CAR T in Heme Malignancies: Patient Outcomes Inform Care Needs

A new observational study in Europe revealed that quality of life (QOL) among patients receiving chimeric antigen receptor T-cell therapy (CAR T) for hematologic malignancies largely matched that of the European general population.¹ Nevertheless, a sizable proportion still reported physical, mental, and social challenges, necessitating additional tailored supportive care to address ongoing functional limitations and psychosocial needs.

A key highlight from the study, which captured survey responses from nearly 400 patients, was that mean health-related QOL was found to be similar or better (73.8; standard deviation [SD], 18.7) compared with reference cohorts despite known toxicities associated with CAR T.

Improved QOL was particularly noted in patients who were more than 2 years out from CAR T infusion (79.0; SD, 16.0) and those whose disease had not progressed (75.1; SD, 17.9) vs those who had undergone infusion more recently (70.3; SD, 17.8) and those who had disease progression (70.6; SD, 20.0).

Additional Findings: Functional Challenges Persist

Although general well-being was favorable, the study also showed that patients also experienced ongoing functional challenges compared with the general population. Overall, slightly less than half (41%) reported problems in physical functioning, with common symptoms including dyspnea (38%), pain (33%), and fatigue (28%). Problems in cognitive functioning were noted by 34% of patients.

In terms of mental well-being, anxiety about disease recurrence, infections, and long-term adverse effects were a persistent concern, often affecting patients’ daily lives. Approximately 29% of patients indicated that they would have appreciated more support throughout the CAR T process, specifically mental support, which could help mitigate the anxieties and uncertainties during and after CAR T.

Approximately 22% of patients reported difficulties with social and role functioning, such as in the workplace and in leisure activities, with these challenges more common among patients closer to their CAR T infusion compared with those more than 2 years posttreatment (30% vs 12%).

The survey also uncovered challenges faced by certain subgroups. For instance, women, older patients, and patients who experienced neurotoxicities reported significantly more problems in physical functioning. Additionally, younger patients reported experiencing more financial difficulties compared with older patients (32% vs 9%).

Clinical Implications

Although the study concentrated on patients in western Europe, which potentially limits generalizability to other geographic regions, the findings provide important real-world insights into long-term QOL of CAR T recipients, particularly in identifying persistent unmet needs and vulnerable subgroups. These insights are especially applicable to patients in the early postinfusion period, when physical, mental, and social challenges appear most pronounced.

Considering the findings, the authors stress the importance of patient-reported outcomes (PROs) in capturing the full impact of CAR T beyond traditional clinical end points and advocate for incorporation of PROs in research and clinical practice to ensure the overall well-being of patients.

“In addition to offering a more holistic evaluation of risks and benefits of therapies, PRO measurements can be used to recognize and monitor these clinical problems in individuals and identify subgroups at risk, enabling adequate support,” authors Pennings et al wrote in the Blood Advances publication.¹ “Therefore, alongside clinical outcomes on efficacy and toxicity, PROs should be integrated as a core outcome in future clinical trials, real-world studies, and routine practice.”

Study Design and Patient Population

This cross-sectional study was designed to understand and better characterize patients’ experiences with CAR T, particularly in the European context.

PROs were collected through an online survey available from January to October 2023. The survey consisted of validated questionnaires, such as the EORTC Quality of Life Questionnaire Core 30 and the EuroQol 5-dimension 5-level Questionnaire to assess health-related QOL, as well as other ad hoc items.

All European adult patients at least 18 years of age who had received CAR T as treatment for a hematologic malignancy were eligible for participation. A total of 389 patients from across 10 European countries responded to the survey and were included for analysis. The median age of respondents was 61 years (range, 18–55), most (86%) had lymphoma, and slightly more than half (56%) were over 1 year post-CAR T.

The survey cohort was then compared against 2 reference cohorts. The first cohort consisted of 11,343 individuals from across 11 European countries, representing the European general population; the second cohort was an age- and sex-matched cohort of patients with high-grade non-Hodgkin lymphoma who were treated with treatments other than CAR T, such as chemotherapy or immunotherapy.

REFERENCES

1. Pennings ER, Spanjaart AM, Thielen FW, et al. Patient-reported outcomes in patients with hematologic malignancies treated with CAR T-cell therapy in Europe. Blood Advances. 2025;9(24):6380-6393. doi:10.1182/bloodadvances.2025017081