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Vitamin K Antagonist Warfarin for Palliative Treatment of Metachromatic Leukodystrophy, A Compassionate Study of Four Subjects

Libertas Academica
Publication Date
  • Journal Of Central Nervous System Disease
  • Volume: 2012
  • Issue: 4
  • Biology
  • Chemistry
  • Medicine


Journal of Central Nervous System Disease 2012:4 73–79 doi: 10.4137/JCNSD.S9355 This article is available from © the author(s), publisher and licensee Libertas Academica Ltd. This is an open access article. Unrestricted non-commercial use is permitted provided the original work is properly cited. Open Access Full open access to this and thousands of other papers at Journal of Central Nervous System Disease O r i g i N A L r e S e A r C h Journal of Central Nervous System Disease 2012:4 73 Vitamin K Antagonist Warfarin for palliative Treatment of Metachromatic Leukodystrophy, A compassionate study of Four subjects Mitra Assadi1, Dah-Jyuu Wang3, Kelly Anderson4, Melissa Carran2, Larissa Bilaniuk3 and Paola Leone4 1robert Wood Johnson Medical School, 2Cooper University hospital, 3University of Pennsylvania, 4University of Medicine and Dentistry of New Jersey. Corresponding author email: [email protected] Abstract: MLD is characterized by accumulation of sulfatides in the brain. Vitamin K regulates two enzymes in sphingolipid biosynthe- sis 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 toler- ated 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 fou

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