All issues > Volume 48(11); 2005

Original Article

Korean J Pediatr. 2005;48(11):1232-1238. Published online November 15, 2005.

Lorenzo's Oil Treatment in X-linked Adrenoleukodystrophy

Hyo Jung HJ Kim¹, Eun Young EY Kim¹, Young Jong YJ Woo², Hoon H Kook²

¹Department of Pediatrics, Kwangju Christian Hospital, Gwangju, Korea
²Department of Pediatrics, Chonnam National University Medical School, Gwangju, Korea

Correspondence Eun Young EY Kim ,Email: eykim_kook@yahoo.com

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.

Keywords :Lorenzo's oil therapy; X-linked adrenoleukodystrophy