title: Therapeutic plasma exchange in treatment of adrenoleukodystrophy.
abstract: Adrenoleukodystrophy (ALD) is an X-linked disorder of metabolism of very long-chain fatty acids (VLCFA) with a frequency of up to 1:20,000 in males. VLCFA C 24:0 and C 26:0 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. Presently, no sure therapeutic approaches are established. Efforts were reported by dietary regimens with VLCFA-restriction and glycerol trioleate and glycerol trierucate intake. In the present trial, we report on a 58-year-old white male suffering from progressive ALD with spastic paraparesis. He has a positive family history back to the 18th century. In this patient, although maximum dietary therapy was applied over a period of 60 months, no normalization of VLCFA C24:0 and C26:0 was reached, and neurological disorders were progressive. As a result, therapeutic plasma exchange (TPE) was applied from 1990 to 1994. Then, for more selective adsorption of VLCFA, dextran-sulfate adsorption (Liposorber, Kaneka, Osaka, Japan) until 1996, and after that, immunoadsorption (Therasorb, Baxter, Munchen, Germany) were used. During these periods (total, 101 months), VLCFA C 24:0 and C 26:0 levels were reduced by 55% and 50% (p < 0.001). The patient experienced a significant improvement in performance and general well-being. No further progression of neuronal disorders was documented. This anecdotal data suggest a very beneficial effect of TPE in treatment of progressive ALD.
authors: Institute for Blood Purification, Homburg/Saar, Germany.
authors: R
authors: R
authors: Bambauer
authors: R
authors: R
authors: Schiel
authors: H
authors: H
authors: Cordes
authors: R
authors: R
authors: Latza
mesh_terms: D000326:Adrenoleukodystrophy / Q000097:blood / Q000178:diet therapy / Q000628:therapy*; D003627:Data Interpretation, Statistical; D004041:Dietary Fats / Q000008:administration & dosage; D005227:Fatty Acids / Q000097:blood; D005500:Follow-Up Studies; D006801:Humans; D007163:Immunosorbent Techniques; D008297:Male; D008875:Middle Aged; D010951:Plasma Exchange / Q000379:methods; D013997:Time Factors
chemical_list: D004041:Dietary Fats; D005227:Fatty Acids