Guillermo Villa1, Bruce Wong2, Lucie Kutikova3, Kausik K Ray4, Pedro Mata5, Eric Bruckert6. 1. Economic Modeling, Center of Excellence, Amgen (Europe) GmbH, Dammstrasse 23, PO Box 1557, Zug, CH-6301, Switzerland. 2. Centre for Clinical Epidemiology and Biostatistics, University of Pennsylvania, 423 Guardian Drive, Pennsylvania, PA 19104-6021, USA. 3. Global Health Economics, Amgen [Europe] GmbH, Dammstrasse 23, PO Box 1557, CH-6301, Zug, Switzerland. 4. School of Public Health Imperial College London, South Kensington Campus, London, SW7 2AZ, UK. 5. Fundación Hipercolesterolemia Familiar, General ,varez de Castro, 14 1°E, Madrid, 28010 Spain. 6. Endocrinology - Metabolism and Prevention of Cardiovascular Diseases, Hôpital Pitié Salpêtrière, 47-83 Boulevard de l'Hôpital, Paris, 75651, France.
Abstract
Aims: Patients with familial hypercholesterolaemia (FH) have an elevated cardiovascular (CV) risk. The objective of this analysis was to adjust CV risk equations derived in non-FH populations with hyperlipidaemia to predict CV risk in FH patients, and then to use these adjusted CV risk equations in a decision analytic model in order to predict lifetime CV risk in FH patients. Methods and results: A literature search of publications reporting CV risk in FH patients identified the publication with the most credible estimate of CV risk increase. A CV event rate ratio (RR) (FH vs. non-FH) was derived from reported odds ratios by pooling treated and untreated patients. Predicted CV event risks based on non-FH risk equations were adjusted with the RR to reflect CV risk in FH patients. A decision analytic model incorporating these adjusted risk equations was used to predict 10-year and lifetime CV risk in FH patients. Combining the derived RR of 7.1 (95% CI: 5.7-8.7) with the predicted CV risks in a decision analytic model yielded 10-year and lifetime risk estimates of 45% and 88% in FH patients based on the RUTHERFORD-2 trial population. Based on the initial (cross-sectional) RR of 7.1, FH patients were predicted to have 3.9 times more events over their lifetime than non-FH patients with a similar risk profile. Conclusion: The CV risk in FH is high and represents an unmet medical need for patients. Increased efforts for better diagnosis and management of FH should be employed to improve patient outcomes. Published on behalf of the European Society of Cardiology. All rights reserved.
Aims: Patients with familial hypercholesterolaemia (FH) have an elevated cardiovascular (CV) risk. The objective of this analysis was to adjust CV risk equations derived in non-FH populations with hyperlipidaemia to predict CV risk in FHpatients, and then to use these adjusted CV risk equations in a decision analytic model in order to predict lifetime CV risk in FHpatients. Methods and results: A literature search of publications reporting CV risk in FHpatients identified the publication with the most credible estimate of CV risk increase. A CV event rate ratio (RR) (FH vs. non-FH) was derived from reported odds ratios by pooling treated and untreated patients. Predicted CV event risks based on non-FH risk equations were adjusted with the RR to reflect CV risk in FHpatients. A decision analytic model incorporating these adjusted risk equations was used to predict 10-year and lifetime CV risk in FHpatients. Combining the derived RR of 7.1 (95% CI: 5.7-8.7) with the predicted CV risks in a decision analytic model yielded 10-year and lifetime risk estimates of 45% and 88% in FHpatients based on the RUTHERFORD-2 trial population. Based on the initial (cross-sectional) RR of 7.1, FHpatients were predicted to have 3.9 times more events over their lifetime than non-FHpatients with a similar risk profile. Conclusion: The CV risk in FH is high and represents an unmet medical need for patients. Increased efforts for better diagnosis and management of FH should be employed to improve patient outcomes. Published on behalf of the European Society of Cardiology. All rights reserved.
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