Casula Manuela, Scotti Lorenza, Tragni Elena, Merlino Luca, Corrao Giovanni, Catapano Alberico L
Epidemiology and Preventive Pharmacology Centre (SEFAP), Department of Pharmacological and Biomolecular Sciences, University of Milan, via Balzaretti 9, 20133 Milan, Italy.
Department of Statistics and Quantitative Methods, Division of Biostatistics, Epidemiology and Public Health, University of Milano-Bicocca, via Bicocca degli Arcimboldi 8, 20126 Milan, Italy.
Atherosclerosis. 2016 Nov;254:172-178. doi: 10.1016/j.atherosclerosis.2016.10.020. Epub 2016 Oct 12.
We aimed at describing the therapeutic approach in young adult patients diagnosed with heterozygous familial hypercholesterolemia (HeFH) and their adherence and persistence to treatment.
From regional administrative databases, individuals aged ≤40 years, who received exemption for HeFH between January 1, 2003 and December 31, 2011, and concomitantly started statin treatment, were identified. Within the first year of treatment, we evaluated therapeutic changes, adherence as MPR (medication possession ratio), persistence as continuous drug coverage without gaps ≥60 days, and influencing factors using log binomial models.
Of 1404 patients, 42.4% were initially treated with a high-efficacy statin. 23.4% of patients showed at least one treatment change. Mean MPR was 68.7% (29.9), and patients showing continued statin use were 47.0%. Therapy modification was significantly associated with a past cardiovascular event (relative risk, RR [95% confidential interval] 2.28 [1.69-3.09]) and at least one lipid test (RR 1.82 [1.31-2.53]). MPR ≥80% was significantly associated with the first statin prescribed (atorvastatin RR 1.28 [1.09-1.51] and rosuvastatin RR 1.21 [1.01-1.44], vs. simvastatin), a past cardiovascular event (RR 1.33 [1.12-1.59]), at least one therapy change (RR 1.28 [1.15-1.43]), at least a lipid test (RR 1.26 [1.07-1.49]). A similar pattern was observed for persistence.
This analysis of young adult HeFH patients showed that therapy change was quite frequent, and probably reflected adjustments according to individual response. Adherence and persistence were inadequate, even in this population at high cardiovascular risk, and they need to be improved through proper patient education and shared treatment decision-making approach.
我们旨在描述确诊为杂合子家族性高胆固醇血症（HeFH）的年轻成年患者的治疗方法以及他们对治疗的依从性和持续性。
从地区行政数据库中，识别出年龄≤40岁、在2003年1月1日至2011年12月31日期间因HeFH获得豁免且同时开始他汀类药物治疗的个体。在治疗的第一年内，我们评估了治疗变化、以药物持有率（MPR）衡量的依从性、以连续无间断药物覆盖≥60天衡量的持续性，并使用对数二项模型分析影响因素。
在1404例患者中，42.4%最初接受高效他汀类药物治疗。23.4%的患者至少有一次治疗变化。平均MPR为68.7%（29.9），持续使用他汀类药物的患者为47.0%。治疗调整与既往心血管事件（相对风险，RR[95%置信区间]2.28[1.69 - 3.09]）和至少一次血脂检测（RR 1.82[1.31 - 2.53]）显著相关。MPR≥80%与首次处方的他汀类药物（阿托伐他汀RR 1.28[1.09 - 1.51]和瑞舒伐他汀RR 1.21[1.01 - 1.44]，对比辛伐他汀）、既往心血管事件（RR 1.33[1.12 - 1.59]）、至少一次治疗变化（RR 1.28[1.15 - 1.43]）、至少一次血脂检测（RR 1.26[1.07 - 1.49]）显著相关。持续性方面观察到类似模式。
对年轻成年HeFH患者的这项分析表明，治疗变化相当频繁，可能反映了根据个体反应进行的调整。即使在这个心血管风险高的人群中，依从性和持续性也不足，需要通过适当的患者教育和共同参与的治疗决策方法加以改善。
