Ten years of lipoprotein apheresis for familial hypercholesterolemia in Malaysia: A creative approach by a cardiologist in a developing country

• Published in: Journal of clinical lipidology Vol. 10; no. 5; pp. 1188 - 1194
• Main Authors: Khoo, Kah Lin, MD, FRCP, FACC, FESC, FCCP, Page, Michael M., BPharm, MBBS(Hons), Liew, Yin Mei, MBBS, FAMM, FRCP, Defesche, Joep C., PhD, Watts, Gerald F., DSc, MD, PhD, FRCP, FRACP
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.09.2016
• Subjects: Adolescent; Adult; Anaphylaxis - etiology; Blood Component Removal - adverse effects; Blood Component Removal - methods; Cardiologists; Cardiovascular; Child; Coronary artery disease; Developing Countries; Familial hypercholesterolemia; Female; Humans; Hyperlipoproteinemia Type II - therapy; Lipoprotein apheresis; Malaysia; Male; Middle Aged; Quality of Life; Retrospective Studies; Service delivery; Young Adult; Service delivery; Malaysia; Familial hypercholesterolemia; Coronary artery disease; Lipoprotein apheresis
• ISSN: 1933-2874; 1876-4789
• DOI: 10.1016/j.jacl.2016.05.006

Background Familial hypercholesterolemia (FH) leads to premature coronary artery disease and aortic stenosis, with undertreated severe forms causing death at a young age. Lipoprotein apheresis (LA) is often required for lowering low-density lipoprotein cholesterol levels in severe FH. Objectives The objective of this study was to present the first experiences with LA in Malaysia, between 2004 and 2014. Methods We retrospectively collected data from patient records to assess the effectiveness, adverse effects, patient quality of life, and costs associated with an LA service for genetically confirmed homozygous and heterozygous FH. Results We treated 13 women and 2 men aged 6 to 59 years, 10 with homozygous and 5 with heterozygous FH, all on maximally tolerated cholesterol-lowering drug therapy, for a total of 65 patient-years. Acute lowering of low-density lipoprotein cholesterol post apheresis was 56.3 ± 7.2%, with time-averaged mean lowering of 34.9 ± 13.9%. No patients experienced any cardiovascular events during the period of receiving LA. Patients receiving LA experienced few side effects and enjoyed reasonable quality of life, but inability to continue treatment was frequent because of cost. Conclusion LA for severe FH can be delivered effectively in the short term in developing nations, but costs are a major barrier to sustaining this mode of treatment for this high-risk group of patients. New drug therapies for FH, such as the proprotein convertase subtilisin/kexin type 9 inhibitors, microsomal triglyceride transfer protein inhibitors, and apolipoprotein-B100 antisense oligonucleotides may allow improved care for these patients, but costs and long-term safety remain as issues to be addressed.