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blooda_adv-2025-017081-main.pdf (551.67 KB)

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2025-09-23

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cc-by-nc-nd (c) American Society of Hematology, 2025
Si us plau utilitzeu sempre aquest identificador per citar o enllaçar aquest document: https://hdl.handle.net/2445/228752

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

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Recurs relacionat

https://doi.org/10.1182/bloodadvances.2025017081

Resum

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.

Matèries

Tractament adjuvant del càncer, Cèl·lules T, Epidemiologia

Matèries (anglès)

Adjuvant treatment of cancer, T cells, Epidemiology

Citació

Col·leccions

Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL))
Articles publicats en revistes (IDIBAPS: Institut d'investigacions Biomèdiques August Pi i Sunyer)

Citació

PENNINGS, Elise R. A., et al. Patient-reported outcomes in patients with hematologic malignancies treated with CAR T-cell therapy in Europe. Blood Advances. 2025. Vol. 9, num. 24, pags. 6380-6393. ISSN 2473-9537. [consulted: 18 of August of 2026]. Available at: https://hdl.handle.net/2445/228752

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