Longitudinal Patient Reported Outcomes with CAR-T Cell Therapy Versus Autologous and Allogeneic Stem Cell Transplant

Abstract

There are limited data on patient experience after chimeric antigen receptor (CAR) T-cell therapy, especially in comparison to autologous and allogeneic transplantation, which are more established forms of cellular therapy. We prospectively evaluated longitudinal patient-reported quality of life (QoL), symptom burden and cognition after CAR-T cell therapy and compared it with prospective cohorts of patients undergoing autologous stem cell transplantation (autoSCT) and allogeneic SCT (alloSCT). This was a single center study. The primary endpoint was change in QoL. Secondary endpoints were patient-reported adverse events (PRO-AEs) measured by Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and cognitive function (NeuroQOLv2 questionnaire). Time profile of PRO-AEs was evaluated using longitudinal analysis, Toxicity over Time (ToxT). Patients completed questionnaires at baseline, week 2 and monthly for 6 months. One hundred four patients were evaluable (CAR-T: 34, autoSCT: 33, alloSCT: 37). Baseline QoL was similar across groups. We observed a short-term decline in QoL in all groups that gradually returned to baseline. The nadir in QoL was at week 2 and coincided with peak in symptom burden. The decline in overall QoL, physical and functional well-being was significantly less with CAR-T versus SCT groups and returned to baseline faster. Patients in the alloSCT group experienced the greatest symptom burden, greater decrease in performance status, largest short-term decline in QoL and slowest recovery. This study provides comprehensive data comparing QoL, PRO-AEs and cognition following CAR-T cell therapy versus autoSCT and alloSCT, and the first application of ToxT to PRO-CTCAE data. Short-term QOL, including physical and functional domains was better in the CAR-T group versus SCT groups, although all groups experienced an initial decline coinciding with peak symptoms. These data can serve as a guide for patient education, symptom management, and future studies in CAR-T cell therapy.

Keywords: Adverse events; CAR-T therapy; Chimeric antigen receptor; Cognition; Patient experience; Patient-reported outcomes; Quality of life.