype by reducing the formation of sulfatides. This compassionate study recruited four advanced patients with clinical, biochemical and genetic confirmation of MLD. The patients were treated with warfarin according to the approved protocol for a total of 45 days. The battery of tests included proton MR spectroscopy (H-MRS) of brain and urinary sulfatide levels recorded at defined intervals. The patients tolerated the medication and there were no bleeding complications. The urinary sulfatide levels did not decline during the study period. The H-MRS showed decreased N-acetyl aspartate and elevated myoinositol levels in the basal ganglia which remained unchanged after treatment. Our study did not demonstrate any beneficial effects of warfarin in four advanced cases of MLD. The drug intervention however, was safe and deserves further evaluation through a larger study of longer duration. The metabolite abnormalities reported on H-MRS may be useful in longitudinal follow up of patients with MLD during drug trials.
Indexed as
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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.