Background: Axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor (CAR) T-cell therapy, has significantly improved clinical outcomes in adult patients with relapsed/refractory large B-cell lymphoma (LBCL). However, few studies have examined patient-reported outcomes (PROs) or neurocognitive performance in patients treated with axi-cel. Moreover, no longitudinal PRO study has reported on patients treated with axi-cel as standard of care in the United States, to our knowledge.
Objective: This paper reports on real-world changes in PROs [i.e., quality of life (QOL) and perceived cognition] and objective neurocognitive performance before treatment with axi-cel and in the first year after.
Study design: Patients scheduled to receive axi-cel as standard of care were recruited from a single cancer center between March 2020 and June 2022. QOL was assessed using the EORTC QLQ-C30 and EQ-5D-5L at baseline recruitment (i.e., prior to conditioning chemotherapy before axi-cel), and at 7, 14, 30, 60, 90, 180 and 360 days after receiving axi-cel. Perceived cognition was assessed using the PROMIS Cognitive Function 4a scale. Objective neurocognitive performance was assessed using a battery of tests at baseline, and 30, 90, and 360 days after receiving axi-cel. Random-effects mixed models evaluated changes in QOL, perceived cognition, and neurocognitive performance using all available data. Clinically meaningful change in QOL was defined as a difference of 10 points on the EORTC QLQ-C30. Clinically meaningful change in perceived cognition or neurocognitive performance was defined as a difference of 5 points.
Results: On average, participants (N=53) were 63 years of age (SD=13), and predominantly male (62%), White (92%), and college graduates (60%). Participants reported statistically significant improvements from baseline to day 360 in overall QOL, physical functioning, role functioning, and social functioning (ps<0.05) after axi-cel, despite clinically significant worsening in the first 14 days. For role functioning and social functioning, improvements also met criteria for clinical significance. There were no statistically (ps>0.05) or clinically significant changes in perceived cognition over time. Despite some transient declines, neurocognitive performance generally returned to or exceeded baseline levels by day 360 (ps<0.01). However, visuospatial ability worsened by day 90 and did not recover to baseline levels by day 360 (p<.0001).
Conclusions: These real-world data suggest that axi-cel is associated with significant improvements in overall QOL in the first year after infusion. These data are generally consistent with, or exceed, improvements in QOL reported from clinical trials of axi-cel therapy. Despite transient worsening in the acute period after treatment, neurocognitive performance in most domains also recovered to pre-treatment levels by one year after infusion. These findings extend previous research by reporting on patients' perspectives on axi-cel therapy received as standard of care in the real-world setting, and neurocognitive changes after treatment with axi-cel.
Keywords: axicabtagene ciloleucel; chimeric antigen receptor t-cell therapy; neurocognitive performance; patient-reported outcomes; perceived cognition; quality of life.
Copyright © 2024. Published by Elsevier Inc.