Contemporary lipid-lowering management and risk of cardiovascular events in homozygous familial hypercholesterolaemia: insights from the Italian LIPIGEN Registry

被引:4
|
作者
D'Erasmo, Laura [1 ]
Bini, Simone [1 ]
Casula, Manuela [1 ,2 ,3 ]
Gazzotti, Marta [4 ]
Bertolini, Stefano [5 ]
Calandra, Sebastiano [6 ]
Tarugi, Patrizia [7 ]
Averna, Maurizio [8 ,9 ]
Iannuzzo, Gabriella [10 ]
Fortunato, Giuliana [11 ,12 ]
Catapano, Alberico L. [2 ,3 ]
Arca, Marcello [1 ,13 ]
机构
[1] Sapienza Univ Rome, Dept Translat & Precis Med, Viale Univ 37, I-00185 Rome, Italy
[2] IRCCS MultiMed, Milan, Italy
[3] Univ Milan, Dept Pharmacol & Biomol Sci, Epidemiol & Prevent Pharmacol Serv SEFAP, Milan, Italy
[4] SISA Fdn, Via Balzaretti 7, I-20133 Milan, Italy
[5] Univ Genoa, Dept Internal Med, Genoa, Italy
[6] Univ Modena & Reggio Emilia, Dept Biomed Metab & Neural Sci, Modena, Italy
[7] Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy
[8] Univ Palermo, Dept Hlth Promot Mother & Child Care, Internal Med, Palermo, Italy
[9] Univ Palermo, Med Specialties G DAlessandro PROMISE, Palermo, Italy
[10] Federico II Univ Naples, Dept Clin Med & Surg, Naples, Italy
[11] Federico II Univ Naples, Dept Mol Med & Med Biotechnol, Naples, Italy
[12] CEINGE SCarl Adv Biotechnol, Naples, Italy
[13] Azienda Osped Univ Policlin Umberto I, Rome, Italy
关键词
Homozygous familial hypercholesterolaemia; Lipid-lowering therapies; Real-world; PCSK9; inhibitors; Lomitapide; Evinacumab; Cardiovascular risk; INHIBITION; UPDATE;
D O I
10.1093/eurjpc/zwae036
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Aims The availability of novel lipid-lowering therapies (LLTs) has remarkably changed the clinical management of homozygous familial hypercholesterolaemia (HoFH). The impact of these advances was evaluated in a cohort of 139 HoFH patients followed in a real-world clinical setting. Methods and results The clinical characteristics of 139 HoFH patients, along with information about LLTs and low-density lipoprotein cholesterol (LDL-C) levels at baseline and after a median follow-up of 5 years, were retrospectively retrieved from the records of patients enrolled in the LIPid transport disorders Italian GEnetic Network-Familial Hypercholesterolaemia (LIPIGEN-FH) Registry. The annual rates of major atherosclerotic cardiovascular events (MACE-plus) during follow-up were compared before and after baseline. Additionally, the lifelong survival free from MACE-plus was compared with that of the historical LIPIGEN HoFH cohort. At baseline, LDL-C level was 332 +/- 138 mg/dL. During follow-up, the potency of LLTs was enhanced and, at the last visit, 15.8% of patients were taking quadruple therapy. Consistently, LDL-C decreased to an average value of 124 mg/dL corresponding to a 58.3% reduction (P-t < 0.001), with the lowest value (similar to 90 mg/dL) reached in patients receiving proprotein convertase subtilisin/kexin type 9 inhibitors and lomitapide and/or evinacumab as add-on therapies. The average annual MACE-plus rate in the 5-year follow-up was significantly lower than that observed during the 5 years before baseline visit (21.7 vs. 56.5 per 1000 patients/year; P = 0.0016). Conclusion Our findings indicate that the combination of novel and conventional LLTs significantly improved LDL-C control with a signal of better cardiovascular prognosis in HoFH patients. Overall, these results advocate the use of intensive, multidrug LLTs to effectively manage HoFH.
引用
收藏
页码:1038 / 1047
页数:10
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