1. Efficacy of two vitamin E formulations in patients with abetalipoproteinemia and chylomicron retention disease
- Author
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Philippe Moulin, Jocelyne Drai, Emmanuelle Reboul, Emile Levy, Charlotte Cuerq, Lioara Restier, Noël Peretti, Mathilde Di Filippo, Marie-Caroline Michalski, Alain Lachaux, Emilie Blond, Pierre Poinsot, Agnès Sassolas, Sybil Charrière, Cyrielle Caussy, Christian Laveille, Christophe Marçais, Emilie Henin, Cardiovasculaire, métabolisme, diabétologie et nutrition (CarMeN), Institut National de la Recherche Agronomique (INRA)-Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Institut National des Sciences Appliquées de Lyon (INSA Lyon), Université de Lyon-Institut National des Sciences Appliquées (INSA)-Institut National des Sciences Appliquées (INSA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Hospices Civils de Lyon (HCL), Laboratoire de Mécanique des Systèmes et des Procédés (LMSP), Centre National de la Recherche Scientifique (CNRS), Centre Hospitalier Lyon Sud [CHU - HCL] (CHLS), Hospices Civils de Lyon (HCL), Nutrition, obésité et risque thrombotique (NORT), Aix Marseille Université (AMU)-Institut National de la Recherche Agronomique (INRA)-Institut National de la Santé et de la Recherche Médicale (INSERM), Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research (C2VN), Institut National de la Recherche Agronomique (INRA)-Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM), Université Claude Bernard Lyon 1 (UCBL), Université de Lyon, Université de Montréal (UdeM), Centre recherche en CardioVasculaire et Nutrition (C2VN), and ProdInra, Migration
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Male ,0301 basic medicine ,apoprotein-b ,[SDV]Life Sciences [q-bio] ,medicine.medical_treatment ,tissu adipeux ,métabolisme des lipides ,Anderson disease ,adipose-tissue ,Biochemistry ,Gastroenterology ,Hypobetalipoproteinemias ,chemistry.chemical_compound ,0302 clinical medicine ,Endocrinology ,liquid-chromatography ,Vitamin E ,chronic cholestasis ,triglyceride transfer protein ,Research Articles ,Vitamin E Acetate ,lipoprotéine ,[SDV.MHEP] Life Sciences [q-bio]/Human health and pathology ,chylomicron ,Middle Aged ,tocopherol ,metabolic disease ,Abetalipoproteinemia ,adipose tissue ,3. Good health ,lipid and lipoprotein metabolism ,absorption ,hypocholesterolemia ,Female ,030211 gastroenterology & hepatology ,Vitamin E deficiency ,Safety ,Chylomicron retention disease ,Adult ,Biochemistry & Molecular Biology ,medicine.medical_specialty ,Drug Compounding ,Drug Storage ,Médecine humaine et pathologie ,Biological Availability ,andersons disease ,QD415-436 ,Tocofersolan ,03 medical and health sciences ,Malabsorption Syndromes ,alpha-tocopherol ,Internal medicine ,abetalipoproteinémie ,medicine ,Humans ,hypocholestérolemie ,adipose ,business.industry ,tissue ,Cell Biology ,fatty-acids ,maladie métabolique ,mutations ,medicine.disease ,030104 developmental biology ,Fat-Soluble Vitamin ,Intestinal Absorption ,chemistry ,Case-Control Studies ,Human health and pathology ,business ,alpha-Tocopherol ,[SDV.MHEP]Life Sciences [q-bio]/Human health and pathology ,lipid-metabolism - Abstract
International audience; Abetalipoproteinemia (ABL) and chylomicron retention disease (CMRD) are extremely rare recessive forms of hypobetalipoproteinemia characterized by intestinal lipid malabsorption and severe vitamin E deficiency. Vitamin E is often supplemented in the form of fat-soluble vitamin E acetate, but fat malabsorption considerably limits correction of the deficiency. In this crossover study, we administered two different forms of vitamin E, tocofersolan (a water-soluble derivative of RRR-alpha-tocopherol) and alpha-tocopherol acetate, to three patients with ABL and four patients with CMRD. The aims of this study were to evaluate the intestinal absorption characteristics of tocofersolan versus alpha-tocopherol acetate by measuring the plasma concentrations of alpha-tocopherol over time after a single oral load and to compare efficacy by evaluating the ability of each formulation to restore vitamin E storage after 4 months of treatment. In patients with ABL, tocofersolan and alpha-tocopherol acetate bioavailabilities were extremely low (2.8% and 3.1%, respectively). In contrast, bioavailabilities were higher in patients with CMRD (tocofersolan, 24.7%; alpha-tocopherol acetate, 11.4%). Plasma concentrations of alpha-tocopherol at 4 months were not significantly different by formulation type in ABL or CMRD. This study provides new insights about vitamin E status in ABL and CMRD and suggests the potential of different formulations as treatment options.
- Published
- 2018
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