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

项目摘要 肾结石影响11个美国人之一。每年的相关支出为45亿美元， 巨大的资源负担。此外，它们不是一次性事件，50%的患者会发生 五年内复发的第一块石头。因此，减少复发性结石的干预措施可能会促进 健康，并产生积极的经济影响。使用噻嗪类利尿剂，碱性柠檬酸盐治疗，或 别嘌呤醇-称为预防性药物治疗（PPT）-可以代表这样的干预。 然而，虽然目前的指南推荐PPT，但它很少使用。最近一项系统性综述由 医疗保健研究与质量机构强调了PPT证据基础中的三个差距， 首先，先前研究的结果主要是由中间结果驱动的，但是 对患者和提供者来说，最重要的是干预是否能预防未来需要治疗的疼痛发作。 急诊科（艾德）就诊、住院或手术。其次，先前的研究报告很少有关于 治疗耐受性。第三，PPT对石材成型机未来成本的影响在很大程度上是未知的。如果这 从长远来看，治疗为医疗保健系统节省了大量资金，对它的接受程度可能会增加。 填补这三个空白的一个障碍是缺乏具有必要样本量的数据来源， 临床粒度和长期随访，以检查PPT的真实有效性。克服 这一障碍，我们提出了一项研究，利用创新的行政和临床数据的联系。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 从Litholink公司的数据，我们将确定成人肾结石，区分病人处方 噻嗪类、柠檬酸类或别嘌呤醇。我们将评估艾德访视、住院和 这两组的手术率。目的2：检查患者对PPT的耐受性。评估了 耐受性，然后我们将测量处方患者的药物依从性和不良事件发生率 噻嗪类、柠檬酸类或别嘌呤醇。我们将检查患者亚组之间耐受性的差异。目标3： 确定与PPT使用相关的医疗费用。最后，我们将评估纵向支付 肾结石的治疗方法我们将确定这些付款是否存在差异， 接受PPT治疗的患者与接受常规治疗的患者。我们的研究结果有助于为最佳的 肾结石的治疗最终，患者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.