OBJECTIVES: Lowering LDL-C in HoFH patients increases life expectancy. This analysis modelled the potential life-expectancy gain, and time to first major cardiovascular event (MACE) of HoFH patients when lomitapide was added to standard of care (SOC) in clinical practice.

METHODS: This analysis uses a published model of life expectancy and time to first MACE (EJPC 2017;24:1843) to explore the implications of LDL-C lowering achieved by lomitapide using baseline (treated with statins, ezetimibe, apheresis (67%)) and lomitapide-treated LDL-C levels from a cohort of HoFH patients (AdvTher 2017;34:1200).

RESULTS: Modelling predicted this cohort of HoFH patients, mean age 37.6 years with mean baseline LDL-C level 11.02mmol/L, who had LDL-C reduced by lomitapide by 68% (mean last visit value), to gain median 11.6 life years (LY) (mean 9.4) and delay median age of death to 53. If nadir LDL-C lowering of 76% is used, median LY gained is 12.5 (mean 10). ~80% of patients are modelled to have had a MACE by age 37.6; therefore, lomitapide has marginal impact on this. Treating the same cohort of HoFH patients with lomitapide at age 18 is modelled to further extend life expectancy, by a median 19.4 years (mean 14.5) to age 61, and delay time to first MACE by median 11.5 years (mean 8.3) to age 40. This increases to median gain of 20.2 LY and delay of 12.6 years for first MACE if the 76% nadir LDL-C reduction is used.

CONCLUSIONS: LDL-C reductions achieved with lomitapide in the real world are modelled to increase life-expectancy by a median of >11 years, even when started at an average age 37.6, or by >19 years when prescribed from age 18. This suggests that lomitapide has the potential to show significant survival benefits via lowering of LDL-C when used as an adjunct to SOC in HoFH patients.