Multicenter trial of d-alpha-tocopheryl polyethylene glycol 1000 succinate for treatment of vitamin E deficiency in children with chronic cholestasis.
BACKGROUND
Malabsorption and deficiency of vitamin E causing neurological degeneration are common consequences of chronic childhood cholestatic liver disease. The objective of this study was to determine the long-term efficacy and safety of d-alpha-tocopheryl polyethylene glycol 1000 succinate (TPGS) in correcting vitamin E deficiency in children with chronic cholestasis who were unresponsive to other forms of oral vitamin E.


METHODS
Sixty vitamin E-deficient children with chronic cholestasis unresponsive to 70-212 IU.kg-1.day-1 of oral vitamin E were entered into a trial at eight centers in the United States. After initial evaluation, treatment was started with 25 IU.kg-1.day-1 of TPGS. Vitamin E status, neurological function quantitated by a specific scoring system, and clinical and biochemical parameters were monitored during therapy.


RESULTS
All children responded to TPGS with normalization of vitamin E status. Neurological function, which had deteriorated before entry in the trial, improved in 25 patients, stabilized in 27, and worsened in only 2 after a mean of 2.5 years of therapy. No adverse effects were observed.


CONCLUSIONS
TPGS (20-25 IU.kg-1.day-1) appears to be a safe and effective form of vitamin E for reversing or preventing vitamin E deficiency during chronic childhood cholestasis.
Sokol
R J
RJ
Department of Pediatrics, University of Colorado School of Medicine, Denver.
Butler-Simon
N
N
Conner
C
C
Heubi
J E
JE
Sinatra
F R
FR
Suchy
F J
FJ
Heyman
M B
MB
Perrault
J
J
Rothbaum
R J
RJ
Levy
J
J
D000293:Adolescent; D000328:Adult; D000367:Age Factors; D002648:Child; D002675:Child, Preschool; D002779:Cholestasis / Q000150:complications* / Q000188:drug therapy; D002908:Chronic Disease; D003300:Copper / Q000097:blood; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D011092:Polyethylene Glycols; D013237:Stereoisomerism; D014810:Vitamin E / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D014811:Vitamin E Deficiency / Q000150:complications / Q000188:drug therapy*
D014810:Vitamin E; D011092:Polyethylene Glycols; D003300:Copper; C014225:tocophersolan