Early lipoprotein apheresis yields long-term heart benefits in young patients with HoFH
Initiating lipoprotein apheresis in childhood or adolescence for patients with homozygous familial hypercholesterolaemia (HoFH) significantly improves long-term cardiovascular outcomes compared to using lipid-lowering drugs alone, according to a study. This early intervention leads to greater reductions in LDL cholesterol levels, longer atherosclerotic cardiovascular disease-free survival, and lower rates of cardiovascular death.
The study, utilizing data from the HoFH International Clinical Collaboration (HICC) and the CHAIN registry, included 404 patients diagnosed with HoFH before the age of 18. Patients were divided into 2 groups: those who began lipoprotein apheresis alongside lipid-lowering drugs (lipoprotein apheresis group) and those who received only pharmacotherapy (pharmacotherapy-only group). These groups were matched by sex and untreated plasma LDL cholesterol levels to ensure comparable baselines.
Findings demonstrated that patients in the lipoprotein apheresis group experienced a greater reduction in LDL cholesterol levels (-55% vs -31%) compared to the pharmacotherapy-only group. Importantly, the lipoprotein apheresis group also showed longer atherosclerotic cardiovascular disease-free survival and longer cardiovascular death-free survival. Cardiovascular death rates were significantly lower in the lipoprotein apheresis group (1% vs. 8%).
However, the median age for undergoing coronary artery bypass grafting was lower in the lipoprotein apheresis group (15 years vs 30.5 years), suggesting a complex interplay between intensive treatment and disease progression, according to the study authors.
Reference
Reijman MD, Tromp TR, Hutten BA, et al; Homozygous Familial Hypercholesterolaemia International Clinical Collaborators (HICC); Children with Homozygous Hypercholesterolemia on Lipoprotein Apheresis: an International Registry (CHAIN) consortia. Cardiovascular outcomes in patients with homozygous familial hypercholesterolaemia on lipoprotein apheresis initiated during childhood: long-term follow-up of an international cohort from two registries. Lancet Child Adolesc Health. 2024;S2352-4642(24)00073-7. doi: 10.1016/S2352-4642(24)00073-7. Epub ahead of print. PMID: 38759658.
