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Abstract
Objectives: 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.
Methods: 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.
Results: 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 β 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.
Conclusions: DHA supplementation did not improve the visual function or growth of treated individuals with peroxisome assembly disorders.
Classification of evidence: 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.
Footnotes
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Study funding: Supported by the Food and Drug FD-R-001289 (G.V.) and the Johns Hopkins University School of Medicine General Clinical Research Center M01 RR00052 from the National Center for Research Resources. Supply of agent and placebo were provided by Martek Biosciences Corp, Columbia, Maryland.
Disclosure: Author disclosures are provided at the end of the article.
Received April 3, 2009. Accepted in final form May 13, 2010.
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