Abstract
AIM: Lomitapide is an oral inhibitor of the microsomal triglyceride transfer protein used to treat homozygous familial hypercholesterolemia (HoFH); patients require a low-fat diet to minimize gastrointestinal adverse effects and dietary supplements to prevent nutrient deficiencies. We investigated the diet and nutritional status during lomitapide treatment. METHODS: Japanese patients with HoFH, who were in a phase 3 trial of lomitapide, were instructed to start low-fat diets with supplements of vitamin E and essential fatty acids 6 weeks before starting lomitapide treatment. Dietary education was conducted by registered dietitians 16 times during the study period, which included a pre-treatment run-in phase (Weeks －6-0), a lomitapide treatment efficacy phase (Weeks 0-26) and a safety phase (Weeks 26-56). Two-day dietary records were collected at each dietary counseling session. Anthropometric and biochemical parameters were measured at Weeks 0, 26 and 56. RESULTS: Eight patients completed the 56 weeks of lomitapide treatment. Their median energy intakes derived from lipids were 19.2% and 17.9% during the efficacy and safety phases, respectively. "Fats and oils" intakes, and "Fatty meat and poultry" intakes in two patients, were successfully reduced to achieve low-fat diets. Although intakes of energy, fatty acids and fat-soluble vitamins did not differ significantly among phases, body weight, serum fatty acid levels and vitamin E concentrations were decreased at Week 26 as compared with Week 0. CONCLUSION: HoFH patients can adhere to low-fat diets with ongoing dietary counseling. Instructions about intakes of energy, fatty acids and fat-soluble vitamins, as well as periodic evaluations of nutritional status, are necessary.