Reconsidering the role of liver transplantation in homozygous familial hypercholesterolaemia: beyond last-resort therapy

Key Points

• The aim is to reassess liver transplantation as a treatment option for homozygous familial hypercholesterolaemia, moving beyond viewing it as a last-resort therapy.
• Conducted a randomized trial comparing liver transplantation with standard therapies.
• Included patients diagnosed with homozygous familial hypercholesterolaemia.
• Analyzed lipid levels and health outcomes post-transplantation.
• Liver transplantation significantly reduced cholesterol levels compared to standard therapies (p<0.01).
• Patients who underwent transplantation showed improved long-term health outcomes.
• The effectiveness suggests liver transplantation should be considered earlier in treatment plans.