Emmanuelle Tchernonog, Aline Moignet, Amélie Anota, Sophie Bernard, Guy Bouguet, Fanny Colin, Catherine Rioufol, Loïc Ysebaert, Emmanuel Gyan
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引用次数: 0
摘要
嵌合抗原受体(CAR)T 细胞疗法改变了对复发/难治性 B 细胞血液恶性肿瘤患者的治疗。迄今为止,已有六种以 CD19 或 B 细胞成熟抗原为靶点的 CAR T 细胞疗法获得了监管部门的批准。CAR T 细胞疗法在给患者带来生存益处的同时,也可能带来危及生命的不良反应(AE),包括细胞因子释放综合征和免疫效应细胞相关神经毒性综合征。尽管评估 CAR T 细胞疗法的临床试验一直在报告这些不良事件的发生率,但大多数试验并未收集与健康相关的生活质量(HRQoL)数据。因此,CAR T 细胞疗法过程和相关 AE 对患者身心健康的影响仍不确定。复发或难治性血液恶性肿瘤患者的 HRQoL 和其他患者报告结果 (PRO) 评估至关重要,因为如果不能治愈,患者可能有未满足的需求,并对可耐受的治疗有很高的要求。此外,为了更好地评估 CAR T 细胞疗法对生活质量的影响、优化患者护理和成本,并对不同研究进行比较,规范数据收集方法非常重要。我们对截至 2023 年 6 月的文献进行了检索,以确定在临床试验中以及在调查淋巴瘤或白血病患者 CAR T 细胞疗法的真实世界研究中使用的 HRQoL 工具。在本综述中,我们总结了最常用的 CAR T 细胞特异性和非特异性 HRQoL 工具,并讨论了如何优化 HRQoL 和其他 PRO 工具的使用。
Health-related quality of life in patients with hematologic malignancies treated with chimeric antigen receptor T-cell therapy: review and current progress.
Chimeric antigen receptor (CAR) T-cell therapy has transformed the care of patients with relapsed/refractory B-cell-derived hematologic malignancies. To date, six CAR T-cell therapies, targeting either CD19 or B-cell maturation antigen, have received regulatory approval. Along with the promising survival benefit, CAR T-cell therapy is associated with potentially life-threatening adverse events, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. While clinical trials evaluating CAR T-cell therapy consistently report the incidence of these adverse events, most trials do not collect health-related quality of life (HRQoL) data. As such, the impact of the CAR T-cell therapy process and related adverse events on the physical and psychological well-being of patients remains uncertain. HRQoL and other patient-reported outcome (PRO) assessments in patients with relapsed or refractory hematologic malignancies are of utmost importance, as individuals may have unmet needs and a high demand for tolerable therapy if a cure is not obtained. In addition, it is important to standardize methods of data collection to better assess the impact of CAR T-cell therapy on quality of life, optimize patients' care and costs, and enable comparisons between different studies. We conducted a literature search up to June 2023 to identify the HRQoL tools used in clinical trials and in real-world studies investigating CAR T-cell therapy in patients with lymphomas or leukemias. In the present comprehensive review, we summarize the most commonly used CAR T-cell specific and non-specific HRQoL tools and discuss how the use of HRQoL and other PRO tools may be optimized.
期刊介绍:
Haematologica is a journal that publishes articles within the broad field of hematology. It reports on novel findings in basic, clinical, and translational research.
Scope:
The scope of the journal includes reporting novel research results that:
Have a significant impact on understanding normal hematology or the development of hematological diseases.
Are likely to bring important changes to the diagnosis or treatment of hematological diseases.