Patient-Reported Outcomes among Multiple Myeloma Patients Treated with Standard of Care Idecabtagene Vicleucel

Simple Summary In clinical trials, patients treated with idecabtagene vicleucel (ide-cel) chimeric antigen receptor T-cell therapy (CAR T) have reported meaningful improvements in patient-reported outcomes, such as health-related quality of life. To test whether these findings are generalizable to the broader, real-world patient population, this study aimed to prospectively characterize patient-reported outcomes (i.e., health-related quality of life, symptom burden) among patients with relapsed/refractory multiple myeloma treated with ide-cel CAR T in standard of care. Patient-reported outcomes were assessed across 14 timepoints from pre-CAR T infusion through day 90 post-infusion. Patients reported significant and meaningful improvements in health-related quality of life and physical well-being by day 60 after CAR T infusion. Overall, most patients had meaningful improvement or maintenance of patient-reported outcomes collected over time. Findings have implications for treatment decision-making, patient education, and supportive interventions to improve patient outcomes post-CAR T.Abstract Idecabtagene vicleucel (ide-cel) was the first FDA-approved chimeric antigen receptor T-cell therapy for relapsed/refractory multiple myeloma (RRMM) patients. This was the first study to evaluate patient-reported outcomes (PROs) among RRMM patients receiving ide-cel in standard of care (SOC). We prospectively assessed health-related quality of life (HRQOL) and symptoms from pre-infusion (baseline) through day (D)90 post-infusion. Baseline PRO associations with patient characteristics, mean PRO changes, and time to stable change were evaluated with t-tests, linear mixed-effects models, and Kaplan-Meier analyses, respectively. Within-person change scores and minimally important difference thresholds determined clinical and meaningful significance. Participants (n = 42) were a median of 66 years old (range: 43-81). At baseline, extramedullary disease was associated with worse physical well-being (p = 0.008), global pain (p < 0.001), performance status (p = 0.002), and overall symptom burden (p < 0.001). Fatigue (p < 0.001) and functional well-being (p = 0.003) worsened by D7 before returning to baseline levels. Overall HRQOL (p = 0.008) and physical well-being (p < 0.001) improved by D60. Most participants reported PRO improvement (10-57%) or maintenance (23-69%) by D90. The median time it took to stabile deterioration in functional well-being was 14 days. The median time it took to stabile improvement in physical and emotional well-being was 60 days. Overall, RRMM patients reported improvements or maintenance of HRQOL and symptom burden after SOC ide-cel.