Treating homozygous familial hypercholesterolemia in a real-world setting: Experiences with lomitapide

Roeters Van Lennep, J.
Averna, M.
Alonso, R.

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Publication date

January 2015

DOI

10.1016/j.jacl.2015.05.001

Publisher

Elsevier BV

ISSN

1933-2874

Citation count (estimate)

Abstract

Homozygous familial hypercholesterolaemia (HoFH) is a rare genetic disease characterised by markedly elevated plasma levels of low-density lipoprotein-cholesterol (LDL-C). Lomitapide is a microsomal triglyceride transfer protein (MTP) inhibitor approved as an adjunct to other lipid-lowering therapies (LLTs), with or without lipoprotein apheresis (LA), for the treatment of adult HoFH. Diet with <20% calories from fat is required. Due to a varying genetic and phenotypic profile of patients with HoFH, individual patients may respond to therapy differently; therefore examining individual cases in a 'real-world' setting provides valuable information on the effective day-to-day management of HoFH cases. Four HoFH cases were selected for analysis ...