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

1993; Elsevier BV; Volume: 104; Issue: 6 Linguagem: Inglês

10.1016/0016-5085(93)90652-s

1528-0012

Ronald J. Sokol, Nancy Butler–Simon, Colin Conner, James E. Heubi, Frank Sinatra, Frederick J. Suchy, Melvin B. Heyman, Jean Perrault, Robert Rothbaum, Joseph Levy, Susan T. Iannaccone, Benjamin L. Shneider, Thomas Koch, Michael R. Narkewicz,

Pharmacological Effects and Toxicity Studies

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.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.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.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.