OPEN Health, Bethesda, MD, USA.
Mallinckrodt Pharmaceuticals, Hampton, NJ, USA.
J Med Econ. 2023 Jan-Dec;26(1):1342-1348. doi: 10.1080/13696998.2023.2260693. Epub 2023 Nov 14.
Hepatorenal syndrome (HRS) is characterized by severely reduced renal perfusion that precipitates rapid morbidity and mortality. Terlipressin is the only US Food and Drug Administration-approved treatment to improve kidney function for adults with HRS with a rapid reduction in kidney function. Prior to the approval of terlipressin, unapproved vasoconstrictive agents used in HRS treatment were octreotide/midodrine and norepinephrine with albumin.
A cohort decision-tree model representing a US hospital perspective assessed the clinical outcomes and direct medical costs (based primarily on hospital charges) of treating HRS with terlipressin + albumin (ALB) versus midodrine/octreotide (MID/OCT)+ALB, or norepinephrine (NorEp)+ALB. Treatment efficacy was defined by clinical response (complete/HRS reversal, partial, or no response) based on change of serum creatinine derived from published clinical trial reports. The proportions of patients with complete response were: terlipressin + ALB (36.2%), NorEp + ALB (19.1%), and MID/OCT + ALB (3.1%). Model outcomes included utilization of HRS-related healthcare resources (hospital and intensive care, outpatient and emergency department, dialysis, and transplantations), adverse events, and HRS-related mortality. Outcomes were assessed for the initial hospitalization in the base case and at 30, 60, and 90 days post-discharge.
Total costs incurred over the initial hospitalization with terlipressin + ALB were lower vs NorEp + ALB, primarily due to higher ICU costs with NorEp + ALB ($7,433 vs $61,897). TER + ALB was associated with higher total costs vs MID/OCT + ALB due to higher pharmacy costs with terlipressin + ALB. The cost per complete response achieved of terlipressin + ALB ($451,605) was half that of NorEp + ALB ($930,571) and one-tenth that of MID/OCT + ALB ($4,942,123).
HRS patients treated with terlipressin experienced better clinical outcomes and a lower cost per treatment response vs other unapproved treatments. ICU days and pharmacy costs were key cost drivers distinguishing the treatment groups. These outcomes suggest that terlipressin is cost-effective on the basis of total cost per response achieved.
肝肾综合征（HRS）的特点是严重的肾脏灌注减少，导致快速发病和高死亡率。特利加压素是唯一获得美国食品和药物管理局批准的治疗药物，可改善 HRS 成人的肾功能，迅速降低肾功能。在特利加压素获得批准之前，HRS 治疗中使用的未经批准的血管收缩剂是奥曲肽/米多君和去甲肾上腺素加白蛋白。
一个代表美国医院视角的队列决策树模型评估了使用特利加压素+白蛋白（ALB）与米多君/奥曲肽（MID/OCT）+ALB 或去甲肾上腺素（NorEp）+ALB 治疗 HRS 的临床结果和直接医疗成本（主要基于医院收费）。治疗效果根据血清肌酐的变化定义为临床反应（完全/逆转 HRS、部分或无反应），基于发表的临床试验报告。完全反应的患者比例为：特利加压素+ALB（36.2%）、NorEp+ALB（19.1%）和 MID/OCT+ALB（3.1%）。模型结果包括 HRS 相关医疗资源（医院和重症监护、门诊和急诊、透析和移植）的使用、不良事件和 HRS 相关死亡率。在基线病例中以及在出院后 30、60 和 90 天评估了初始住院期间的结果。
与 NorEp+ALB 相比，特利加压素+ALB 治疗期间的初始住院总费用较低，主要原因是 NorEp+ALB 的 ICU 费用较高（7433 美元对 61897 美元）。由于特利加压素+ALB 的药房费用较高，特利加压素+ALB 与 MID/OCT+ALB 相比，总成本较高。特利加压素+ALB 获得完全反应的每例成本（451605 美元）是 NorEp+ALB（930571 美元）的一半，是 MID/OCT+ALB（4942123 美元）的十分之一。
与其他未经批准的治疗方法相比，使用特利加压素治疗的 HRS 患者获得了更好的临床结果和更低的治疗反应成本。重症监护病房天数和药房成本是区分治疗组的关键成本驱动因素。这些结果表明，特利加压素在基于获得的总反应成本方面具有成本效益。
