Real-world effectiveness of preventive pharmacological therapy for patients with kidney stones

项目总结 每11个美国人中就有一个患肾结石。由于每年相关支出为45亿美元，它们还构成了 巨大的资源负担。此外，它们不是一次性事件，50%的患者会患上 第一颗结石在五年内复发。因此，减少结石复发的干预措施可能会促进 健康，并具有积极的经济影响。使用噻嗪利尿剂，碱性柠檬酸盐治疗，或 别嘌醇--被称为预防性药物疗法(PPT)--可能就是这样一种干预。 然而，尽管目前的指南建议使用PPT，但它并不经常使用。最近的一项系统审查，由 医疗研究和质量机构强调了PPT、THEMING的证据基础中的三个差距 对它的热情。首先，先前研究的结果主要是由中间结果驱动的，但什么？ 对于患者和提供者来说，重要的是干预是否能防止未来需要 急诊科(ED)就诊、住院或手术。其次，之前的研究报告的数据很少 治疗耐受性。第三，PPT对石雕未来成本的影响在很大程度上是未知的。如果这个 从长远来看，治疗为医疗系统节省了大量资金，人们可能会更多地接受它。 填补这三个空白的一个障碍是具有必要样本大小的数据源的可用性， 临床粒度和长期随访，以检查PPT的真实效果。要克服 针对这一障碍，我们提出了一项使用管理数据和临床数据的创新联系的研究。我们的建议 有三个具体目标。目的1：评价PPT对临床结局的影响。使用 退伍军人健康管理局企业数据仓库和医疗保险索赔与尿液化学有关 来自Litholink Corporation的数据，我们将识别患有肾结石的成年人，区分开出的患者 接受常规护理的患者提供的噻嗪类药物、柠檬酸盐或别嘌醇。我们将评估急诊室就诊、住院和 这两组的手术率。目的2：检测患者对PPT的耐受性。作为对 耐受性，然后我们将测量患者的服药依从性和不良事件发生率 硫氮化物、柠檬酸盐或别嘌醇。我们将检查不同患者亚组之间的耐受性差异。目标3： 以确定与PPT使用相关的医疗成本。最后，我们将评估纵向付款。 适用于肾结石患者。我们将确定这些付款之间是否存在差异 患者服用PPT与接受常规护理的患者相比。我们的研究结果有助于为最佳 肾结石的治疗。为此，患者最终将从我们的研究中受益最多。

PROJECT SUMMARY Kidney stones affect one in 11 Americans. With related expenditures of $4.5 billion annually, they also pose a tremendous resource burden. In addition, they are not a one-off event with 50% of patients developing a recurrence within five years of their first stone. Thus, interventions that reduce recurrent stones may promote health and have positive economic implications. The use of thiazide diuretics, alkali citrate treatment, or allopurinol—referred to as preventive pharmacological therapy (PPT)—may represent one such intervention. Yet, while current guidelines recommend PPT, it is infrequently used. A recent systematic review funded by the Agency for Healthcare Research & Quality highlighted three gaps in the evidence base for PPT, tempering enthusiasm for it. First, findings from prior studies were driven mainly by intermediate outcomes, but what matters to patients and providers is whether an intervention will prevent future pain episodes that require an emergency department (ED) visit, hospitalization, or surgery. Second, prior studies reported few data on treatment tolerability. Third, the impact that PPT has on future costs in stone formers is largely unknown. If this therapy yields a substantial savings to the healthcare system over the long run, acceptance of it may increase. An obstacle to filling these three gaps has been the availability of data sources with the necessary sample size, clinical granularity, and long-term follow-up for examining the real-world effectiveness of PPT. To overcome this obstacle, we propose a study using innovative linkages of administrative and clinical data. Our proposal has three Specific Aims. Aim 1: To evaluate the impact that PPT has on clinical outcomes. Using the Veterans Health Administration Corporate Data Warehouse and Medicare claims linked with urine chemistry data from Litholink Corporation, we will identify adults with kidney stones, distinguishing patients prescribed thiazides, citrates, or allopurinol from those receiving usual care. We will evaluate ED visit, hospitalization, and surgery rates in these two groups. Aim 2: To examine patient tolerability for PPT. As an assessment of tolerability, we will then measure medication adherence and adverse event rates among patients prescribed thiazides, citrates, or allopurinol. We will examine differences in tolerability across patient subgroups. Aim 3: To determine the healthcare costs associated with PPT use. Finally, we will assess longitudinal payments for patients with kidney stones. We will determine whether variability in these payments exists between patients prescribed PPT versus those receiving usual care. Findings from our study serve to inform the optimal management of kidney stones. To this end, patients will ultimately benefit most from our research.