Treatment of the
vitamin E deficiency neurologic syndrome in children with chronic
cholestasis is hampered by the very poor intestinal absorption of available forms of
vitamin E, thus requiring prolonged treatment with
intramuscular injections of
vitamin E in many patients. D-
alpha-tocopheryl polyethylene glycol 1000 succinate (
TPGS) is a water-soluble investigational form of
vitamin E that is well absorbed during
cholestasis. We studied the effect of
TPGS therapy on the neurologic function in 12 children with
vitamin E deficiency (aged 9 months to 6 years) with prolonged forms of neonatal
cholestasis. Each child had failed to respond to up to 100 to 200 IU/kg/d of standard oral preparations of
vitamin E. Treatment with 15 to 25 IU/kg/d
TPGS for a mean of 19.3 months normalized the biochemical indices of
vitamin E status and was well tolerated by all patients. Neurologic function, assessed by serial neurologic examinations, remained normal during
therapy in the two children with no
neurologic symptoms younger than age 3 years at onset of
therapy. Neurologic function, which had deteriorated before this study, improved in six of seven patients with symptoms who were younger than 3 years and in all three with symptoms older than 3 years.
TPGS appears to be a safe and effective form of orally administered
vitamin E for use in children with chronic
cholestasis who are unresponsive to available oral preparations of
vitamin E.