Tchernonog Emmanuelle, Moignet Aline, Anota Amélie, Bernard Sophie, Bouguet Guy, Colin Fanny, Rioufol Catherine, Ysebaert Loïc, Gyan Emmanuel
Hematology department, University hospital, Montpellier.
Hematology department, Pontchaillou University Hospital, Rennes.
Haematologica. 2024 Aug 1;109(8):2401-2419. doi: 10.3324/haematol.2022.282363.
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.
嵌合抗原受体（CAR）T细胞疗法已经改变了复发/难治性B细胞来源的血液系统恶性肿瘤患者的治疗方式。迄今为止，六种靶向CD19或B细胞成熟抗原的CAR T细胞疗法已获得监管批准。除了有前景的生存获益外，CAR T细胞疗法还与潜在的危及生命的不良事件相关，包括细胞因子释放综合征和免疫效应细胞相关的神经毒性综合征。虽然评估CAR T细胞疗法的临床试验一直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细胞治疗过程及相关不良事件对患者身心健康的影响仍不确定。复发或难治性血液系统恶性肿瘤患者的HRQoL和其他患者报告结局（PRO）评估至关重要，因为如果无法治愈，个体可能有未满足的需求且对可耐受治疗有很高要求。此外，标准化数据收集方法对于更好地评估CAR T细胞疗法对生活质量的影响、优化患者护理和成本以及实现不同研究之间的比较很重要。我们进行了截至2023年6月的文献检索，以确定在淋巴瘤或白血病患者中研究CAR T细胞疗法的临床试验和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，并讨论如何优化HRQoL和其他PRO工具的使用。
