Sanofi, Paris, France.
Avalere Health, Washington, DC, USA.
J Med Econ. 2021 Jan-Dec;24(1):706-716. doi: 10.1080/13696998.2021.1922262.
Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare hematologic disorder that can lead to serious life-threatening medical complications.
The aim of this study was to describe aTTP-related hospital resource utilization, cost, complications, and overall survival among US Medicare and non-Medicare populations following aTTP episodes prior to the US approval of caplacizumab.
This retrospective study utilized administrative claims data for Medicare Fee-for-Service (FFS) beneficiaries (100% sample) and a sample of commercial, managed Medicaid [MM], Medicare Advantage [MA] plan members from the Inovalon MORE Registry. aTTP patients ages 18+ were identified between 2010 and 2018 using a published validated algorithm: ≥1 hospitalization for thrombotic microangiopathy + therapeutic plasma exchange (TPE). 2,279 patients were identified; 65.2% were enrolled in Medicare FFS, 13.6% in commercial, 15.7% in MM, and 5.4% in MA. Mean hospitalization days for aTTP index episode ranged between 12 and 17 days; ∼60% of patients required intensive care. Mean payments for index hospitalization varied by payer [Medicare FFS: $29,024; MA: $12,860; commercial: $9,996 and MM: $10,470]. Among FFS patients, 15.7% died during initial hospitalization and 21.0% died within first 30 days of the event. During follow-up, 11.6-19.6% experienced aTTP-related exacerbation. Incidence rate of relapse and complications per 100 person-years was 5.6 [Medicare FFS: 3.6; MA: 8.7; commercial: 10.4 and MM: 14.7] and 16.7 [FFS: 15.5; MA: 20.5; commercial: 21.7 and MM: 19.1], respectively. Among Medicare patients with and without aTTP, mortality risk was 2.9 (95 % CI: 2.4-3.4) times higher for aTTP vs. non-aTTP patients.
This is the first real-world study evaluating burden of illness among aTTP patients in the US across payer types. Despite being treated with TPE, patients with aTTP have lower survival rates in comparison to a matched cohort without aTTP. These findings highlight the need for more effective and novel therapies to reduce disease burden for this population.Key pointsIn US Medicare and managed care populations with aTTP between 2010 and 2018, aTTP can lead to significant utilization of ICU services due to clinical complications, and/or relapse following hospital discharge.Despite treatment with therapeutic plasma exchange, acute mortality remains high (15.7%) indicating the need for more effective and novel treatments.
获得性血栓性血小板减少性紫癜（aTTP）是一种罕见的血液疾病，可导致严重的危及生命的医疗并发症。
本研究旨在描述美国在批准 caplacizumab 之前，接受 TPE 治疗的 aTTP 患者在 aTTP 发作后的美国医疗保险和非医疗保险人群中的与疾病相关的医院资源利用、成本、并发症和总体生存率。
这项回顾性研究利用了医疗保险按服务收费（FFS）受益人的行政索赔数据（100%样本）和 Inovalon MORE 注册中心商业、管理型医疗补助[MM]、医疗保险优势[MA]计划成员的样本。使用已发表的验证算法在 2010 年至 2018 年期间确定了 18 岁以上的 aTTP 患者：≥1 次血栓性微血管病住院治疗+治疗性血浆置换（TPE）。共确定了 2279 名患者；65.2%的患者参加了医疗保险 FFS，13.6%参加了商业保险，15.7%参加了 MM，5.4%参加了 MA。aTTP 指数发作的平均住院天数在 12 至 17 天之间；约 60%的患者需要重症监护。指数住院治疗的平均支付额因支付人而异[医疗保险 FFS：29024 美元；MA：12860 美元；商业：9960 美元和 MM：10470 美元]。在 FFS 患者中，15.7%在初次住院期间死亡，21.0%在事件发生后 30 天内死亡。在随访期间，11.6%-19.6%的患者出现了与 aTTP 相关的恶化。每 100 人年的复发和并发症发生率分别为 5.6（医疗保险 FFS：3.6；MA：8.7；商业：10.4 和 MM：14.7）和 16.7（FFS：15.5；MA：20.5；商业：21.7 和 MM：19.1）。在有和没有 aTTP 的 Medicare 患者中，aTTP 患者的死亡率风险是无 aTTP 患者的 2.9 倍（95%CI：2.4-3.4）。
这是第一项评估美国不同类型支付者中 aTTP 患者疾病负担的真实世界研究。尽管接受了 TPE 治疗，但与未发生 aTTP 的患者相比，aTTP 患者的生存率较低。这些发现强调了需要更有效和新颖的治疗方法来减轻这一人群的疾病负担。
在 2010 年至 2018 年期间，美国医疗保险和管理式医疗人群中患有 aTTP 的患者，由于临床并发症和/或出院后复发，可导致 ICU 服务的大量利用。尽管接受了治疗性血浆置换，但急性死亡率仍然很高（15.7%），表明需要更有效的治疗方法。
