ASH 2024 | Real-world outcomes of CAR T-cell therapy in RRMM

We all know that CAR T-cell therapy has revolutionized treatment of many relapsed refractory hematological malignancies. There is enough data now supporting that CAR T-cell therapy has significantly improved the progression-free survival and overall survival of many relapsed/refractory hematological malignancies and, importantly, relapsed/refractory multiple myeloma. In heavily pre-treated patients, it almost tripled the progression-free survival and the overall survival was as high as 18 months. Median overall survival was as high as 18 months in various randomized controlled trials. But then in randomized controlled trials, you have a controlled setting where everyone listed for CAR-T ends up getting CAR-T, but this is not what happens in real-world. A lot of patients who are listed for CAR-T have to face lengthy wait times and they may not end up getting CAR-T. They may have disease progression while waiting for CAR-T, requiring alternate modes of treatment. So in this study, what we wanted to do was see what our single center experience is with CAR T-cell therapy, see what the outcomes are in patients who get CAR-T versus those who do not get CAR-T even after being listed for CAR-T. So for this, what we did was we identified all the patients in our center with relapsed/refractory multiple myeloma who were listed for CAR T-cell therapy and saw what their outcomes were. So we identified a total of 152 patients in our center who were listed for CAR T-cell therapy and we saw that close to 50% of the patients ended up getting CAR-T at a median follow-up of 15 months. So again, half of the patients did not get CAR-T. The number one reason for not getting CAR-T was because they had disease progression while waiting for CAR-T requiring use of alternate forms of therapy. Most commonly used alternate forms of therapy included either cyclophosphamide-based regimens or daratumumab-based regimens. When we looked at the overall outcomes, the overall response rate, including all the patients who were listed for CAR-T, was close to 50%. The overall response rate was higher in the CAR-T group, where the overall response rate was 63% compared to 39% in patients who had alternate forms of therapy. So that was the main difference that we found. Overall, CAR-T caused CRS, cytokine release syndrome, in close to 90% of the patients, and neurotoxicity was seen in close to 15% of the patients. Cytopenias were fairly frequent, but thrombocytopenia, anemia, and neutropenia, they were fairly frequent. So that was the safety-related aspect of the CAR T-cell therapy. Coming to the efficacy, as I mentioned, the overall response rate was close to 50%. The progression-free survival in patients in the CAR-T group was close to 12 months, and the overall survival was not reached at the end of follow-up, compared to 5.4 months in the non-CAR-T group and an overall survival of 15 months. So that was very different. What we concluded with this study was that more than half of the patients who are listed for CAR-T do not get it. And the reason this study is different from the other real-world experience is that we are including all the patients who are listed for CAR-T. So we are going by intention to treat cohort instead of what the patient ends up getting, the half of the patients who actually end up getting CAR-T.

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