The lipid-lowering effects of lomitapide are unaffected by adjunctive apheresis in patients with homozygous familial hypercholesterolaemia - a post-hoc analysis of a phase 3, single-arm, open-label trial

dc.contributor.authorStefanutti, Claudia
dc.contributor.authorBlom, Dirk J.
dc.contributor.authorAverna, Maurizio R.
dc.contributor.authorMeagher, Emma A.
dc.contributor.authorTheron, Hendrik Du Toit
dc.contributor.authorMarais, A. David
dc.contributor.authorHegele, Robert A.
dc.contributor.authorSirtori, Cesare R.
dc.contributor.authorShah, Prediman K.
dc.contributor.authorGaudet, Daniel
dc.contributor.authorVigna, Giovanni B.
dc.contributor.authorSachais, B.S.
dc.contributor.authorDi Giacomo, S.
dc.contributor.authorDu Plessis, Anna M.E.
dc.contributor.authorBloeden, LeAnne T.
dc.contributor.authorBalser, J.
dc.contributor.authorRader, Daniel J.
dc.contributor.authorCuchel, Marina
dc.date.accessioned2015-06-03T07:23:36Z
dc.date.available2015-06-03T07:23:36Z
dc.date.issued2015-06
dc.description.abstractOBJECTIVE : Lomitapide (a microsomal triglyceride transfer protein inhibitor) is an adjunctive treatment for homozygous familial hypercholesterolaemia (HoFH), a rare genetic condition characterised by elevated low-density lipoprotein-cholesterol (LDL-C), and premature, severe, accelerated atherosclerosis. Standard of care for HoFH includes lipid-lowering drugs and lipoprotein apheresis.We conducted a posthoc analysis using data from a Phase 3 study to assess whether concomitant apheresis affected the lipidlowering efficacy of lomitapide. 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 ¼ 0.545). Changes in Lp(a) levels were modest and not different between groups (p ¼ 0.436). CONCLUSION : The LDL-C lowering efficacy of lomitapide is unaffected by lipoprotein apheresis.en_ZA
dc.description.librarianhb2015en_ZA
dc.description.sponsorshipThe Phase 3 study was funded by a grant from the FDA Office of the Orphan Product Development (FD-R-003098) to Dr Cuchel and by Aegerion Pharmaceuticals Inc. Editorial support was provided by Nigel Eastmond of Eastmond Medicomm Ltd, which was funded by Aegerion Pharmaceuticals.en_ZA
dc.description.urihttp://www.elsevier.com/locate/atherosclerosisen_ZA
dc.identifier.citationStefanutti, C, Blom, DJ, Averna, MR et al 2015, 'The lipid-lowering effects of lomitapide are unaffected by adjunctive apheresis in patients with homozygous familial hypercholesterolaemia - a post-hoc analysis of a phase 3, single-arm, open-label trial', Atherosclerosis, vol. 240, no. 2, pp. 408-414.en_ZA
dc.identifier.issn0021-9150 (print)
dc.identifier.issn1879-1484 (online)
dc.identifier.other10.1016/j.atherosclerosis.2015.03.014
dc.identifier.urihttp://hdl.handle.net/2263/45377
dc.language.isoenen_ZA
dc.publisherElsevieren_ZA
dc.rights© 2015 The Authors. Published by Elsevier Ireland Ltd. This is an open access article under the CC BY-NCND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).en_ZA
dc.subjectLomitapideen_ZA
dc.subjectLipoprotein apheresisen_ZA
dc.subjectHomozygous familial hypercholesterolaemia (HoFH)en_ZA
dc.subjectLow-density lipoprotein-cholesterol (LDL-C)en_ZA
dc.titleThe lipid-lowering effects of lomitapide are unaffected by adjunctive apheresis in patients with homozygous familial hypercholesterolaemia - a post-hoc analysis of a phase 3, single-arm, open-label trialen_ZA
dc.typeArticleen_ZA

Files

Original bundle

Now showing 1 - 2 of 2
Loading...
Thumbnail Image
Name:
Stephanutti_Lipid_2015.pdf
Size:
548.34 KB
Format:
Adobe Portable Document Format
Description:
Postprint Article
Loading...
Thumbnail Image
Name:
Stephanutti_LipidSuppl_2015.pdf
Size:
176.62 KB
Format:
Adobe Portable Document Format
Description:
Suppl

License bundle

Now showing 1 - 1 of 1
Loading...
Thumbnail Image
Name:
license.txt
Size:
1.71 KB
Format:
Item-specific license agreed upon to submission
Description: