Format

Send to

Choose Destination
J Lipid Res. 2018 Sep;59(9):1640-1648. doi: 10.1194/jlr.M085043. Epub 2018 Jul 18.

Efficacy of two vitamin E formulations in patients with abetalipoproteinemia and chylomicron retention disease.

Author information

1
Biochemistry Department, Lyon Sud Hospital, Hospices Civils de Lyon, Lyon, France.
2
INSERM U1060, INRA UMR 1397, INSA-Lyon, CarMeN Laboratory, Université Lyon 1, Lyon, France.
3
Calvagone, Liergues, France.
4
Pediatric Hepato-Gastroenterology and Nutrition Unit, Hôpital Femme Mère Enfant de Lyon, Dyslipidemia Unity Hospices Civils de Lyon, Lyon, Bron, France.
5
Department of Biochemistry and Molecular Biology, Centre de Biologie et de Pathologie Est, Hospices Civils de Lyon, Lyon, Bron, France.
6
Fédération d'Endocrinologie, Maladies Métaboliques, Diabète et Nutrition, Hôpital Louis Pradel, Hospices Civils de Lyon, Lyon, Bron, France.
7
INRA, INSERM, Aix-Marseille University, Marseille, France.
8
Research Centre, CHU Sainte-Justine, and Department of Nutrition, Université de Montréal, Montréal, Québec, Canada.
9
INSERM U1060, INRA UMR 1397, INSA-Lyon, CarMeN Laboratory, Université Lyon 1, Lyon, France noel.peretti@chu-lyon.fr.

Abstract

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-α-tocopherol) and α-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 α-tocopherol acetate by measuring the plasma concentrations of α-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 α-tocopherol acetate bioavailabilities were extremely low (2.8% and 3.1%, respectively). In contrast, bioavailabilities were higher in patients with CMRD (tocofersolan, 24.7%; α-tocopherol acetate, 11.4%). Plasma concentrations of α-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.

KEYWORDS:

Anderson disease; absorption; adipose tissue; hypocholesterolemia; lipid and lipoprotein metabolism; metabolic disease; tocopherol

PMID:
30021760
PMCID:
PMC6121919
[Available on 2019-09-01]
DOI:
10.1194/jlr.M085043

Supplemental Content

Full text links

Icon for HighWire
Loading ...
Support Center