Abstract | OBJECTIVE: METHODS: Existing lipid-lowering therapy, including apheresis, was to remain stable from Week -6 to Week 26. Lomitapide dose was escalated on the basis of individual safety/tolerability from 5 mg to 60 mg a day (maximum). The primary endpoint was mean percent change in LDL-C from baseline to Week 26 (efficacy phase), after which patients remained on lomitapide through Week 78 for safety assessment and further evaluation of efficacy. During this latter period, apheresis could be adjusted. We analysed the impact of apheresis on LDL-C reductions in patients receiving lomitapide. RESULTS: Of the 29 patients that entered the efficacy phase, 18 (62%) were receiving apheresis at baseline. Twenty-three patients (13 receiving apheresis) completed the Week 26 evaluation. Of the six patients who discontinued in the first 26 weeks, five were receiving apheresis. There were no significant differences in percent change from baseline of LDL-C at Week 26 in patients treated (-48%) and not treated (-55%) with apheresis (p = 0.545). Changes in Lp(a) levels were modest and not different between groups (p = 0.436). CONCLUSION:
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Authors | C Stefanutti, D J Blom, M R Averna, E A Meagher, H dT Theron, A D Marais, R A Hegele, C R Sirtori, P K Shah, D Gaudet, G B Vigna, B S Sachais, S Di Giacomo, A M E du Plessis, L T Bloedon, J Balser, D J Rader, M Cuchel, Phase 3 HoFH Lomitapide Study Investigators |
Journal | Atherosclerosis
(Atherosclerosis)
Vol. 240
Issue 2
Pg. 408-14
(Jun 2015)
ISSN: 1879-1484 [Electronic] Ireland |
PMID | 25897792
(Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, Non-P.H.S.)
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Copyright | Copyright © 2015 The Authors. Published by Elsevier Ireland Ltd.. All rights reserved. |
Chemical References |
- Anticholesteremic Agents
- BMS201038
- Benzimidazoles
- Biomarkers
- Cholesterol, LDL
- Lipoprotein(a)
|
Topics |
- Adult
- Anticholesteremic Agents
(administration & dosage, adverse effects)
- Benzimidazoles
(administration & dosage, adverse effects)
- Biomarkers
(blood)
- Blood Component Removal
(adverse effects, methods)
- Cholesterol, LDL
(blood)
- Combined Modality Therapy
- Female
- Genetic Predisposition to Disease
- Homozygote
- Humans
- Hyperlipoproteinemia Type II
(blood, diagnosis, genetics, therapy)
- Lipoprotein(a)
(blood)
- Male
- Phenotype
- Time Factors
- Treatment Outcome
- Young Adult
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