Liver transplantation in children with familial hypercholesterolemia shows limited long-term success

Although liver transplantation (LT) can reduce cholesterol levels in children and adolescents with homozygous familial hypercholesterolemia (HoFH), it does not achieve target LDL levels or prevent cardiovascular disease, indicating that LT alone is not a curative treatment for the condition, according to a study.

The study included 5 boys, with an average age of 6.2 years, who underwent LT between 2007 and 2023.

Prior to transplantation, patients had an average total cholesterol level of 923 mg/dL and an LDL-cholesterol level of 864 mg/dL. After an average follow-up of 9.2 years, cholesterol levels decreased significantly, with an average total cholesterol of 197 mg/dL and LDL-cholesterol of 138 mg/dL. However, 80% of the patients developed cardiovascular disease, and 2 of them passed away due to related complications.

Reference
Yılmaz Dağlı H, Artan R, Civan DD, et al. Is Liver Transplantation Alone an Effective Treatment for Homozygotic Familial Hypercholesterolemia in Children? Pediatr Transplant. 2024;28(7):e14853. doi: 10.1111/petr.14853. PMID: 39282976.