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

Elise R A Pennings ^1,2,3,4, Anne M Spanjaart ^1,2,3, Frederick W Thielen ^4,5

¹Department of Hematology, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands.
²Cancer Center Amsterdam, Amsterdam, The Netherlands.
³Lymphoma and Myeloma Center Amsterdam, Amsterdam, The Netherlands.
⁴Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, The Netherlands.
⁵Erasmus Centre for Health Economics Rotterdam, Erasmus University Rotterdam, Rotterdam, The Netherlands.
⁶Department of Research and Development, Netherlands Comprehensive Cancer Organisation, Utrecht, The Netherlands.
⁷Lehrstuhl für Zelluläre Immuntherapie, Medizinische Klinik und Poliklinik II, Universitätsklinikum Würzburg, Würzburg, Germany.
⁸Department of Hematology, Portuguese Institute of Oncology, Lisboa, Portugal.
⁹European Hematology Association, The Hague, The Netherlands.
¹⁰Institut National du Cancer (French National Cancer Institute), Boulogne Billancourt, France.
¹¹Patvocates GmbH, Riemerling, Germany.
¹²Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria.
¹³Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
¹⁴Department of Hematology, Hospital Clinic of Barcelona, Barcelona, Spain.
¹⁵Information Technology for Translational Medicine S.A., Esch sur Alzette, Luxembourg.
¹⁶Lymphoma Coalition Europe, Paris, France.
¹⁷Myeloma Patients Europe, Brussels, Belgium.
¹⁸Acute Leukemia Advocates Network, Bern, Switzerland.
¹⁹Department of Epidemiology and Data Science, Amsterdam UMC location Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.
²⁰Division of Hematology and Center for Cell Therapy and Regenerative Medicine, Antwerp University Hospital, Edegem, Belgium.
²¹Department of Hematology and Bone Marrow Transplantation, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris, Université de Paris Cité, Paris, France.
²²The French Society of Bone Marrow Transplantation and Cellular Therapy, Lyon, France.
²³Department of Immunology, Institute of Immunity and Transplantation, University College London, London, United Kingdom.
²⁴Department of Haematology, University College London Hospitals NHS Foundation Trust, London, United Kingdom.
²⁵Clinical Hematology Department, Institut Catala d'Oncologia Hospitalet, Institut d'Investigacions Biomediques de Bellvitge, Universitat de Barcelona, Barcelona, Spain.
²⁶Social Psychology Division, Lyon 2 University, INSERM Unit 1296 Radiations, Bron, France.
²⁷Department of Epidemiology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
²⁸BioSci Consulting, Maasmechelen, Belgium.
²⁹Institut de Recherches internationals Servier, Gif sur Yvette, France.
³⁰University Claude Bernard Lyon 1, Villeurbanne, France.
³¹Service de Biostatistique et Bioinformatique, Pôle Santé Publique, Hospices Civils de Lyon, Lyon, France.
³²CNRS UMR 5558, Laboratoire de Biométrie et Biologie Évolutive, Équipe Biostatistique-Santé, Villeurbanne, France.
³³National Center for Tumor Diseases WERA, Würzburg, Germany.
³⁴Bavarian Center for Cancer Research, Würzburg, Germany.
³⁵Division of Cellular Immunotherapy, Fraunhofer Institute for Cell Therapy and Immunology, Würzburg, Germany.

⁰Department of Hematology, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands.

Blood Advances +

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September 24, 2025

View abstract on PubMed

Summary

Chimeric antigen receptor T-cell (CAR-T) therapy shows favorable general quality of life, but patients report issues with physical, mental, and social well-being. Specific subgroups need targeted follow-up care to address unmet needs.

Area Of Science

Hematology
Oncology
Patient-Reported Outcomes Research

Background

Chimeric antigen receptor T-cell (CAR-T) therapy has transformed hematologic malignancy treatment.
Patient-reported outcomes (PROs) are crucial for assessing CAR-T's long-term impact but remain underreported.
Long-term health-related quality of life (HRQoL), mental well-being, social function, and financial burden post-CAR-T require further investigation.

Purpose Of The Study

To evaluate multidimensional PROs in European patients treated with CAR-T therapy for hematologic malignancies.
To assess long-term HRQoL, mental health, social well-being, and financial burden.
To identify patient subgroups with specific unmet care needs.

Main Methods

Cross-sectional study involving 389 European patients who received CAR-T therapy.
Utilized validated questionnaires (EQ-5D-5L, EORTC-QLQ-C30, PCL-5, modified-iPCQ) and ad hoc items.
Compared outcomes with European general population and a matched CAR-T-naive cohort; analyzed subgroups.

Main Results

Overall HRQoL was comparable or better than reference groups, with exceptions in role, social, and cognitive functioning.
Physical functioning issues were common (41%), especially among women, older patients, and those with neurotoxicity.
Anxiety regarding disease recurrence, infections, and side effects was prevalent; 72% of working-age patients returned to paid work, with younger patients reporting more financial difficulties.

Conclusions

CAR-T therapy is associated with generally favorable HRQoL compared to reference cohorts.
A significant proportion of patients experience persistent physical, mental, and social well-being challenges.
Identifying high-risk subgroups and addressing specific care needs during follow-up is essential for optimizing long-term patient outcomes.

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