Successful treatment of homozygous familial hypercholesterolemia using cascade filtration plasmapheresis

Abstract

Objective: The aim of this study was to report the efficacy of low-density lipoprotein cholesterol (LDL-C) apheresis using a cascade filtration system in pediatric patients with homozygous familial hypercholesterolemia (FH), and to clarify the associated adverse effects and difficulties. Material and Methods: LDL-C apheresis using a cascade filtration system was performed in 3 pediatric patients with homozygous FH; in total, 120 apheresis sessions were performed. Results: Cascade filtration therapy significantly reduced the mean LDL-C values from 418 ± 62 mg/dL to 145 ± 43 mg/ dL (p= 0.011). We observed an acute mean reduction in the plasma level of total cholesterol (57.9%), LDL-C (70.8%), and high-density lipoprotein cholesterol (HDL-C) (40.7%). Treatments were well tolerated. The most frequent clinical adverse effects were hypotension in 3 sessions (2.5%), chills (1.7%) in 2 sessions, and nausea/vomiting in 3 sessions (2.5%). Conclusion: Our experience using the cascade filtration system with 3 patients included good clinical outcomes and laboratory findings, safe usage, and minor adverse effects and technical problems.