Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia

Maurizio Averna, Marina Cuchel, Dirk J. Blom

Risultato della ricerca: Article

23 Citazioni (Scopus)

Abstract

The microsomal triglyceride transfer protein (MTP) inhibitor lomitapide is a licenced adjunct to a low-fat diet and other lipid-lowering medication, with or without low-density lipoprotein apheresis, for the treatment of adults with homozygous familial hypercholesterolaemia (HoFH). In a recently published phase 3 study, patients with HoFH received lomitapide in addition to maximally tolerated lipid-lowering therapy. Treatment with lomitapide resulted in a mean approximate 50% reduction in LDL-C levels after 26 weeks compared with baseline levels (p < 0.0001). This decrease in LDL-C was maintained at Weeks 56 and 78 (44% [p < 0.0001] and 38% [p = 0.0001], respectively). This paper offers clinical perspectives based on selected case histories of patients participating in the phase 3 lomitapide study. These cases provide illustrative examples of the efficacy of lomitapide, with or without apheresis, and show that the effective management of adverse effects can enable patients to remain on effective treatment.
Lingua originaleEnglish
pagine (da-a)33-45
Numero di pagine13
RivistaAtherosclerosis Supplements
Volume15
Stato di pubblicazionePublished - 2014

Fingerprint

Hyperlipoproteinemia Type II
Blood Component Removal
Lipids
Therapeutics
Fat-Restricted Diet
LDL Lipoproteins
BMS201038

All Science Journal Classification (ASJC) codes

  • Internal Medicine
  • Cardiology and Cardiovascular Medicine

Cita questo

Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia. / Averna, Maurizio; Cuchel, Marina; Blom, Dirk J.

In: Atherosclerosis Supplements, Vol. 15, 2014, pag. 33-45.

Risultato della ricerca: Article

@article{f3dab1fb82f341fab1770cfb1a65d92e,
title = "Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia",
abstract = "The microsomal triglyceride transfer protein (MTP) inhibitor lomitapide is a licenced adjunct to a low-fat diet and other lipid-lowering medication, with or without low-density lipoprotein apheresis, for the treatment of adults with homozygous familial hypercholesterolaemia (HoFH). In a recently published phase 3 study, patients with HoFH received lomitapide in addition to maximally tolerated lipid-lowering therapy. Treatment with lomitapide resulted in a mean approximate 50{\%} reduction in LDL-C levels after 26 weeks compared with baseline levels (p < 0.0001). This decrease in LDL-C was maintained at Weeks 56 and 78 (44{\%} [p < 0.0001] and 38{\%} [p = 0.0001], respectively). This paper offers clinical perspectives based on selected case histories of patients participating in the phase 3 lomitapide study. These cases provide illustrative examples of the efficacy of lomitapide, with or without apheresis, and show that the effective management of adverse effects can enable patients to remain on effective treatment.",
author = "Maurizio Averna and Marina Cuchel and Blom, {Dirk J.}",
year = "2014",
language = "English",
volume = "15",
pages = "33--45",
journal = "Atherosclerosis Supplements",
issn = "1567-5688",
publisher = "Elsevier Ireland Ltd",

}

TY - JOUR

T1 - Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia

AU - Averna, Maurizio

AU - Cuchel, Marina

AU - Blom, Dirk J.

PY - 2014

Y1 - 2014

N2 - The microsomal triglyceride transfer protein (MTP) inhibitor lomitapide is a licenced adjunct to a low-fat diet and other lipid-lowering medication, with or without low-density lipoprotein apheresis, for the treatment of adults with homozygous familial hypercholesterolaemia (HoFH). In a recently published phase 3 study, patients with HoFH received lomitapide in addition to maximally tolerated lipid-lowering therapy. Treatment with lomitapide resulted in a mean approximate 50% reduction in LDL-C levels after 26 weeks compared with baseline levels (p < 0.0001). This decrease in LDL-C was maintained at Weeks 56 and 78 (44% [p < 0.0001] and 38% [p = 0.0001], respectively). This paper offers clinical perspectives based on selected case histories of patients participating in the phase 3 lomitapide study. These cases provide illustrative examples of the efficacy of lomitapide, with or without apheresis, and show that the effective management of adverse effects can enable patients to remain on effective treatment.

AB - The microsomal triglyceride transfer protein (MTP) inhibitor lomitapide is a licenced adjunct to a low-fat diet and other lipid-lowering medication, with or without low-density lipoprotein apheresis, for the treatment of adults with homozygous familial hypercholesterolaemia (HoFH). In a recently published phase 3 study, patients with HoFH received lomitapide in addition to maximally tolerated lipid-lowering therapy. Treatment with lomitapide resulted in a mean approximate 50% reduction in LDL-C levels after 26 weeks compared with baseline levels (p < 0.0001). This decrease in LDL-C was maintained at Weeks 56 and 78 (44% [p < 0.0001] and 38% [p = 0.0001], respectively). This paper offers clinical perspectives based on selected case histories of patients participating in the phase 3 lomitapide study. These cases provide illustrative examples of the efficacy of lomitapide, with or without apheresis, and show that the effective management of adverse effects can enable patients to remain on effective treatment.

UR - http://hdl.handle.net/10447/98644

M3 - Article

VL - 15

SP - 33

EP - 45

JO - Atherosclerosis Supplements

JF - Atherosclerosis Supplements

SN - 1567-5688

ER -