Table 3.
Rates for MACE from recent data sources in HoFH
| HoFH background [8] | Mipomersen-treated HoFH [8] | Lomitapide-treated [7] | Evolocumab treated [9] | |
|---|---|---|---|---|
| Number of patients | 23 | 23 | 19 | 106 |
| Mean age at baseline | 31 years | 30.7 years | 34 years | |
| Mean baseline LDL-C | 455 mg/dL | 336 mg/dL | 324 mg/dL | |
| Mean LDL-C between 6 and 12 months on treatmenta | NA | 331 mg/dL | 166 mg/dL | 286 mg/dL |
| Apheresis | NR | None | 62% | 32% |
| CVD at baseline | NR | NR | 93% | 51% |
| Number of major CV eventsb | 12 | 4 | 2 | 4 |
| Number of patient years | 46 | 35 | 98 | 185 |
| Annualized event rate | 26.1% | 11.4% | 2.0% | 2.1% |
| Events/1000 months | 21.7 | 9.5 | 1.7 | 1.8 |
LDL-C low-density lipoprotein cholesterol, NA not applicable, NR not reported [7–9]
aRange of follow-up based on data availability: 1-year data for mipomersen, 26-week data for lomitapide, 48-week data for evolocumab, calculated from % reduction reported; CVD, cardiovascular disease [7–9]
bMACE was defined as CV death, non-fatal myocardial infarction (MI), unstable angina pectoris and/or ischemic stroke, the evolocumab publication did not include a MACE definition; however, it is believed to be defined as death (CV or non CV), MI, UA, and coronary revascularisation as this is the definition used in the CHMP assessment report for evolocumab [10]