Oral Tocofersolan Corrects or Prevents Vitamin E Deficiency in Children With Chronic Cholestasis.

Autor: Thébaut A; 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., Nemeth A, Le Mouhaër J, Scheenstra R, Baumann U, Koot B, Gottrand F, Houwen R, Monard L, de Micheaux SL, Habes D, Jacquemin E
Jazyk: angličtina
Zdroj: Journal of pediatric gastroenterology and nutrition [J Pediatr Gastroenterol Nutr] 2016 Dec; Vol. 63 (6), pp. 610-615.
DOI: 10.1097/MPG.0000000000001331
Abstrakt: Objectives: 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.
Methods: 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).
Results: 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.
Conclusions: 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.
Databáze: MEDLINE
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