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

Patient-reported outcomes (PROs) give direct insights into the treatment's impact on patient's life and complement clinical outcomes. However, since the advent of chimeric antigen receptor T-cell therapy (CAR-T), PROs have been underreported. Particularly, little is known about long-term health-related quality of life (HRQoL) and dimensions such as mental- and social well-being, 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. From 10 European countries, 389 patients participated (>1 year post-CAR-T: 56%). Mean EQ-VAS was 73.1 (standard deviation, 18.5). HRQoL was similar or better than reference cohorts, except for role-, social-, and cognitive-functioning. Physical-functioning problems were most frequently reported (41%), particularly by women, older individuals, 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. 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 with reference cohorts. However, a notable proportion of patients experienced problems in physical-, mental- and social well-being. We identified high-risk subgroups and care needs that should be addressed during follow-up.

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Publication Details

Journal
Blood Advances
Published
2025-09-24
DOI
https://doi.org/10.1182/bloodadvances.2025017081
Primary Topic
CAR-T cell therapy research
Type
article
Field-Weighted Citation Impact
0.00

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article

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

Anna Fleischer, Emma Morris, Yolanda Cabrerizo, Solène Clavreul et al.
Blood Advances
CAR-T cell therapy research
article

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

Anna Fleischer, Emma Morris, Yolanda Cabrerizo, Solène Clavreul, Elise R. A. Pennings, Sébastien Anguille, Marie Robin, Julio Delgado, Hélène Trebeden‐Negre, Anne M. Spanjaart, Simone Oerlemans, R. D. Liu, Andreas Kremer, Myriam Pannard, María Gomes da Silva, Olga Millán, Ulrich Jäger, Carin A. Uyl‐de Groot, Birgit I. Lissenberg‐Witte, Samantha Nier, Natacha Bolaños, Maik Luu, Carmen Sanges, Maria T. Kuipers, Delphine Maucort‐Boulch, Geertruida H. de Bock, Anna Sureda, Frederick W. Thielen, Eglys González‐Marcano, Marie Préau, Scott Wagers, Michael Hudecek, Pacôme Lecot, Margot Lorrain, Caroline Dreuillet, Marta Pina, Barbara Huber, Lutgart Roux-Opstaele
article en

Abstract

Patient-reported outcomes (PROs) give direct insights into the treatment's impact on patient's life and complement clinical outcomes. However, since the advent of chimeric antigen receptor T-cell therapy (CAR-T), PROs have been underreported. Particularly, little is known about long-term health-related quality of life (HRQoL) and dimensions such as mental- and social well-being, 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. From 10 European countries, 389 patients participated (>1 year post-CAR-T: 56%). Mean EQ-VAS was 73.1 (standard deviation, 18.5). HRQoL was similar or better than reference cohorts, except for role-, social-, and cognitive-functioning. Physical-functioning problems were most frequently reported (41%), particularly by women, older individuals, 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. 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 with reference cohorts. However, a notable proportion of patients experienced problems in physical-, mental- and social well-being. We identified high-risk subgroups and care needs that should be addressed during follow-up.

Blood AdvancesVol. 9(24)
Université Claude Bernard Lyon 1 (FR), Centre National de la Recherche Scientifique (FR), Advocates for Youth (US), University Medical Center Groningen (NL), University College London Hospitals NHS Foundation Trust (GB), RUAG (Germany) (DE), International Society for Optics and Photonics (US), Inserm (FR), University of Luxembourg (LU), Université Paris Cité (FR), Erasmus Hospital (BE), European Hematology Association (NL), FIT Consulting (Italy) (IT), Institut d'Investigació Biomédica de Bellvitge (ES), Universitäts-Kinderklinik Würzburg (DE), Augenklinik Universitätsklinikum Würzburg (DE), Assistance Publique – Hôpitaux de Paris (FR), Hospices Civils de Lyon (FR), Antwerp University Hospital (BE), Instituto Português de Oncologia Francisco Gentil (PT), Koenig & Bauer (Germany) (DE), Institut Català d'Oncologia (ES), Amsterdam Neuroscience (NL), National Center for Tumor Diseases (DE), Luxembourg Institute of Science and Technology (LU), HOPE Clinic (US), Groupe de Recherche en Psychologie Sociale (FR), Hospital Clínic de Barcelona (ES), European Federation of Allergy and Airways Diseases Patients Associations (BE), Laboratoire de Biométrie et Biologie Evolutive (FR), Netherlands Comprehensive Cancer Organisation (NL), Hôpital Saint-Louis (FR), Fraunhofer Institute for Cell Therapy and Immunology (DE), Cellular Therapeutics (United Kingdom) (GB), Institut National du Cancer (FR), Lymphoma Study Association (FR), Institut de Relations Internationales et Stratégiques (FR), Amsterdam University Medical Centers (NL), Délégation Régionale Auvergne-Rhône-Alpes (FR), International Myeloma Foundation (US), Consorci Institut D'Investigacions Biomediques August Pi I Sunyer (ES), Arbed (Luxembourg) (LU), European Society for Blood and Marrow Transplantation (FR), Institute of Infection and Immunity (CA), Cancer Center Amsterdam (NL), University College London (GB), Amsterdam University of Applied Sciences (NL), Medical University of Vienna (AT), Vrije Universiteit Amsterdam (NL), University of Amsterdam (NL), Erasmus University Rotterdam (NL)
Pulmonary Fibrosis Foundation, Eli Lilly and Company, AstraZeneca, Gilead Sciences, Celgene, National Institute for Health and Care Excellence, European Federation of Pharmaceutical Industries and Associations, European Commission, Bundesamt für Gesundheit, Ministerie van Volksgezondheid, Welzijn en Sport
Openalex Percentile: Top 64%
CAR-T cell therapy research
DOIOpen Access PDFOpenAlex
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