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血液系统恶性肿瘤患者接受嵌合抗原受体T细胞(CAR T)治疗后的患者报告结局。

Patient-reported outcomes after CAR T-cell therapy in patients with hematological malignancies.

作者信息

Wang Xin Shelley, Srour Samer A

机构信息

University of Texas M.D. Anderson Cancer Center, Houston, TX.

出版信息

Hematology Am Soc Hematol Educ Program. 2024 Dec 6;2024(1):102-108. doi: 10.1182/hematology.2024000536.

Abstract

The remarkable improvement in survival among individuals with hematological malignancies receiving chimeric antigen receptor (CAR) T-cell therapy has highlighted the growing unmet need to incorporate patient-centered assessments in management guidelines for these patients. That CAR T-cell therapy is associated with unique toxicities and relatively high symptom burden in the first few weeks after cell infusion is well known. Magnifying the patient's voice by using patient-reported outcomes (PROs) might support personalized intervention in the acute-care setting, optimize the use of medical resources, improve satisfaction with therapy, and enhance survival benefit. However, various factors impede PRO use in routine patient care: (1) the feasibility of PRO assessment during the acute phase of treatment, especially in patients experiencing neurological toxicities, is not well established; (2) although PROs are widely used in drug- development trials, the assessment tools used in clinical trials primarily inform quality-of-life or safety comparisons among study arms and are rarely the proper tools for assessing and capturing clinically meaningful adverse events that should be monitored in routine patient care; (3) PRO data that could guide how best to monitor and capture the delayed effects of CAR T-cell therapy in long-term survivors are limited. There is a pressing need to overcome these barriers to integrating evidence-based PROs into standard-of-care guidelines for patients receiving CAR T-cell therapy. In this review, we present the current state of PRO utilization in CAR T-cell therapy. We also discuss practical approaches and future directions for successful implementation of PROs in the care of patients receiving CAR T-cell therapy.

摘要

接受嵌合抗原受体(CAR)T细胞疗法的血液系统恶性肿瘤患者生存率显著提高,这凸显了在这些患者的管理指南中纳入以患者为中心评估的未满足需求日益增长。众所周知,CAR T细胞疗法在细胞输注后的最初几周内会产生独特的毒性,且症状负担相对较高。通过使用患者报告结局(PRO)来放大患者的声音,可能有助于在急性护理环境中进行个性化干预,优化医疗资源的使用,提高对治疗的满意度,并增强生存获益。然而,多种因素阻碍了PRO在常规患者护理中的应用:(1)治疗急性期PRO评估的可行性,尤其是在出现神经毒性的患者中,尚未明确;(2)尽管PRO在药物研发试验中广泛使用,但临床试验中使用的评估工具主要用于研究组间生活质量或安全性的比较,很少是用于评估和捕捉常规患者护理中应监测的具有临床意义的不良事件的合适工具;(3)能够指导如何最佳监测和捕捉CAR T细胞疗法对长期存活者延迟影响的PRO数据有限。迫切需要克服这些障碍,将基于证据的PRO纳入接受CAR T细胞疗法患者的标准护理指南。在本综述中,我们介绍了PRO在CAR T细胞疗法中应用的现状。我们还讨论了在接受CAR T细胞疗法患者的护理中成功实施PRO的实用方法和未来方向。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e7f3/11665578/36ff31f3fd64/hem.2024000536_s1.jpg

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