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晚期或转移性肾细胞癌一线治疗患者不良事件的成本

Costs of Adverse Events in Patients with Advanced or Metastatic Renal Cell Carcinoma with First-Line Treatment.

作者信息

Chen Yan, Du Ella X, Sundar Manasvi, Betts Keith A, Yin Xin, Eiffert Samantha, Beauchamp Karen, Delgado Andrew, Rosenblatt Lisa

机构信息

Analysis Group, Inc., Los Angeles, CA, USA.

Bristol Myers Squibb, Princeton, NJ, USA.

出版信息

Pharmacoecon Open. 2025 Jan;9(1):125-136. doi: 10.1007/s41669-024-00534-2. Epub 2024 Nov 6.

Abstract

AIM

This study evaluated costs associated with adverse events (AEs) in previously untreated real-world patients with advanced renal cell carcinoma (aRCC) in the USA.

MATERIALS AND METHODS

This retrospective longitudinal cohort study analyzed data from the Merative MarketScan Research Database (1 January 2014-30 September 2021). Adult patients with aRCC receiving first-line systemic treatments for aRCC (tyrosine kinase inhibitors [TKIs], or combination therapies of TKIs and immunotherapy) on or after the date of aRCC diagnosis were included. A total of 27 AEs of interest were included based on a review of product labels of the first-line treatments included in the study and identified using International Classification of Diseases, Ninth/Tenth Revision, Clinical Modification codes. Incremental costs associated with AEs between cases and controls (unadjusted and adjusted for relevant baseline characteristics) were estimated by two-part modeling. Analyses were performed over three AE cost assessment periods (7, 14, and 30 days).

RESULTS

The study included 1681 patients with aRCC (mean [standard deviation; SD] age, 60.8 [10.6] years; 73.1% male), of which 1542 (91.7%) had at least one AE. AEs were mostly diagnosed in the outpatient (OP) setting. For most AEs, cases had significantly higher unadjusted and adjusted costs than controls. Costs associated with AEs ranged from < 300 US dollars (USD) for proteinuria to nearly 60,000 USD for hypophosphatemia. Seventeen AEs had adjusted 30-day costs exceeding 10,000 USD; of these, nine (pancreatitis, acute kidney injury, dyspnea, hypotension, hyperkalemia, hypomagnesemia, hyponatremia, hypophosphatemia, and neutrophil decreased/neutropenia) had 30-day costs exceeding 20,000 USD.

LIMITATIONS

The study was subject to limitations of all observational analyses of claims data (e.g., residual confounding). Observed cost differences may not have been solely attributable to an AE of interest. Study findings may not be generalizable to aRCC patient populations outside the USA.

CONCLUSION

Most patients experienced at least one AE after initiation of first-line treatment with a TKI or combination therapies of TKIs and immunotherapy. There were substantial costs associated with AEs. Considering both safety and efficacy profiles when selecting optimal treatments can potentially mitigate healthcare costs for aRCC.

摘要

目的

本研究评估了美国既往未接受治疗的晚期肾细胞癌(aRCC)真实世界患者中不良事件(AE)的相关成本。

材料与方法

这项回顾性纵向队列研究分析了来自默克多市场扫描研究数据库(2014年1月1日至2021年9月30日)的数据。纳入了在aRCC诊断日期或之后接受aRCC一线全身治疗(酪氨酸激酶抑制剂 [TKI],或TKI与免疫疗法的联合疗法)的成年患者。基于对研究中纳入的一线治疗产品标签的审查并使用国际疾病分类第九/十版临床修订版代码进行识别,共纳入了27种感兴趣的AE。通过两部分建模估计病例与对照之间(未调整以及针对相关基线特征进行调整)与AE相关的增量成本。在三个AE成本评估期(7天、14天和30天)进行分析。

结果

该研究纳入了1681例aRCC患者(平均 [标准差;SD] 年龄,60.8 [10.6] 岁;73.1% 为男性),其中1542例(91.7%)至少发生了一次AE。AE大多在门诊(OP)环境中被诊断出来。对于大多数AE,病例的未调整和调整后成本均显著高于对照。与AE相关的成本范围从蛋白尿的 < 300美元(USD)到低磷血症的近60,000美元不等。17种AE的调整后30天成本超过10,000美元;其中,9种(胰腺炎、急性肾损伤、呼吸困难、低血压、高钾血症、低镁血症、低钠血症、低磷血症以及中性粒细胞减少/中性粒细胞缺乏)的30天成本超过20,000美元。

局限性

该研究受到所有索赔数据观察性分析的局限性(例如,残余混杂)影响。观察到的成本差异可能并非完全归因于感兴趣的AE。研究结果可能不适用于美国以外的aRCC患者群体。

结论

大多数患者在开始使用TKI或TKI与免疫疗法的联合疗法进行一线治疗后经历了至少一次AE。AE相关成本巨大。在选择最佳治疗方案时考虑安全性和疗效概况可能会潜在地降低aRCC的医疗成本。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ee86/11718028/557465fda642/41669_2024_534_Fig1_HTML.jpg

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