title: 5-Lipoxygenase inhibition: a new treatment strategy for Sjögren-Larsson syndrome.
abstract: The Sjögren-Larsson syndrome (SLS) is a severe neurocutaneous disorder due to fatty aldehyde dehydrogenase (FALDH) deficiency. The recent discovery of the role of FALDH in the degradation of leukotriene B4 (LTB4) opened the way to the development of a new therapeutic strategy for SLS, i.e. 5-lipoxygenase inhibition. We treated one SLS patient with zileuton during five weeks. During the treatment period we found decreased values of LTB4 and omega-OH-LTB4. The severity of the pruritus diminished, and favorable changes in the child's behavior were observed. The height of the prominent "lipid peak" of cerebral white matter (that is characteristically found on proton magnetic resonance spectroscopy in SLS patients) decreased during treatment, and increased again when treatment was stopped. In conclusion, the beneficial effects of 5-lipoxygenase inhibition in SLS are very promising and encourage further research.
authors.affiliation: Department of Pediatric Neurology, University Hospital Nijmegen, The Netherlands.
authors.forename: M A
authors.initials: MA
authors.lastname: Willemsen
authors.forename: J J
authors.initials: JJ
authors.lastname: Rotteveel
authors.forename: P M
authors.initials: PM
authors.lastname: Steijlen
authors.forename: A
authors.initials: A
authors.lastname: Heerschap
authors.forename: E
authors.initials: E
authors.lastname: Mayatepek
mesh_terms: D000445:Aldehyde Oxidoreductases / Q000172:deficiency; D002648:Child; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D007975:Leukotriene B4 / Q000652:urine; D016859:Lipoxygenase Inhibitors / Q000008:administration & dosage* / Q000009:adverse effects; D016111:Sjogren-Larsson Syndrome / Q000175:diagnosis / Q000188:drug therapy* / Q000201:enzymology
chemical_list: D016859:Lipoxygenase Inhibitors; D007975:Leukotriene B4; D000445:Aldehyde Oxidoreductases; C038828:long-chain-aldehyde dehydrogenase; C063449:zileuton; D006918:Hydroxyurea