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