Docosahexaenoic acid therapy in peroxisomal diseases: results of a double-blind, randomized trial.
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.
Paker
A M
AM
University of Texas Medical Branch, Galveston, TX, USA.
Sunness
J S
JS
Brereton
N H
NH
Speedie
L J
LJ
Albanna
L
L
Dharmaraj
S
S
Moser
A B
AB
Jones
R O
RO
Raymond
G V
GV
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
D004281:Docosahexaenoic Acids
Brain Res. 1992 Jun 26;583(1-2):171-82
1504825
Proc Natl Acad Sci U S A. 1986 Jun;83(11):4021-5
3459166
J Mol Neurosci. 2001 Apr-Jun;16(2-3):309-16; discussion 317-21
11478386
Neurology. 1993 Jul;43(7):1389-97
8327143
N Engl J Med. 1984 May 3;310(18):1141-6
6709009
Neurology. 1998 Jul;51(1):26-32
9674774
Neurochem Res. 1999 Feb;24(2):187-97
9972864
Ann Neurol. 1999 Jan;45(1):100-10
9894883
Am J Med Genet. 1986 May;24(1):69-82
3706414
Mol Aspects Med. 1998 Apr;19(2):69-154
9827430
Science. 1973 Oct 5;182(4107):62-4
4730055