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

Journal of lipid research 2018 Sep

filter terms:

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. Copyright © 2018 Cuerq et al.

Citation

Charlotte Cuerq, Emilie Henin, Lioara Restier, Emilie Blond, Jocelyne Drai, Christophe Marçais, Mathilde Di Filippo, Christian Laveille, Marie-Caroline Michalski, Pierre Poinsot, Cyrielle Caussy, Agnès Sassolas, Philippe Moulin, Emmanuelle Reboul, Sybil Charriere, Emile Levy, Alain Lachaux, Noël Peretti. Efficacy of two vitamin E formulations in patients with abetalipoproteinemia and chylomicron retention disease. Journal of lipid research. 2018 Sep;59(9):1640-1648