Docosahexaenoic acid therapy in peroxisomal diseases: results of a double-blind, randomized trial.

Mark McIntosh,'s picture
PubMed URL: 
http://www.ncbi.nlm.nih.gov/pubmed/20805528
Author: 
Raymond GV
Author List: 
Paker AM
Sunness JS
Brereton NH
Speedie LJ
Albanna L
Dharmaraj S
Moser AB
Jones RO
Raymond GV
Journal: 
Neurology
PubMed ID: 
20805528
Pagination: 
826-30
Volume: 
75
Issue: 
9
Abstract: 
Peroxisome assembly disorders are genetic disorders characterized by biochemical abnormalities, including low docosahexaenoic acid (DHA). The objective was to assess whether treatment with DHA supplementation would improve biochemical abnormalities, visual function, and growth in affected individuals.This was a randomized, double-blind, placebo-controlled trial conducted at a single center. Treatment groups received supplements of DHA (100 mg/kg per day). The primary outcome measures were the change from baseline in the visual function and physical growth during the 1 year follow-up period.Fifty individuals were enrolled and randomized. Two were subsequently excluded from study analysis when it was determined that they had a single enzyme disorder of peroxisomal beta oxidation. Thirty-four returned for follow-up. Nine patients died during the trial of their disorder, and 5 others were lost to follow-up. DHA supplementation was well tolerated. There was no difference in the outcomes between the treated and untreated groups in biochemical function, electroretinogram, or growth. Improvements were seen in both groups in certain individuals.DHA supplementation did not improve the visual function or growth of treated individuals with peroxisome assembly disorders.This interventional study provides Class II evidence that DHA supplementation did not improve the visual function or growth of treated individuals with peroxisome assembly disorders during an average of 1 year of follow-up in patients aged 1 to 144 months.
Published Date: 
August, 2010

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