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

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

Research output: Contribution to journalArticle

100 Citations (Scopus)

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-α-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.

Original languageEnglish (US)
Pages (from-to)1727-1735
Number of pages9
JournalGastroenterology
Volume104
Issue number6
StatePublished - 1993

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Vitamin E Deficiency
Cholestasis
Succinic Acid
Vitamin E
Multicenter Studies
Therapeutics
polyethylene glycol 1000
Liver Diseases
Safety

ASJC Scopus subject areas

  • Gastroenterology

Cite this

Sokol, R. J., Butler-Simon, N., Conner, C., Heubi, J. E., Sinatra, F. R., Suchy, F. J., ... Narkewicz, M. R. (1993). Multicenter trial of d-α-tocopheryl polyethylene glycol 1000 succinate for treatment of vitamin E deficiency in children with chronic cholestasis. Gastroenterology, 104(6), 1727-1735.

Multicenter trial of d-α-tocopheryl polyethylene glycol 1000 succinate for treatment of vitamin E deficiency in children with chronic cholestasis. / Sokol, Ronald J.; Butler-Simon, Nancy; Conner, Colin; Heubi, James E.; Sinatra, Frank R.; Suchy, Frederick J.; Heyman, Melvin B.; Perrault, Jean; Rothbaum, Robert J.; Levy, Joseph; Iannaccone, Susan T.; Shneider, Benjamin L.; Koch, Thomas K.; Narkewicz, Michael R.

In: Gastroenterology, Vol. 104, No. 6, 1993, p. 1727-1735.

Research output: Contribution to journalArticle

Sokol, RJ, Butler-Simon, N, Conner, C, Heubi, JE, Sinatra, FR, Suchy, FJ, Heyman, MB, Perrault, J, Rothbaum, RJ, Levy, J, Iannaccone, ST, Shneider, BL, Koch, TK & Narkewicz, MR 1993, 'Multicenter trial of d-α-tocopheryl polyethylene glycol 1000 succinate for treatment of vitamin E deficiency in children with chronic cholestasis', Gastroenterology, vol. 104, no. 6, pp. 1727-1735.
Sokol, Ronald J. ; Butler-Simon, Nancy ; Conner, Colin ; Heubi, James E. ; Sinatra, Frank R. ; Suchy, Frederick J. ; Heyman, Melvin B. ; Perrault, Jean ; Rothbaum, Robert J. ; Levy, Joseph ; Iannaccone, Susan T. ; Shneider, Benjamin L. ; Koch, Thomas K. ; Narkewicz, Michael R. / Multicenter trial of d-α-tocopheryl polyethylene glycol 1000 succinate for treatment of vitamin E deficiency in children with chronic cholestasis. In: Gastroenterology. 1993 ; Vol. 104, No. 6. pp. 1727-1735.
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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-α-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.",
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AU - Sokol, Ronald J.

AU - Butler-Simon, Nancy

AU - Conner, Colin

AU - Heubi, James E.

AU - Sinatra, Frank R.

AU - Suchy, Frederick J.

AU - Heyman, Melvin B.

AU - Perrault, Jean

AU - Rothbaum, Robert J.

AU - Levy, Joseph

AU - Iannaccone, Susan T.

AU - Shneider, Benjamin L.

AU - Koch, Thomas K.

AU - Narkewicz, Michael R.

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N2 - 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-α-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.

AB - 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-α-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.

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