CAR T-Cell Therapy Is Safe, Effective in Minorities, Study Finds
The real-world analysis addresses a clear gap in data from previous and ongoing cell therapy clinical trials.
Investigators from Montefiore Einstein Cancer Center recently published findings from a real-world retrospective cohort study that showed similar efficacy with CAR T-cell therapy in black and Hispanic patients as their white and Asian counterparts.1
The findings, published in Bone Marrow Transplantation, help address gaps in knowledge of safety and efficacy in these patient populations not addressed by the major clinical trials used to approve and/or currently study these breakthrough therapies.
Notably, late-stage clinical trials used to support approval of several CAR-T candidates, including tisa-cel, axi-cel, and liso-cel, as well as those currently ongoing such as ZUMA-7, which is examining axi-cel in the second-line setting, have failed to disclose efficacy and safety data for minority patients as part of their readouts.
“Minorities cannot rely on extrapolation from trials with only Caucasian patients, as disease biology of B-cell malignancies and side effect profiles for immune-related adverse events are known to vary in different ethnic backgrounds,” the study authors wrote.
In order to better characterize outcomes in these underserved patient populations, investigators led by Asha Thakkar, MD, of the department of oncology at Montefiore Einstein Cancer Center, Blood Cancer Institute, compared outcomes between 26 minority patients (17 Hispanic, 9 African American) and 20 non-minority patients who received CAR T-cell therapy between 2015 and 2021. The majority of patients in the minority group had a diagnosis of diffuse large B-cell lymphoma (DLBCL; 92%), while 1 patient had follicular lymphoma and 1 patient and mantle cell lymphoma (MCL). In the nonminority group, 85% had a diagnosis of DLBCL and 15% had a diagnosis of MCL. Overall, 25 and 17 patients in the minority and nonminority groups, respectively, received treatment with axi-cel, with the remaining receiving treatment with brexucabtageneautoleucel.
At first imaging post-infusion, 58% of minority patients achieved a complete response (CR), 19% a partial response (PR), and 23% experienced disease progression compared with 70% of patients in the nonminority group who achieved a CR, 20% PR, and 10% disease progression.
An analysis of progression-free survival (PFS) of 43 of the patients revealed no difference between the 2 groups. Overall survival was 46.3 months in the minority group and was not reached in the nonminority group. Further analysis based on progression of disease showed that the cumulative incidence of progression of disease and death were similar in both groups.
Similar rates of toxicities were observed across the groups, with 96% and 95% of patients in the minority and nonminority groups experiencing cytokine release syndrome, and 92% and 95% experiencing grade 0-2 immune effector cell associated neurotoxicity syndrome, respectively.
Echoing recent guidance from the FDA about increasing diversity in the clinical trial process, the authors emphasized the importance of inclusion of these populations in future clinical studies, especially as CAR T-cell therapies begin to move up in the treatment continuum.
“Representation in cancer clinical trials is vital to ensuring that treatments are safe and effective for everyone,” said co-author Mendel Goldfinger, MD, assistant professor of medicine at Albert Einstein College of Medicine, and member of the MECC Cancer Therapeutics Program, in a statement.2 “We couldn’t have been happier to learn that our patients who identify as Black and Hispanic have the same benefits from CAR-T therapy as white patients. We can only begin to say that a cancer treatment is transformational when these therapies benefit everyone who comes to us for care.”
