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Efficacy and safety of lomitapide in familial chylomicronaemia syndrome

  • Authors: Cefalu', Angelo Baldassare; D'Erasmo, Laura; Iannuzzo, Gabriella; Noto, Davide; Giammanco, Antonina; Montali, Anna; Zambon, Alberto; Forte, Francesco; Suppressa, Patrizia; Giannini, Stefano; Barbagallo, Carlo M; Ganci, Antonina; Nardi, Emilio; Vernuccio, Federica; Caldarella, Rosalia; Ciaccio, Marcello; Arca, Marcello; Averna, Maurizio
  • Publication year: 2022
  • Type: Articolo in rivista
  • OA Link: http://hdl.handle.net/10447/571065

Abstract

Familial chylomicronaemia syndrome (FCS) is a rare autosomal recessive disorder, resulting in elevated triglycerides (TGs), abdominal pain and pancreatitis. Treatment options are limited. Lomitapide, a microsomal triglyceride transfer protein inhibitor, is approved for the treatment of homozygous familial hypercholesterolaemia. Whether its therapeutic use may be extended to FCS remains unknown. The aim of this study was to evaluate the efficacy and safety of lomitapide in adult patients with FCS.