Long-term efficacy of lipoprotein apheresis and lomitapide in the treatment of homozygous familial hypercholesterolemia (HoFH): a cross-national retrospective survey

Autor: Tiziana Sampietro, Marcello Arca, Paolo Calabrò, Olga Kalmykova, Livia Pisciotta, Stefano Bertolini, Gabriella Iannuzzo, Francesco Sbrana, Fabio Nota, Antonio Gallo, Carlo Sabbà, Patrizia Suppressa, Arturo Cesaro, Giuliana Fortunato, Randa Bittar, Arturo Puja, Marco Bucci, Katia Bonomo, Angelo B. Cefalù, Samir Saheb, Maurizio Averna, Tiziana Montalcini, Alessio Buonaiuto, Sergio D'Addato, Alain Carrié, Chiara Pavanello, Sophie Béliard, Alessia Di Costanzo, S. Palmisano, Eric Bruckert, Giovanni Battista Vigna, Antonina Giammanco, Laura Calabresi, Fabio Fimiani, Laura D'Erasmo
Přispěvatelé: D'Erasmo L., Gallo A., Cefalu A.B., Di Costanzo A., Saheb S., Giammanco A., Averna M., Buonaiuto A., Iannuzzo G., Fortunato G., Puja A., Montalcini T., Pavanello C., Calabresi L., Vigna G.B., Bucci M., Bonomo K., Nota F., Sampietro T., Sbrana F., Suppressa P., Sabba C., Fimiani F., Cesaro A., Calabro P., Palmisano S., D'Addato S., Pisciotta L., Bertolini S., Bittar R., Kalmykova O., Beliard S., Carrie A., Arca M., Bruckert E., D'Erasmo, L., Gallo, A., Cefalu, A. B., Di Costanzo, A., Saheb, S., Giammanco, A., Averna, M., Buonaiuto, A., Iannuzzo, G., Fortunato, G., Puja, A., Montalcini, T., Pavanello, C., Calabresi, L., Vigna, G. B., Bucci, M., Bonomo, K., Nota, F., Sampietro, T., Sbrana, F., Suppressa, P., Sabba, C., Fimiani, F., Cesaro, A., Calabro, Paolo, Palmisano, S., D'Addato, S., Pisciotta, L., Bertolini, S., Bittar, R., Kalmykova, O., Beliard, S., Carrie, A., Arca, M., Bruckert, E., Calabro, P., Università degli Studi di Roma 'La Sapienza' = Sapienza University [Rome] (UNIROMA), CHU Pitié-Salpêtrière [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Sorbonne Université (SU), Laboratoire d'Imagerie Biomédicale [Paris] (LIB), Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Università degli studi di Palermo - University of Palermo, University of Naples Federico II = Università degli studi di Napoli Federico II, CEINGE - Biotecnologie Avanzate, Università degli Studi 'Magna Graecia' di Catanzaro = University of Catanzaro (UMG), Università degli Studi di Milano = University of Milan (UNIMI), Università degli Studi di Ferrara = University of Ferrara (UniFE), Università degli studi 'G. d'Annunzio' Chieti-Pescara [Chieti-Pescara] (Ud'A), Ospedale San Luigi Gonzaga, Fondazione Toscana Gabriele Monasterio, University of the Study of Campania Luigi Vanvitelli, Policlinico S. Orsola-malpighi, Alma Mater Studiorum Università di Bologna [Bologna] (UNIBO)-Servizio sanitario regionale Emilia-Romagna, Università degli studi di Genova = University of Genoa (UniGe), Ospedale Policlinico San Martino [Genoa], Unité de Recherche sur les Maladies Cardiovasculaires, du Métabolisme et de la Nutrition = Research Unit on Cardiovascular and Metabolic Diseases (ICAN), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Institut de Cardiométabolisme et Nutrition = Institute of Cardiometabolism and Nutrition [CHU Pitié Salpêtrière] (IHU ICAN), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-CHU Pitié-Salpêtrière [AP-HP], Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research (C2VN), Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE), Hôpital de la Conception [CHU - APHM] (LA CONCEPTION)
Rok vydání: 2021
Předmět:
Zdroj: Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, 2021, 16 (1), pp.381. ⟨10.1186/s13023-021-01999-8⟩
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-12 (2021)
ISSN: 1750-1172
DOI: 10.1186/s13023-021-01999-8
Popis: Abstract Background Homozygous familial hypercholesterolemia (HoFH) is a rare life-threatening condition that represents a therapeutic challenge. The vast majority of HoFH patients fail to achieve LDL-C targets when treated with the standard protocol, which associates maximally tolerated dose of lipid-lowering medications with lipoprotein apheresis (LA). Lomitapide is an emerging therapy in HoFH, but its place in the treatment algorithm is disputed because a comparison of its long-term efficacy versus LA in reducing LDL-C burden is not available. We assessed changes in long-term LDL-C burden and goals achievement in two independent HoFH patients’ cohorts, one treated with lomitapide in Italy (n = 30) and the other with LA in France (n = 29). Results The two cohorts differed significantly for genotype (p = 0.004), baseline lipid profile (p padj = 0.004). Yearly on-treatment LDL-C padj = 0.002). A trend towards fewer total cardiovascular events was observed in the Lomitapide than in the LA cohort. Conclusions In comparison with LA, lomitapide appears to provide a better control of LDL-C in HoFH. Further studies are needed to confirm this data and establish whether this translates into a reduction of cardiovascular risk.
Databáze: OpenAIRE
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