Patient-reported outcomes in patients with hematologic malignancies treated with CAR T-cell therapy in Europe

Abstract

Patient-reported outcomes (PROs) give direct insights into the treatment's impact on patient's life and are an essential addition to clinical outcomes. However, since the advent of CAR T-cell therapy (CAR-T), PROs have been underreported. Particularly little is known about long-term health-related quality of life (HRQoL) and dimensions as mental- and social wellbeing, working-life and financial burden. Therefore, we evaluated multidimensional PROs in a cross-sectional study among European patients who received CAR-T for hematologic malignancies. Patients completed validated questionnaires (EQ-5D-5L/EORTC-QLQ-C30/PCL-5/modified-iPCQ) and ad hoc items on treatment experiences, unmet care needs and HRQoL. The survey was available online (January-October 2023) in 7 languages. Outcomes were compared with the European general population, a matched CAR-T naive cohort with hematologic malignancies and across subgroups, using established thresholds for clinically important differences/problems and regression models. Patients from 10 European countries participated (N=389; CAR-T>1 year ago:56%). Mean EQ-VAS and EQ-index were 73.1(SD:18.5) and 0.842(SD:0.177). HRQoL was similar/better than reference cohorts, except for role-, social-, and cognitive-functioning. Physical-functioning problems were most frequently reported(41%), particularly by women, elderly, and those who experienced neurotoxicity. The latter subgroup also reported more cognitive- and social-functioning problems. Anxiety regarding disease recurrence(76%), infections(66%) and long-term side-effects(59%) was common and 4% met provisional PTSD-diagnosis criteria. Among working-age patients, 72% could continue paid work after CAR-T. Younger patients(32%) reported more financial difficulties than older patients(9%). This study shows favorable general HRQoL after CAR-T compared to reference cohorts. However, a notable proportion of patients experienced problems in physical-, mental- and social wellbeing. We identified high-risk subgroups and care needs that should be addressed during CAR-T follow-up.