title: Plasmapheresis in treatment of adrenoleukodystrophy.
abstract: Adrenoleukodystrophy (ALD) is an X-linked disorder of metabolism of very long chain fatty acids (VLCFAs) with a frequency of up to 1:20,000 in males. VLCFAs C24:0 and C26:0 accumulate in the cholesterol ester and ganglioside fraction in plasma and red cells. Symptoms of ALD are ataxia, loss of visual and auditory functions, and cerebral convulsions. Up to the present, no sure therapeutic approaches have been established. Efforts were reported by dietary regimens with VLCFA restriction and glyceroltrioleate and glyceroltrierucate intake. In the present trial, we report a 55-year-old Caucasian male suffering from progressive ALD with spastic paraparesis. He has had a positive family history since the eighteenth century. In this patient treated with maximum dietary therapy over a period of 60 months, no normalization of C24:0 and C26:0 was reached, and neurological disorders were progressive. As a result, plasmapheresis was applied during the period 1990-1994 and since then for more selective adsorption of VLCFAs, dextran sulfate adsorption (Liposorber, Kaneka, Japan). During this period (64 months), C24:0 and C26:0 levels were reduced by 54.5% and 51.8%, respectively (p = 0.0001). The patient experienced a significant improvement in performance and general well-being. There has been no further progression of neuronal disorders to document.
authors.affiliation: University of Saarland, Homburg, Germany.
authors.forename: R
authors.initials: R
authors.lastname: Bambauer
authors.forename: R
authors.initials: R
authors.lastname: Schiel
authors.forename: H
authors.initials: H
authors.lastname: Cordes
authors.forename: R
authors.initials: R
authors.lastname: Latza
authors.forename: J
authors.initials: J
authors.lastname: Klinkmann
authors.forename: J M
authors.initials: JM
authors.lastname: Schneidewind
mesh_terms: D000326:Adrenoleukodystrophy / Q000628:therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D010956:Plasmapheresis