Lorenzo's Oil Treatment in X-linked Adrenoleukodystrophy

Kim, Hyo Jung; Kim, Eun Young; Woo, Young Jong; Kook, Hoon

Abstract

PURPOSE
Though more than 15 years have passed after introduction of Lorenzo's oil therapy in X- linked adrenoleukodystrophy (ALD), its efficacy is still fully not known. In patients who already have neurologic symptoms, most reports indicate that the neurologic disability continues to increase. We evaluated its efficacy in X-linked ALD patients. METHODS: Four patients who were treated with Lorenzo's oil for at least 12 months were enrolled from 1996 to 2003. During treatment, changes of neurologic symptoms, brain magnetic resonance imaging (MRI) findings, and serum saturated very long-chain fatty acid (C26: 0) were assessed. RESULTS: Two patients with childhood cerebral ALD had progression of neurologic symptoms and MRI lesions during treatment. One asymptomatic patient developed childhood cerebral ALD after six year treatment of Lorenzo's oil. One "Addison only" patient remained neurologically intact after three years. During Lorenzo's oil therapy, serum C26: 0 levels which had increased at diagnosis decreased in all four patients. CONCLUSION: Treatment with Lorenzo's oil did not prevent disease progression in childhood cerebral ALD patients who were already symptomatic. Long term follow up will be needed in asymptomatic ALD including "Addison only" to reveal the efficacy of Lorenzo's oil.