Efficacy of Lomitapide in the Treatment of Familial Homozygous Hypercholesterolemia: Results of a Real-World Clinical Experience in Italy

Angelo Baldassare Cefalu', Antonina Giammanco, Maurizio Averna, Patrizia Suppressa, Paolo Calabrò, Francesco Sbrana, Giovanni Battista Vigna, Cesare Sirtori, Laura D’Erasmo, Chiara Pavanello, Paolo Medde, Federico Bigazzi, Paolo Pintus, Tiziana Sampietro, Fulvio Sileo, Francesco Natale, Carlo Sabbà, Laura Calabresi, Marcello Arca, Marco BucciKatia Bonomo, Davide Noto

Risultato della ricerca: Article

18 Citazioni (Scopus)

Abstract

Introduction: Homozygous familial hypercholesterolaemia (HoFH) is a rare form of inherited dyslipidemia resistant to conventional cholesterol-lowering medications so that lipoprotein apheresis (LA) is usually required. Lomitapide has been approved for the treatment of HoFH. The aim of this study was to evaluate the benefits of lomitapide in HoFH patients followed with the usual clinical care. Methods: Clinical and biochemical data were retrospectively collected in 15 HoFH patients (10 with mutations in the LDLR gene and 5 in the LDLRAP1 gene) treated for at least 6 months with lomitapide in addition to lipid-lowering therapies (LLT) in different Lipid Clinics across Italy. Results: The mean follow-up period was 32.3 ± 29.7 months. During background therapies, HoFH patients showed a mean LDL-C level of 426.0 ± 204.0 mg/dl. The addition of lomitapide at the average dosage of 19 mg/day lowered LDL-C levels by 68.2 ± 24.8%. At their last visit, 60% of patients showed LDL-C <100 mg/dl and 46.6% <70 mg/dl. During follow-up, 8 of 10 patients receiving LA (80%) stopped this treatment due to marked LDL-C reduction. A wide range (13â95%) of individual LDL-C reduction was observed, but this was not related to genotype. During follow-up, 53.3% of patients reported at least one episode of diarrhea, but none was referred as severe; none had liver transaminase >5àULN or had to stop treatment due to side effects. A subset of patients was evaluated by liver ultrasound and fibroscan (n = 5) or nuclear magnetic resonance with spectroscopy (MRS) (n = 1) not showing clinical evidence of liver damage. Conclusion: In this real-world experience, lomitapide was confirmed to be a very powerful cholesterol-lowering agent in HoFH showing a good safety profile.
Lingua originaleEnglish
pagine (da-a)1200-1210
Numero di pagine11
RivistaAdvances in Therapy
Volume34
Stato di pubblicazionePublished - 2017

All Science Journal Classification (ASJC) codes

  • Pharmacology (medical)

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    Cefalu', A. B., Giammanco, A., Averna, M., Suppressa, P., Calabrò, P., Sbrana, F., Vigna, G. B., Sirtori, C., D’Erasmo, L., Pavanello, C., Medde, P., Bigazzi, F., Pintus, P., Sampietro, T., Sileo, F., Natale, F., Sabbà, C., Calabresi, L., Arca, M., ... Noto, D. (2017). Efficacy of Lomitapide in the Treatment of Familial Homozygous Hypercholesterolemia: Results of a Real-World Clinical Experience in Italy. Advances in Therapy, 34, 1200-1210.