Multicenter trial of d-alpha-tocopheryl polyethylene glycol 1000 succinate for treatment of vitamin E deficiency in children with chronic cholestasis.

Abstract

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.

• Authors: R J Sokol, N Butler-Simon, C Conner, J E Heubi, F R Sinatra, F J Suchy, M B Heyman, J Perrault, R J Rothbaum, J Levy
• Journal: Gastroenterology (Gastroenterology) Vol. 104 Issue 6 Pg. 1727-35 (Jun 1993) ISSN: 0016-5085 [Print] United States
• PMID: 8500733 (Publication Type: Clinical Trial, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S.)

Chemical References

• Vitamin E
• Polyethylene Glycols
• Copper
• tocophersolan

Topics

• Adolescent
• Adult
• Age Factors
• Child
• Child, Preschool
• Cholestasis (complications, drug therapy)
• Chronic Disease
• Copper (blood)
• Female
• Humans
• Infant
• Male
• Polyethylene Glycols
• Stereoisomerism
• Vitamin E (administration & dosage, analogs & derivatives, therapeutic use)
• Vitamin E Deficiency (complications, drug therapy)