Grace periods in comparative effectiveness studies of sustained treatments

Kerollos Nashat Wanis, Aaron L Sarvet, Lan Wen, Jason P Block, Sheryl L Rifas-Shiman, James M Robins, Jessica G Young
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Abstract Researchers are often interested in estimating the effect of sustained use of a treatment on a health outcome. However, adherence to strict treatment protocols can be challenging for individuals in practice and, when non-adherence is expected, estimates of the effect of sustained use may not be useful for decision making. As an alternative, more relaxed treatment protocols which allow for periods of time off treatment (i.e. grace periods) have been considered in pragmatic randomized trials and observational studies. In this article, we consider the interpretation, identification, and estimation of treatment strategies which include grace periods. We contrast natural grace period strategies which allow individuals the flexibility to take treatment as they would naturally do, with stochastic grace period strategies in which the investigator specifies the distribution of treatment utilization. We estimate the effect of initiation of a thiazide diuretic or an angiotensin-converting enzyme inhibitor in hypertensive individuals under various strategies which include grace periods.
持续治疗比较效果研究中的宽限期
摘要 研究人员通常有兴趣估计持续使用某种治疗方法对健康结果的影响。然而,在实践中,坚持严格的治疗方案对个人来说可能具有挑战性,而且,当预期不坚持治疗时,对持续使用效果的估计可能对决策没有帮助。作为一种替代方案,务实的随机试验和观察性研究考虑了更为宽松的治疗方案,允许患者有一段时间不接受治疗(即宽限期)。在本文中,我们将对包含宽限期的治疗策略进行解释、识别和估算。我们将自然宽限期策略与随机宽限期策略进行了对比,前者允许个体按照自己的意愿灵活地接受治疗,而后者则由研究者指定治疗利用率的分布。我们估算了在包含宽限期的各种策略下,高血压患者开始服用噻嗪类利尿剂或血管紧张素转换酶抑制剂的效果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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