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

Laura D’erasmo 1, 2, 3 Antonio Gallo 2, 4 Angelo Baldassare Cefalù 5 Alessia Di Costanzo 1 Samir Saheb 2, 3 Antonina Giammanco 5 Maurizio Averna 5 Alessio Buonaiuto 6 Gabriella Iannuzzo 6 Giuliana Fortunato 6, 7 Arturo Puja 8 Tiziana Montalcini 8 Chiara Pavanello 9 Laura Calabresi 9 Giovanni Battista Vigna 10 Marco Bucci 11 Katia Bonomo 12 Fabio Nota 12 Tiziana Sampietro 13 Francesco Sbrana 13 Patrizia Suppressa Carlo Sabbà Fabio Fimiani 14 Arturo Cesaro 14 Paolo Calabrò 14 Silvia Palmisano 15 Sergio D’addato 15 Livia Pisciotta 16, 17 Stefano Bertolini 16, 17 Randa Bittar 18, 2 Olga Kalmykova 2, 3 Sophie Béliard 19, 20 Alain Carrié 18, 2 Marcello Arca 1 Eric Bruckert 2, 3 
Abstract : 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 < 0.001), age of treatment initiation (p < 0.001), occurrence of cardiovascular disease (p = 0.003) as well as follow-up duration (p < 0.001). The adjunct of lomitapide to conventional lipid-lowering therapies determined an additional 58.0% reduction of last visit LDL-C levels, compared to 37.1% when LA was added ( p adj = 0.004). Yearly on-treatment LDL-C < 70 mg/dl and < 55 mg/dl goals were only achieved in 45.5% and 13.5% of HoFH patients treated with lomitapide. The long-term exposure to LDL-C burden was found to be higher in LA than in Lomitapide cohort (13,236.1 ± 5492.1 vs. 11,656.6 ± 4730.9 mg/dL-year respectively, p adj = 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.
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Submitted on : Wednesday, April 27, 2022 - 4:47:28 PM
Last modification on : Friday, November 25, 2022 - 7:04:09 PM

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Laura D’erasmo, Antonio Gallo, Angelo Baldassare Cefalù, Alessia Di Costanzo, Samir Saheb, et al.. Long-term efficacy of lipoprotein apheresis and lomitapide in the treatment of homozygous familial hypercholesterolemia (HoFH): a cross-national retrospective survey. Orphanet Journal of Rare Diseases, 2021, 16 (1), pp.381. ⟨10.1186/s13023-021-01999-8⟩. ⟨hal-03653394⟩



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