Baxalta U.S. Inc., a Takeda company, Lexington, MA.
Baxalta U.S. Inc., a Takeda company, Cambridge, MA.
J Manag Care Spec Pharm. 2021 Feb;27(2):175-185. doi: 10.18553/jmcp.2020.20327. Epub 2020 Dec 14.
von Willebrand disease (VWD) can lead to serious, life-threatening bleeding events associated with substantial clinical and economic burden. To estimate the prevalence, health care resource utilization (HCRU), and costs associated with major bleeding events in patients with VWD. This was a retrospective analysis of the IBM MarketScan database (2008-2016). Selected patients had ≥ 2 VWD diagnoses, no diagnosis of acquired coagulation factor deficiency, and continuous health care plan enrollment for ≥ 12 months from eligibility start date. Prevalence was calculated as the proportion of eligible patients with ≥ 1 major bleeding event during the observation period (start to end of continuous eligibility). HCRU and costs in the 12-month continuous enrollment period following the first major bleeding event were compared with those from a comparable 12-month period for patients without major bleeding events. Of the 19,785 patients with VWD, 15% experienced ≥ 1 major bleeding event during a median follow-up of 4 years; 89% of these events were gastrointestinal bleeds. For the economic analysis, 773 patients with ≥ 1 major bleeding event and 4,285 patients without major bleeding events met the selection criteria. Controlling for baseline covariates, patients with major bleeding events had significantly ( < 0.0001) more inpatient admissions (incidence rate ratio [IRR] = 3.2; 95% CI = 2.78-3.77), longer inpatient stays (IRR = 3.9; 95% CI = 3.12-4.93), and more emergency department visits (IRR = 2.0; 95% CI = 1.77-2.27) and outpatient visits (IRR = 1.3; 95% CI = 1.19-1.34) than patients without major bleeding events. Annual health care costs were significantly higher ( < 0.01) for patients with major bleeding events than those without them (predicted mean cost differences: total = $20,890, pharmacy = $2,593, and medical = $18,293). Major bleeding events were associated with increased HCRU and costs, mostly inpatient costs. Therefore, optimizing therapy to prevent or reduce major bleeding events has the potential to reduce health care use and costs in patients with VWD. This study was funded by Baxalta U.S. Inc., a Takeda company (Lexington, MA). The study sponsor was involved with the study design, analysis, and interpretation of data; writing of the manuscript; and the decision to publish the article. Lu, Wu, and Ewenstein are employees of Baxalta U.S. Inc., a Takeda company, and are Takeda stock owners. Farahbakhshian is an employee of Shire U.S. Inc., a Takeda company, and is a Takeda stock owner. Oladapo was an employee of Baxalta U.S. Inc., a Takeda company, at the time the analysis was completed and the manuscript developed, and is a Takeda stock owner.
血管性血友病（VWD）可导致严重的、危及生命的出血事件，伴有大量的临床和经济负担。本研究旨在评估血管性血友病患者中与主要出血事件相关的患病率、医疗资源利用（HCRU）和成本。
这是对 IBM MarketScan 数据库（2008-2016 年）的回顾性分析。入选患者至少有 2 次 VWD 诊断，无获得性凝血因子缺乏症诊断，且在入选起始日期起连续 12 个月有完整的健康保险计划。采用在观察期（连续资格起始日期至结束日期）内发生≥1 次主要出血事件的合格患者比例来计算患病率。在首次主要出血事件后的连续 12 个月内比较了 HCRU 和成本，并与无主要出血事件的患者的可比 12 个月期间进行了比较。在 19785 例 VWD 患者中，15%的患者在中位随访 4 年内经历了≥1 次主要出血事件；其中 89%的事件为胃肠道出血。对于经济分析，773 例有≥1 次主要出血事件和 4285 例无主要出血事件的患者符合入选标准。在控制基线协变量后，与无主要出血事件的患者相比，有主要出血事件的患者的住院入院率显著更高（<0.0001；发生率比[IRR] = 3.2；95%置信区间[CI] = 2.78-3.77），住院时间更长（IRR = 3.9；95% CI = 3.12-4.93），急诊就诊次数（IRR = 2.0；95% CI = 1.77-2.27）和门诊就诊次数（IRR = 1.3；95% CI = 1.19-1.34）也更多。与无主要出血事件的患者相比，有主要出血事件的患者的年度医疗保健费用显著更高（<0.01）（预测的平均费用差异：总费用 = 20890 美元，药房费用 = 2593 美元，医疗费用 = 18293 美元）。主要出血事件与 HCRU 和成本的增加相关，主要是住院成本。因此，优化治疗以预防或减少主要出血事件有可能降低 VWD 患者的医疗保健使用和成本。
本研究由 Baxalta US Inc.（Takeda 公司的子公司）资助。研究赞助商参与了研究设计、数据分析和解释、手稿撰写以及发表文章的决策。Lu、Wu 和 Ewenstein 是 Baxalta US Inc.（Takeda 公司的子公司）的员工，也是 Takeda 的股东。Farahbakhshian 是 Shire US Inc.（Takeda 公司的子公司）的员工，也是 Takeda 的股东。Oladapo 在完成分析和制定手稿时是 Baxalta US Inc.（Takeda 公司的子公司）的员工，并且是 Takeda 的股东。
