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Table 3 Rates for MACE from recent data sources in HoFH

From: Target achievement and cardiovascular event rates with Lomitapide in homozygous Familial Hypercholesterolaemia

 

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

  1. LDL-C low-density lipoprotein cholesterol, NA not applicable, NR not reported [7,8,9]
  2. 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 [7,8,9]
  3. 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]