Efficace Fabio, Cannella Laura, Sparano Francesco, Giesinger Johannes M, Vignetti Marco, Baron Frédéric, Bruera Eduardo, Luppi Mario, Platzbecker Uwe
Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
University Hospital of Psychiatry II, Medical University of Innsbruck, Austria.
Hemasphere. 2022 Dec 1;6(12):e802. doi: 10.1097/HS9.0000000000000802. eCollection 2022 Dec.
The inclusion of patient-reported outcome (PRO) measures in chimeric antigen receptor (CAR) T-cell therapy research is critical for understanding the impact of this novel approach from a unique patient standpoint. We performed a scoping review to map the available literature on the use of PRO measures in CAR T-cell therapy studies of patients with hematologic malignancies published between January 2015 and July 2022. Fourteen studies were identified, of which 7 (50%) were investigational early-phase trials, 6 (42.9%) were observational studies, and 1 (7.1%) was a pilot study. The EQ-5D and the PROMIS-29 were the 2 most frequently used PRO measures, being included in 6 (42.9%) and 5 (35.7%) studies, respectively. Despite differences in study designs, there seems to be evidence of improvements over time since CAR T-cell infusion in important domains such as physical functioning and fatigue, at least in patients who respond to therapy. Overall, the studies identified in our review have shown the added value of PRO assessment in CAR T-cell therapy research by providing novel information that complements the knowledge on safety and efficacy. However, there are several questions which remain to be answered in future research. For example, limited evidence exists regarding patient experience during important phases of the disease trajectory as only 4 (28.6%) and 5 (35.7%) studies provided information on PROs during the first 2 weeks from CAR T-cell infusion and after the first year, respectively. Time is ripe for a more systematic implementation of high-quality PRO assessment in future clinical trials and in real-life settings of patients treated with CAR T-cell therapy.
将患者报告结局(PRO)指标纳入嵌合抗原受体(CAR)T细胞疗法研究,对于从独特的患者角度理解这种新方法的影响至关重要。我们进行了一项范围综述,以梳理2015年1月至2022年7月间发表的关于血液系统恶性肿瘤患者CAR T细胞疗法研究中使用PRO指标的现有文献。共确定了14项研究,其中7项(50%)为早期试验性研究,6项(42.9%)为观察性研究,1项(7.1%)为试点研究。EQ-5D和PROMIS-29是最常用的两项PRO指标,分别有6项(42.9%)和5项(35.7%)研究使用。尽管研究设计存在差异,但至少在对治疗有反应的患者中,有证据表明自CAR T细胞输注后,在身体功能和疲劳等重要领域随时间推移有改善。总体而言,我们综述中确定的研究通过提供补充安全性和疗效知识的新信息,显示了PRO评估在CAR T细胞疗法研究中的附加价值。然而,未来研究仍有几个问题有待解答。例如,关于疾病轨迹重要阶段患者体验的证据有限,因为分别只有4项(28.6%)和5项(35.7%)研究提供了CAR T细胞输注后前2周和第一年之后的PRO信息。现在是在未来临床试验和接受CAR T细胞疗法治疗的患者的实际生活环境中更系统地实施高质量PRO评估的时候了。
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