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Homozygous Familial Hypercholesterolemia

Information for
Healthcare Professionals

LDL-C reduction is the main goal of HoFH management1

12x higher risk of death in HoFH patients with elevated serum cholesterol levels2

The extent of reduction of serum cholesterol achieved by a therapy is a major determinant of survival in patients with HoFH.2

In a retrospective study of 133 patients with HoFH, patients with an on-treatment serum cholesterol > 15.1 mmol/L were at a 12X higher risk of death from a cardiac cause than those with serum cholesterol < 8.1 mmol/L.*

*Retrospective survey of lipid levels and clinical outcomes of HoFH patients treated with a combination of lipid lowering measures between 1990 and 2014 in South Africa and the UK

2023 ESC/EAS LDL-C Reduction Target Recommendations

Recommendations from the 2023 EAS Consensus Statement for LDL-C target level3


<3.0 mmol/L

Adults (>18 years old)

< 1.8 mmol/L

Adults with ASCVD

< 1.4 mmol/L

HoFH is typically managed with multiple treatment approaches

Lifestyle intervention, including4

  • Low-saturated fat, low-cholesterol diet
  • Physical activity
  • Effective management of hypertension and diabetes
  • Smoking cessation

Pharmacologic treatments, plasmapheresis, and in rare circumstances for unresponsive patients, liver transplantation


1. Brunham L et al. Can J Cardiol. 2018;35:1553-1563; 2. Thompson GR et al. Eur Heart J. 2018;39:1162–1168; 3. Cuchel M et al. Eur Heart J. 2023;00:1-15. 4. Cuchel M et al. Eur Heart J. 2014;35:2146–2157.