Abstract | BACKGROUND AND AIMS: METHODS: The open-label, single-arm 'LOCHNES' study of lomitapide in FCS enrolled patients >18 years with genetically confirmed FCS, elevated fasting TG ≥ 750 mg/dL and history of pancreatitis. Patients were administered lomitapide to the maximum tolerated dose for 26 weeks. The primary endpoint was the percent change in TGs from baseline to Week 26. RESULTS: Eighteen patients were enrolled with median baseline TG levels 1803.5 mg/dL (97.5% CI, 1452-2391 mg/dL). At Week 26, median fasting TGs were reduced to 305 mg/dL (97.5% CI 219-801 mg/dL; 70.5% reduction); median lomitapide dose was 35 mg/day; 13 patients achieved TGs ≤750 mg/dL. Adverse events were mild to moderate and mainly related to gastrointestinal tolerability. Liver imaging at baseline and Week 26 revealed hepatic fat increases from median 12.0%-32.5%, while median hepatic stiffness remained normal. No patient experienced acute pancreatitis or severe abdominal pain during lomitapide treatment. CONCLUSIONS:
Lomitapide is effective and well tolerated in reducing TGs in FCS patients with a history of pancreatitis. Larger studies are warranted to determine lomitapide effectiveness in FCS.
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Authors | Angelo B Cefalù, Laura D'Erasmo, Gabriella Iannuzzo, Davide Noto, Antonina Giammanco, Anna Montali, Alberto Zambon, Francesco Forte, Patrizia Suppressa, Stefano Giannini, Carlo M Barbagallo, Antonina Ganci, Emilio Nardi, Federica Vernuccio, Rosalia Caldarella, Marcello Ciaccio, Marcello Arca, Maurizio Averna |
Journal | Atherosclerosis
(Atherosclerosis)
Vol. 359
Pg. 13-19
(10 2022)
ISSN: 1879-1484 [Electronic] Ireland |
PMID | 36152419
(Publication Type: Clinical Study, Journal Article, Research Support, Non-U.S. Gov't)
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Copyright | Copyright © 2022 The Authors. Published by Elsevier B.V. All rights reserved. |
Chemical References |
- BMS201038
- Benzimidazoles
- Triglycerides
|
Topics |
- Abdominal Pain
(epidemiology)
- Adult
- Benzimidazoles
(adverse effects)
- Humans
- Hyperlipoproteinemia Type I
(drug therapy)
- Pancreatitis
(epidemiology)
- Triglycerides
(blood)
|