Table 1.
Patient demographics and baseline clinical characteristics
| Evinacumab-naïve (n = 46) | Evinacumab-continue (n = 70) | Total (n = 116) | |
|---|---|---|---|
| Age, years, mean (SD) | 35.2 (16.5) | 41.2 (15.2) | 38.8 (15.9) |
| Male, n (%) | 26 (56.5) | 33 (47.1) | 59 (50.9) |
| Race, n (%) | |||
| White | 31 (67.4) | 49 (70.0) | 80 (69.0) |
| Black or African American | 2 (4.3) | 2 (2.9) | 4 (3.4) |
| Asian | 3 (6.5) | 9 (12.9) | 12 (10.3) |
| Not reported | 7 (15.2) | 4 (5.7) | 11 (9.5) |
| Other | 3 (6.5) | 6 (8.6) | 9 (7.8) |
| BMI, kg/m2, mean (SD) | 24.5 (5.2) | 26.4 (6.1) | 25.7 (5.8) |
| HoFH diagnosis, n (%) | |||
| Genotyping | 33 (71.7) | 52 (74.3) | 85 (73.3) |
| Clinical diagnosis | 13 (28.3) | 18 (25.7) | 31 (26.7) |
| Genotype state, n (%) | |||
| Homozygous | 22 (47.8) | 33 (47.1) | 55 (47.4) |
| Compound heterozygous | 18 (39.1) | 23 (32.9) | 41 (35.3) |
| Double heterozygous | 0 | 1 (1.4) | 1 (0.9) |
| Other (heterozygous or undetermined) | 5 (10.9) | 12 (17.1) | 17 (14.7) |
| No mutations | 0 | 1 (1.4) | 1 (0.9) |
| Missing | 1 (2.2) | 0 | 1 (0.9) |
| Baseline LLT, n (%) | |||
| Statin | 44 (95.7) | 63 (90.0) | 107 (92.2) |
| High-intensity statina | 37 (80.4) | 55 (78.6) | 92 (79.3) |
| Ezetimibe | 36 (78.3) | 57 (81.4) | 93 (80.2) |
| PCSK9 inhibitorb | 22 (47.8) | 50 (71.4) | 72 (62.1) |
| Lomitapide | 8 (17.4) | 14 (20.0) | 22 (19.0) |
| Lipoprotein apheresis | 22 (47.8) | 23 (32.9) | 45 (38.8) |
| Calculated LDL-C, mmol/L, mean (SD)c | 7.0 (3.2) | 6.6 (4.7) | 6.8 (4.1) |
| Apolipoprotein B, g/L, mean (SD) | 1.8 (0.7) | 1.7 (0.9) | 1.7 (0.8) |
| HDL-C, mmol/L, mean (SD) | 1.1 (0.4) | 1.1 (0.4) | 1.1 (0.4) |
| Non-HDL-C, mmol/L, mean (SD) | 7.5 (3.1) | 7.2 (4.8) | 7.3 (4.2) |
| Total cholesterol, mmol/L, mean (SD) | 8.6 (3.0) | 8.3 (4.7) | 8.4 (4.1) |
| Fasting triglycerides, mmol/L, median (Q1, Q3) | 1.0 (0.7, 1.6) | 1.0 (0.6, 1.6) | 1.0 (0.7, 1.6) |
| Lipoprotein(a), nmol/L, median (Q1, Q3) | 50.0 (23.0, 113.0) | 73.5 (32.0, 168.0) | 66.0 (28.0, 166.0) |
BMI, body mass index; HDL-C, HDL cholesterol; LDL-C, LDL cholesterol; LLT, lipid-lowering therapy; PCSK9, proprotein convertase subtilisin/kexin type 9.
aHigh-intensity statin corresponds to atorvastatin 40 or 80 mg daily or rosuvastatin 20–40 mg daily.
bAlirocumab or evolocumab.
cLDL-C was calculated using the Friedewald formula.