CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Trends and Disparities in Chimeric Antigen Receptor T-Cell Therapy Utilization, Inpatient Mortality, Length of Stay, and Costs in the United States (2017 to 2022).
Trends and Disparities in Chimeric Antigen Receptor T-Cell Therapy Utilization, Inpatient Mortality, Length of Stay, and Costs in the United States (2017 to 2022).
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自2017年8月美国食品药品监督管理局(FDA)批准以来,CAR-T 细胞疗法迅速改变了复发/难治性血液系统恶性肿瘤的治疗格局。
然而,描述不同疾病适应证、社会人口学群体、医院特征和地理区域间应用差异的全国数据仍有限。此外,住院结局(包括死亡率、住院时长及住院成本)的真实世界数据也尚未充分明确。
本研究旨在考察FDA批准后,按疾病适应证、社会人口学因素和医院特征划分的住院CAR-T 使用趋势,以及住院时长、死亡率和成本等住院结局变化。
我们利用2017至2022年美国全国住院样本开展连续横断面分析。采用调查加权广义线性模型及基于Taylor级数线性化计算的设计型标准误分析连续结局;采用调查加权逻辑回归分析二元结局,并报告比值比及95%置信区间(CI)。CAR-T 应用量从2017年的70例增至2022年的4725例,年增长率为32.6%。非霍奇金淋巴瘤仍是最常见适应证,占受治者50%以上。多发性骨髓瘤(MM)成为第二常见适应证,近期监管批准后,其占比从2018年的13.1%增至2022年的23.5%,增幅79%。白人患者占多数(57.1%–74.1%),最高收入四分位人群持续占最大比例(27.8%–37.4%)。65岁及以上患者的治疗使用量大幅增加,到2022年接近较年轻患者,反映了受治人群结构变化。
以Medicare为主要支付方的CAR-T 住院比例从2018年的25.2%升至2022年的40.6%。75%以上的CAR-T 治疗在大型私立医院实施。住院死亡率稳定在约3%。住院时长每年缩短1.16天,平均住院成本则每年增加约28,400美元。美国住院CAR-T 应用迅速扩大,尤其是在近期适应证扩展后,老年患者及MM患者的应用增加。社会人口学差异仍持续存在,白人、高收入和商业保险患者的使用率较高,凸显细胞疗法公平可及方面的持续挑战。尽管住院时长缩短且住院死亡率保持稳定,住院费用上升反映出CAR-T 治疗的经济负担和资源使用不断增加。这些发现为CAR-T 治疗实施格局的演变提供了重要真实世界认识,可帮助制定策略,在细胞疗法持续扩展的背景下优化可及性和医疗资源配置。
Since Food and Drug Administration (FDA) approval in August 2017, chimeric antigen receptor T-cell (CAR-T) therapy has rapidly transformed the treatment landscape of relapsed or refractory hematologic malignancies.
However, national data describing variation in adoption across disease indications, sociodemographic groups, hospital characteristics, and geographic regions remain limited.
In addition, real-world data describing inpatient outcomes, including mortality, length of stay (LOS), and hospitalization costs, remain incompletely understood.
We aimed to investigate trends in inpatient CAR-T utilization by disease indication, sociodemographic factors, and hospital characteristics, as well as trends in inpatient outcomes including LOS, mortality, and hospitalization costs following FDA approval.
We conducted a serial cross-sectional analysis using the National Inpatient Sample from 2017 to 2022 to evaluate trends in inpatient CAR-T utilization across patient and hospital characteristics and associated outcomes, including LOS, mortality, and costs. Survey-weighted generalized linear models with design-based standard errors calculated using Taylor series linearization were used for continuous outcomes. Binary outcomes were analyzed using survey-weighted logistic regression, with results reported as odds ratios with 95% confidence intervals (CIs). CAR-T adoption increased from 70 cases in 2017 to 4725 cases in 2022, corresponding to an annual growth rate of 32. 6%. Non-Hodgkin lymphoma remained the most common indication, accounting for more than 50% of recipients. Multiple myeloma (MM) emerged as the second most common indication, representing a 79% increase from 13. 1% in 2018 to 23. 5% in 2022 following recent regulatory approvals. White patients comprised the majority of recipients (57. 1% to 74. 1%), and individuals in the highest income quartile consistently represented the largest proportion (27.
8% to 37. 4%). Uptake among patients aged 65 yr increased substantially, nearly equaling younger patients by 2022, consistent with this demographic shift. The proportion of CAR-T hospitalizations with Medicare as the primary payer increased from 25. 2% in 2018 to 40. 6% in 2022. Most CAR-T therapies were administered in large private hospitals ( 75%). Inpatient mortality remained stable at approximately 3%. LOS decreased by 1. 16 d annually, while mean hospitalization costs increased by approximately $28,400 per year.
Inpatient CAR-T utilization has expanded rapidly in the United States, particularly among older adults and patients with MM following recent indication expansion. Persistent sociodemographic disparities remain, with higher utilization among White, higher-income, and privately insured patients, highlighting ongoing challenges in equitable access to cellular therapy.
Although LOS has decreased and inpatient mortality has remained stable, rising hospitalization costs reflect the growing economic burden and increasing resource utilization associated with CAR-T delivery.
These findings provide important real-world insights into the evolving implementation of CAR-T therapy and may help inform strategies to optimize access and healthcare resource allocation as cellular therapies continue to expand.
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