Follow-up of 89 Asymptomatic Patients With Adrenoleukodystrophy Treated With Lorenzo's Oil

Hugo W. Moser, Gerald V. Raymond, Shou-En Lu, Larry R. Muenz, Ann B. Moser, Jiahong Xu, Richard O. Jones, Daniel J. Loes, Elias R. Melhem, Prachi Dubey, Lena Bezman, N. Hong Brereton (+1 others)
2005 Archives of Neurology  
Objectives: To identify asymptomatic boys with Xlinked adrenoleukodystrophy who have a normal magnetic resonance image (MRI), and to assess the effect of 4:1 glyceryl trioleate-glyceryl trierucate (Lorenzo's oil) on disease progression. Method: Eighty-nine boys (mean ± SD baseline age, 4.7±4.1 years; range, 0.2-15 years) were identified by a plasma very long-chain fatty acids assay used to screen at-risk boys. All were treated with Lorenzo's oil and moderate fat restriction. Plasma fatty acids
more » ... nd clinical status were followed for 6.9 ± 2.7 years. Changes in plasma hexacosanoic acid levels were assessed by measuring the length-adjusted area under the curve, and a proportional hazards model was used to evaluate association with the development of abnormal MRI results and neurological abnormalities. Results: Of the 89 boys, 24% developed MRI abnormalities and 11% developed both neurological and MRI
doi:10.1001/archneur.62.7.1073 pmid:16009761 fatcat:ciu5k2dsq5d3flcqnyf356r3vu