Table 1.
Baseline characteristics (randomized population)
| FH I |
FH II |
|||
|---|---|---|---|---|
| Alirocumab (n = 323) | Placebo (n = 163) | Alirocumab (n = 167) | Placebo (n = 82) | |
| Diagnosis of heterozygous familial hypercholesterolaemia, n (%) | ||||
| Genotyping | 129 (39.9) | 62 (38.0) | 117 (70.1) | 66 (80.5) |
| Clinical criteriaa | 193 (59.8) | 101 (62.0) | 50 (29.9) | 16 (19.5) |
| Age, years, mean ± SD | 52.1 ± 12.9 | 51.7 ± 12.3 | 53.2 ± 12.9 | 53.2 ± 12.5 |
| Male, n (%) | 180 (55.7) | 94 (57.7) | 86 (51.5) | 45 (54.9) |
| Race, White, n (%) | 300 (92.9) | 144 (88.3) | 164 (98.2) | 80 (97.6) |
| Body mass index, kg/m2, mean ± SD | 29.0 ± 4.6 | 30.0 ± 5.4 | 28.6 ± 4.6 | 27.7 ± 4.7 |
| Coronary heart disease, n (%)b | 147 (45.5) | 78 (47.9) | 58 (34.7) | 31 (37.8) |
| Coronary heart disease risk equivalents, n (%)c | 54 (16.7) | 25 (15.3) | 15 (9.0) | 4 (4.9) |
| Current smoker, n (%) | 39 (12.1) | 30 (18.4) | 36 (21.6) | 13 (15.9) |
| Hypertension, n (%) | 139 (43.0) | 71 (43.6) | 57 (34.1) | 24 (29.3) |
| Type 2 diabetes, n (%)d | 32 (9.9) | 25 (15.3) | 7 (4.2) | 3 (3.7) |
| Statin use, n (%) | 323 (100) | 163 (100) | 167 (100) | 82 (100) |
| High-dose statin use, n (%)e | 267 (82.7) | 139 (85.3) | 145 (86.8) | 75 (91.5) |
| Ezetimibe use | 181 (56.0) | 97 (59.5) | 112 (67.1) | 53 (64.6) |
FH I was conducted in North America, Europe, and South Africa; FH II was conducted in Europe. P-values comparing baseline data between treatment groups were calculated for descriptive purposes, and no difference was significant at the 5% level.
aOne patient (randomized to the alirocumab arm in FH I) was characterized using clinical (World Health Organization) criteria as ‘probable’ HeFH rather than ‘certain’; however, subsequent genotyping failed to identify a mutation in the LDLR or APOB genes; rare mutations cannot be excluded. The patient was considered as having HeFH by the investigator.
bAcute myocardial infarction, silent myocardial infarction, unstable angina, or coronary revascularization.
cIschaemic stroke, peripheral arterial disease, moderate chronic kidney disease (estimated glomerular filtration rate of ≥30 and ≤60 mL/min/1.73 m2), and diabetes with two or more risk factors.
dBased on medical history.
eAtorvastatin 40–80 mg, rosuvastatin 20–40 mg, or simvastatin 80 mg daily.