Publicaciones científicas
Health-related quality of life with idecabtagene vicleucel in relapsed and refractory multiple myeloma
Michel Delforge 1 , Nina Shah 2 , Jesus F San-Miguel 3 , Julia Braverman 4 , Devender S Dhanda 4 , Ling Shi 5 , Shien Guo 5 , Peiwen Yu 5 , Weiqin Liao 5 , Timothy B Campbell 6 , Nikhil C Munshi 7
Abstract
Idecabtagene vicleucel (ide-cel), a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell therapy, showed deep, durable responses in patients with triple-class exposed (TCE) relapsed and refractory multiple myeloma (RRMM) in the phase 2 KarMMa trial.
We assessed health-related quality of life (HRQoL) among KarMMa patients. EORTC QLQ-C30, EORTC QLQ-MY20, and EQ-5D-5L were given at screening, baseline (≤72 hours prior to or same day as lymphodepletion), day of ide-cel treatment, and after ide-cel treatment.
Mean changes from baseline that exceeded the predetermined threshold of minimally important difference were deemed clinically meaningful. Proportions of patients experiencing clinically meaningful changes in HRQoL were assessed using within-patient change thresholds.
Time to stable improvement (≥2 consecutive visits with clinically meaningful HRQoL improvements) was analyzed using the Kaplan-Meier method. 126/128 (98%) patients treated with ide-cel were included in the HRQoL analysis. Pretreatment baseline RRMM burden was high and meaningfully worse than the age- and sex-weighted general population.
Statistically significant and clinically meaningful improvements from baseline were observed by month 1 for pain (-8.9) and disease symptoms (-10.2), and by month 2 for fatigue (-7.2), physical functioning (+6.1), cognitive functioning (+6.7), and global health status/QoL (+8.0). Clinically meaningful improvements in fatigue, pain, and physical functioning were most prominent at months 9, 12, and 18, respectively, and were sustained through 15 to 18 months after ide-cel treatment.
For TCE patients with RRMM with a poor prognosis and few treatment options, a single ide-cel infusion provides early, sustained, statistically significant, and clinically meaningful improvements in HRQoL.
CITA DEL ARTÍCULO Blood Adv. 2022 Feb 22;6(4):1309-1318. doi: 10.1182/bloodadvances.2021005913