title: Docosahexaenoic acid therapy in peroxisomal diseases: results of a double-blind, randomized trial.
abstract: OBJECTIVE
  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 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.
  
  
  CONCLUSIONS
  DHA supplementation did not improve the visual function or growth of treated individuals with peroxisome assembly disorders.
  
  
  METHODS
  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.
authors.affiliation: University of Texas Medical Branch, Galveston, TX, USA.
authors.forename: A M
authors.initials: AM
authors.lastname: Paker
authors.forename: J S
authors.initials: JS
authors.lastname: Sunness
authors.forename: N H
authors.initials: NH
authors.lastname: Brereton
authors.forename: L J
authors.initials: LJ
authors.lastname: Speedie
authors.forename: L
authors.initials: L
authors.lastname: Albanna
authors.forename: S
authors.initials: S
authors.lastname: Dharmaraj
authors.forename: A B
authors.initials: AB
authors.lastname: Moser
authors.forename: R O
authors.initials: RO
authors.lastname: Jones
authors.forename: G V
authors.initials: GV
authors.lastname: Raymond
mesh_terms: D001827:Body Height / Q000187:drug effects / Q000502:physiology; D002675:Child, Preschool; D019587:Dietary Supplements; D004281:Docosahexaenoic Acids / Q000494:pharmacology / Q000627:therapeutic use*; D004311:Double-Blind Method; D004596:Electroretinography / Q000187:drug effects; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D018901:Peroxisomal Disorders / Q000188:drug therapy* / Q000503:physiopathology; D052919:Refsum Disease, Infantile / Q000188:drug therapy* / Q000503:physiopathology; D016896:Treatment Outcome; D014796:Visual Perception / Q000187:drug effects / Q000502:physiology; D015211:Zellweger Syndrome / Q000188:drug therapy* / Q000503:physiopathology
chemical_list: D004281:Docosahexaenoic Acids
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