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Prescribing Patterns of Evolocumab and Alirocumab in Patients With Familial Hypercholesterolemia: A Cross-Sectional
Efeturi M Okorigba1, Kehinde H Jinadu2, Nneka Muoghalu3,4
1Internal Medicine, West Virginia University, Morgantown, USA.
Insights
Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor prescribing for familial hypercholesterolemia (FH) was rare in U.S. ambulatory visits. This indicates a significant gap between clinical guidelines and actual practice, necessitating broader adoption strategies.
Area of Science:
- Cardiovascular Medicine
- Genetics and Hereditary Diseases
- Pharmacology
Background:
- Familial hypercholesterolemia (FH) is a genetic disorder causing significantly elevated cardiovascular risk.
- Despite established guidelines, real-world utilization of PCSK9 inhibitors in FH patients remains unclear.
Purpose of the Study:
- To assess national trends in PCSK9 inhibitor prescriptions for adult FH patients in U.S. ambulatory care settings.
- To analyze prescribing patterns between 2018 and 2019.
Main Methods:
- Cross-sectional analysis of the 2018-2019 National Ambulatory Medical Care Survey (NAMCS).
- Included adults (≥18 years) with FH diagnosis (ICD-10-CM E78.0).
- Examined PCSK9 inhibitor prescriptions (evolocumab, alirocumab) and associated patient/physician factors.
Main Results:
- PCSK9 inhibitor prescriptions were documented in only 0.3% of 20.4 million FH visits.
- Prescribing occurred predominantly in older, White females within primary care settings.
- Older age was significantly associated with higher odds of PCSK9 inhibitor prescription (OR 1.04 per year).
Conclusions:
- PCSK9 inhibitor use for FH in U.S. ambulatory care is notably infrequent.
- A substantial disparity exists between evidence-based recommendations and clinical application.
- Strategies are required to improve PCSK9 inhibitor adoption for better lipid management and cardiovascular risk reduction in FH.
Background:
Familial hypercholesterolemia (FH) is a hereditary lipid disorder that substantially elevates cardiovascular risk. Despite strong evidence and guideline recommendations, the real-world use of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors in FH remains uncertain.
Objective:
This study aimed to evaluate national trends in PCSK9 inhibitor prescribing during ambulatory visits for adults with FH in the United States from 2018 to 2019.
Methods:
We conducted a cross-sectional analysis of ambulatory care visits recorded in the 2018-2019 National Ambulatory Medical Care Survey (NAMCS), a nationally representative survey of office-based physicians in the United States. Eligible visits included adults (≥18 years) with a diagnosis of FH (International Classification of Diseases, 10th Revision, Clinical Modification (ICD-10-CM) code E78.0). The primary outcome was prescription of a PCSK9 inhibitor (evolocumab or alirocumab), identified through medication codes. Patient demographics, insurance type, and physician specialty were examined. Survey weights were applied to generate national estimates, and logistic regression was used to explore the association between age and prescribing.
Results:
Among the weighted total of 20,414,210 visits for adults with FH, only 69,672 visits (0.3%) included a prescription for a PCSK9 inhibitor. Prescribing was observed exclusively among female, White patients and occurred almost entirely in primary care settings. Patients receiving PCSK9 therapy were older than those not prescribed (mean 71.0 vs. 66.5 years, p=0.004). No prescribing was observed among males, racial/ethnic minority groups, or patients seen in surgical or medical specialties. In survey-weighted logistic regression, each additional year of age was associated with a 4% higher odds of PCSK9 prescribing (OR 1.04, 95% CI: 1.02-1.07, p=0.002).
Conclusion:
PCSK9 inhibitor use in FH patients during U.S. ambulatory visits was rare, highlighting a persistent gap between evidence-based guidelines and clinical practice. Broader adoption strategies are needed to optimize lipid management and reduce cardiovascular risk in FH.
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