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Table 2 Follow-up lipid profile, lipid lowering therapies and MACE in HoFH cohorts

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

  Lomitapide cohort (N = 30) LA cohort
(N = 29)
p
Duration of follow-up (years) 2.2 (1.0–3.7) 16.5 (9.0–25.5)  < 0.001
Risk factors, n (%)
 Smoking 2 (6.7) 7 (24.1) 0.006
 T2DM 1 (3.3) 0 NS
 HTN 11 (36.7) 7 (24.1) NS
Plasma lipids (mg/dl)
 On-treatment LDL-C 111.4 (83.7–177.8) 247.2 (217.9–340.8)  < 0.001
 On-treatment LDL-C burden (mg/dL-year) 293.0 (153.0–454.8) 3849.3 (2238.3–7045.5)  < 0.001
 Last visit TC 169.0 (126.2 – 276.7) 390.5 (321.9 – 500.0)  < 0.001
 Last visit HDL-C 47.5 (38.7 – 60.0) 30.6 (19.7 – 40.8) 0.001
 Last visit LDL-C 114.0 (64.7 – 202.95) 340.5 (280.5 – 418.8)  < 0.001
 Last visit TG 58.0 (35.0 – 81.0) 92.7 (65.7 – 152.9) 0.001
LLT at last visit, n (%)
 LA 0 29 (100)  < 0.001
 PCKS9i 5 (16.7) 1 (3.4) NS
 Statin 29 (96.7) 27 (93.1) NS
 Ezetimibe 28 (93.3) 27 (93.1) NS
 Fibrate 0 0 -
 Porto-caval shunt 0 1 (3.4) NS
 Resins 0 0 -
Incident MACE
 Cumulative number of MACE 7 42 -
 Individual MACE event, n (%) 4 (13.3) 16 (55.2) 0.001
CHD 3 (10.0) 11 (37.9) 0.012
Stroke 0 2 (6.9) NS
PAD 0 14 (48.3) 0.001
Carotid revascularization 1 (3.3) 2 (6.9) NS
Aortic Valve replacement 1 (3.3) 6 (22.2) 0.048
Death for cardiovascular disease 1 (3.3) 4 (14.3) NS
Death for other reason 0 1 (3.6) NS
Number of MACE per patient, n (%)    
0.003
 0 26 (86.7) 13 (44.8)
 1 3 (6.7) 6 (20.7)
 ≥ 2 2 (6.7) 10 (34.5)
IR per 1000 person/years 77.6 86.9 -
  1. Data are represented as median (interquartile range), mean (standard deviation) or number (percentage) as appropriate. For estimation of incident MACE and Incident Rate of incident MACE (× 1000 person-years) please refer to Methods. Duration of follow-up has been calculated as the difference between the date of the last and the baseline visit with LA or lomitapide. On-treatment LDL-C burden was calculated as: \(({LDL-C}_{follow-up}*{Years}_{follow-up})\)
  2. T2DM, type 2 diabetes; HTN, hypertension; LDL-C, low density lipoprotein cholesterol; TC, total cholesterol; HDL-C, high density lipoprotein cholesterol; TG, triglycerides; LLT, Lipid Lowering Therapy; LA, Lipoprotein apheresis; PCKS9i, Proprotein convertase subtilisin/kexin type 9 inhibitors; MACE, Major Atherosclerotic Cardiovascular Events; CHD, coronary heart disease; PAD, peripheral artery disease; IR, incident rate; NS, not significant