title: Oral Tocofersolan Corrects or Prevents Vitamin E Deficiency in Children With Chronic Cholestasis.
abstract: D-Alpha-tocopheryl polyethylene glycol 1000 succinate (Tocofersolan, Vedrop), has been developed in Europe to provide an orally bioavailable source of vitamin E in children with cholestasis. The aim was to analyze the safety/efficacy of Vedrop in a large group of children with chronic cholestasis.
  
  
  
  Two hundred seventy-four children receiving Vedrop for vitamin E deficiency or for its prophylaxis were included from 7 European centers. Median age at treatment onset was 2 months and median follow-up was 11 months. Vedrop was prescribed at a daily dose of 0.34 mL/kg (25 IU/kg) of body weight. Three methods were used to determine a sufficient serum vitamin E status: vitamin E, vitamin E/(total cholesterol), vitamin E/(total cholesterol + triglycerides).
  
  
  
  Before Vedrop therapy, 51% of children had proven vitamin E deficiency, 30% had normal vitamin E status and 19% had an unknown vitamin E status. During the first months of treatment, vitamin E status was restored in the majority of children with insufficient levels at baseline (89% had a normal status at 6 months). All children with a normal baseline vitamin E status had a normal vitamin E status at 6 months. Among children with an unknown vitamin E status at baseline, 93% had a normal vitamin E status at 6 months. A sufficient vitamin E status was observed in 80% of children with significant cholestasis (serum total bilirubin >34.2 μmol/L). No serious adverse reaction was reported.
  
  
  
  Vedrop seems a safe and effective oral formulation of vitamin E that restores and/or maintains sufficient serum vitamin E level in the majority of children with cholestasis, avoiding the need for intramuscular vitamin E injections.
authors.affiliation: *Pediatric Hepatology and Pediatric Liver Transplantation Unit, National Reference Centre for biliary atresia, Assistance Publique-Hôpitaux de Paris, Université Paris Sud, Hepatinov, Le Kremlin-Bicêtre, France †Center for Inherited metabolic Diseases, Karolinska University Hospital Solna, Stockholm, Sweden ‡Medical Department, Orphan Europe, Puteaux, France §Beatrix Childrens Hospital, University Medical Center Groningen, Groningen, The Netherlands ||Division of Paediatric Gastroenterology and Hepatology, Children's Hospital, Hannover Medical School, Hannover, Germany ¶Department of Paediatric Gastroenterology and Hepatology, Academisch Medisch Centrum, Amsterdam, The Netherlands #Department of Pediatric Gastroenterology and Hepatology, CHRU de Lille, Hôpital Jeanne de Flandre, Lille, France **Wilhelmina Childrens Hospital, University Medical Center Utrecht, Utrecht, The Netherlands ††INSERM U1174, Université Paris-Sud, Orsay, France.
authors.forename: Alice
authors.initials: A
authors.lastname: Thébaut
authors.forename: Antal
authors.initials: A
authors.lastname: Nemeth
authors.forename: Jeannie
authors.initials: J
authors.lastname: Le Mouhaër
authors.forename: René
authors.initials: R
authors.lastname: Scheenstra
authors.forename: Ulrich
authors.initials: U
authors.lastname: Baumann
authors.forename: Bart
authors.initials: B
authors.lastname: Koot
authors.forename: Fredéric
authors.initials: F
authors.lastname: Gottrand
authors.forename: Roderick
authors.initials: R
authors.lastname: Houwen
authors.forename: Laure
authors.initials: L
authors.lastname: Monard
authors.forename: Sylvie Lafaye
authors.initials: SL
authors.lastname: de Micheaux
authors.forename: Dalila
authors.initials: D
authors.lastname: Habes
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Jacquemin
mesh_terms: D000284:Administration, Oral; D002648:Child; D002675:Child, Preschool; D002779:Cholestasis / Q000150:complications*; D002908:Chronic Disease; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D012042:Registries; D014810:Vitamin E / Q000008:administration & dosage* / Q000009:adverse effects / Q000097:blood; D014811:Vitamin E Deficiency / Q000097:blood / Q000188:drug therapy* / Q000517:prevention & control*; D014815:Vitamins / Q000008:administration & dosage* / Q000009:adverse effects
chemical_list: D014815:Vitamins; D014810:Vitamin E; C014225:tocophersolan