CAR-T Meeting 2025 | A European study investigating patient-reported outcomes of CAR T-cell therapy

Anne Spanjaart:

In addition to clinical outcomes, patient-reported outcomes are valuable as they give direct insights into how a therapy impacts the well-being of a patient and thereby contribute to more comprehensive therapy evaluations. However, PRO data after CAR-T therapy is still limited, especially long-term PRO data, and certain dimensions such as social well-being are underreported. 

Elise Pennings:

And therefore two European consortia, T2EVOLVE, where I’m working on together with Professor Marie José Kersten and QUALITOP, where Anne is working on together with Professor Marie José Kersten, joined efforts and established a collaborative network of CAR-T treating physicians, patient organisations, CAR-T and health-related quality of life experts, patients and their caregivers to set up a large international cross-sectional survey study to collect patient-reported outcomes among patients who receive CAR T-cell therapy for hematologic malignancy in Europe. And the aim of this study was to better understand patients’ experiences with CAR T-cell treatment, but also to evaluate the impact on their quality of life and to identify unmet care needs. 

And the survey comprised both validated questionnaires and ad hoc items on topics such as disease and treatment characteristics, but also the CAR-T treatment experience, working life, health-related quality of life, stress and anxiety, supportive care, and also information and educational needs. 

And all European adult patients who received CAR-T for any hematologic malignancy could participate, so no specific indication was required, regardless if they received it in a clinical trial or as part of standard of care, and also the time they received CAR T-cell treatment didn’t matter. So really every adult patient could participate. And it was available online from January until October 2023 in seven different languages and it was disseminated by the network we set up. 

Anne Spanjaart:

Yeah, and so for this analysis we used items of the EORTC QLQ-C30, the iPCQ, the modified iPCQ and the EQ-5D. And to place these results into perspective, we compared it with two reference cohorts, the European general population, and a sex- and age-matched cohort of high-grade non-Hodgkin lymphoma patients. And we used established thresholds to determine clinical differences and clinical important problems. We also looked at certain factors associated with health-related quality of life, such as if a patient had progression or not, or experienced neurotoxicity, age, gender, ICU admission. 

So almost 400 patients participated in the study, which is wonderful, making this one of the largest studies on PROs in Europe and showing their willingness to share their experiences with us. And so their median age was 61 years and most patients had a lymphoma and almost a bit more than half of the patients were treated with CAR-T therapy more than one year ago. 

Elise Pennings:

So if we then look at the results, so for example we looked at the mean domain scores of the EORTC QLQ-C30, we saw that in general or overall general health in the CAR-T cohort was even better compared to the non-Hodgkin lymphoma, the matched non-Hodgkin lymphoma cohort, but also compared to the European general population. And if we then zoom in into social well-being, for example looking at role functioning, so work, hobbies, daily and leisure time activities and social functioning, such as social activities and family life, we saw that compared to the matched non-Hodgkin lymphoma cohort, CAR-T patients had a comparable score, but they scored lower compared to the European general population. So social well-being is really something we should pay attention to after CAR T-cell treatment. 

And if we then zoom further in into the prevalence of clinically important problems in the social well-being domains, we saw that 22% reported clinically important problems in role functioning, which was more prevalent among patients who received CAR T-cell therapy within the past three months compared to patients who received CAR T-cell treatment already more than two years ago. And also we saw that 20% of the patients reported clinically important problems in social functioning which was more prevalent, significantly more prevalent, in patients who had experienced neurotoxicity. And then in addition we also looked into financial difficulties, and we saw that in the CAR-T cohort, 28% reported clinically important problems as financial difficulties and these were significantly more prevalent in young patients compared to the older patients above 70. 

Anne Spanjaart:

We also evaluated return to work and a considerable proportion of patients could resume doing paid work after CAR T-cell therapy. 

Elise Pennings:

Yeah, so we saw for example that three out of four patients who had paid work before CAR T-cell treatment could continue paid work after CAR T-cell treatment and more than half could even continue it within the first four months after CAR T-cell treatment. And in addition, we saw that one out of three patients who did not have paid work before CAR T-cell treatment could even start paid work after treatment. So, overall, I think that’s also something remarkable to mention. 

So, overall, we found a general good quality of life compared to the reference cohorts. However, especially in social well-being, we also see a considerable proportion of patients that reported clinically important problems, that deserve attention, we think, in the CAR-T treatment follow-up care plans. 

Anne Spanjaart:

Especially those who experience neurotoxicity or in case of financial difficulties, the younger population. I think this deserves attention in follow-up care plans. 

Elise Pennings:

And something I think that should be mentioned. So PROs are really an important contribution or contribute to more comprehensive therapy evaluations. And can also help us to recognize and monitor these clinical important problems. And enable us to really provide adequate support for the patients in need. 

Anne Spanjaart:

Yeah, and we believe therefore that PROs should be implemented in standard clinical practice, and should be endpoints in clinical trials and real world studies.

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