Economic Modeling, Center of Excellence, Amgen (Europe) GmbH, Dammstrasse 23, PO Box 1557, Zug, CH-6301, Switzerland.
Centre for Clinical Epidemiology and Biostatistics, University of Pennsylvania, 423 Guardian Drive, Pennsylvania, PA 19104-6021, USA.
Eur Heart J Qual Care Clin Outcomes. 2017 Oct 1;3(4):274-280. doi: 10.1093/ehjqcco/qcx011.
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.
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.
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.
家族性高胆固醇血症（FH）患者的心血管（CV）风险升高。本分析的目的是调整来源于高脂血症非 FH 人群的 CV 风险方程，以预测 FH 患者的 CV 风险，然后在决策分析模型中使用这些调整后的 CV 风险方程，以预测 FH 患者的终生 CV 风险。
对发表的 FH 患者 CV 风险的文献进行检索，确定了最可信的 CV 风险增加估计值的出版物。通过对报告的比值比进行汇总，得出了 FH 患者与非 FH 患者之间 CV 事件发生率比（RR）。基于非 FH 风险方程预测的 CV 事件风险通过 RR 进行调整，以反映 FH 患者的 CV 风险。纳入这些调整后风险方程的决策分析模型用于预测 FH 患者的 10 年和终生 CV 风险。结合 RUTHERFORD-2 试验人群中得出的 RR（7.1，95%CI：5.7-8.7）与决策分析模型中预测的 CV 风险，FH 患者的 10 年和终生风险估计值分别为 45%和 88%。基于初始（横断面）RR（7.1），FH 患者预计在其一生中发生的事件比具有相似风险特征的非 FH 患者多 3.9 倍。
FH 的 CV 风险很高，这是 FH 患者未满足的医疗需求。应加大力度更好地诊断和管理 FH，以改善患者的预后。
