Adherence to pharmacotherapy: sine qua non for reducing cumulative risk of premature coronary disease in familial hypercholesterolemia

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Purpose of review
Familial hypercholesterolemia (FH) is a dominant and highly penetrant monogenic disorder present from birth that markedly elevates plasma low-density lipoprotein (LDL)-cholesterol concentration and, if untreated, leads to atherosclerotic cardiovascular disease (ASCVD). The risk of ASCVD can be substantially reduced with lipid-lowering treatment (LLT). However, adherence to LLT remains a major challenge in FH patients and an under-recognized issue. We review several barriers to treatment adherence and implementation strategies for improving adherence in patients with FH.

Recent findings
Barriers that negatively affect patient adherence to treatment include the misunderstanding of perceived and actual risk of FH and the benefits of LLT, inadequate knowledge, lack of standardization of treatment, insufficient monitoring of LDL-cholesterol level, and inequalities in healthcare resources. Education of patients, carers and healthcare providers, guideline-directed treatment goals, regular monitoring, medication regimen simplification and greater access to established and new drugs are crucial enablers for improving adherence to treatment. However, given FH is present from birth, strategies for life-long adherence from childhood or young adulthood is critically important and requires further study. To be effective, strategies should be multifaceted, targeted and patient-centred involving a multidisciplinary-team with support from family, communities and peer groups.

FH confers a significant risk for ASCVD from a young age. Achieving better medication adherence is foundational for improving clinical outcomes and reducing the burden of atherosclerosis over a lifetime. Identification of key barriers and enablers are critical for implementing better adherence to treatment across the life-course of patients with FH.
Original languageEnglish
Pages (from-to)22-30
Number of pages9
JournalCurrent Opinion in Endocrinology, Diabetes and Obesity
Issue number1
Publication statusPublished - 1 Feb 2024


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