Role of disease risk scores in comparative effectiveness research with emerging therapies.
Role of disease risk scores in comparative effectiveness research with emerging therapies.
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DOI：
10.1002/pds.3231
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发表时间：
2012-05
影响因子：
2.6
通讯作者：
Schneeweiss, Sebastian
中科院分区：
文献类型：
--
作者：
Glynn, Robert J.;Gagne, Joshua J.;Schneeweiss, Sebastian
对于使用模式不断变化的新引入的疗法，倾向评分和回归模型在治疗开始时平衡潜在混杂因素的作用可能会受到限制。考虑疾病风险评分作为比较有效性研究中的平衡评分具有理论优势的情况，因为疾病风险的稳定性以及在引入新疗法之前治疗人群的结果的充足历史数据的可用性。我们回顾了在不断发展的疗法中疾病风险评分的适应症和平衡特性，并讨论了替代的估计方法。我们根据 1995 年至 2004 年出院后 30 天内服用他汀类药物处方的 5,668 名老年心肌梗死幸存者的数据，说明了在 1997 年开始引入阿托伐他汀和使用高剂量他汀类药物治疗的背景下疾病风险评分的发展。理论考虑表明在此期间非阿托伐他汀和高剂量他汀类药物使用者的疾病风险评分的发展 1995 年至 1997 年。在疾病风险评分的五分位中，观察到的事件风险从 11% 增加到 35%，其 C 统计值为 0.71。即使在研究终点很少的早期随访期间，该评分也可以控制许多潜在的混杂因素。在某些情况下，例如新上市的药物，平衡疾病风险评分为倾向评分提供了一种有吸引力的替代方案，并为评估潜在效果修改提供了重要的轴。共同考虑倾向和疾病风险评分可能很有价值。
Usefulness of propensity scores and regression models to balance potential confounders at treatment initiation may be limited for newly introduced therapies with evolving use patterns. To consider settings in which the disease risk score has theoretical advantages as a balancing score in comparative effectiveness research, because of stability of disease risk and the availability of ample historical data on outcomes in people treated before introduction of the new therapy. We review the indications for and balancing properties of disease risk scores in the setting of evolving therapies, and discuss alternative approaches for estimation. We illustrate development of a disease risk score in the context of the introduction of atorvastatin and the use of high-dose statin therapy beginning in 1997, based on data from 5,668 older survivors of myocardial infarction who filled a statin prescription within 30 days after discharge from 1995 until 2004. Theoretical considerations suggested development of a disease risk score among non-users of atorvastatin and high-dose statins during the period 1995–1997. Observed risk of events increased from 11% to 35% across quintiles of the disease risk score which had a C-statistic of 0.71. The score allowed control of many potential confounders even during early follow-up with few study endpoints. Balancing on a disease risk score offers an attractive alternative to a propensity score in some settings such as newly marketed drugs and provides an important axis for evaluation of potential effect modification. Joint consideration of propensity and disease risk scores may be valuable.
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影响因子：
2.1
作者：
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通讯作者：
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