Authored by Ann Liu, PhD
Medically Reviewed by Brian Koffman, MDCM )retired), MSEd
The Bottom Line:
In the real world, patients with CLL treated with liso-cel (CAR-T) have response rates that are just as good or even higher than those seen in clinical trials.
Who Performed the Research and Where Was it Presented:
Dr. Emily Tomasulo from the University of Pennsylvania and colleagues presented the results at the American Society of Clinical Oncology (ASCO) Annual Meeting in 2026.
Background:
Chimeric antigen T cell receptor (CAR-T) therapy is a one-time cellular immune treatment that takes T cells (a type of white blood cell) from a patient’s blood, reengineers them ex-vivo (outside the body), and re-injects them to recognize and attack cancer cells. Use of CAR-T therapy in chronic lymphocytic leukemia (CLL) / small lymphocytic lymphoma (SLL) has faced hurdles due to inferior response rates, higher risk of side effects, and shorter response duration compared with other B-cell cancers. In 2024, liso-cel (lisocabtagene maraleucel)became the first CAR-T product approved for use in relapsed / refractory CLL / SLL, specifically for patients who have already been treated with a BTK inhibitor and a BCL2 inhibitor. For this study, researchers used real-world data to evaluate the overall efficacy and safety of liso-cel therapy in patients with relapsed / refractory CLL.
Methods and Participants:
This was an observational study using data from a sample of patients with relapsed / refractory CLL in the United States who were treated with liso-cel between May 2024 and September 2025.
Results:
- A total of 45 patients were included in the study, half of whom were younger than 62 years old and all were functioning well.
- Patients received a median of six prior therapies, and 71% received liso-cel as a fifth or later line of therapy.
- 84% of patients had previously received both a covalent BTK inhibitor and a BCL2 inhibitor.
- 47% of patients had previously been treated with noncovalent BTK inhibitor pirtobrutinib.
- 48% of patients used a bridging therapy, which is a treatment used to keep the disease under control while the CAR-T cells are being made.
- With a median of six months of follow-up, 84% of patients had their cancer shrink, and 55% had had their blood counts and lymph nodes return to normal (complete response).
- Among patients who had a complete response, 81% also had undetectable measurable residual disease.
- Six months after treatment, 77% of patients were progression-free, and 87% were still alive.
- Cytokine release syndrome (80%) and immune effector cell–associated neurotoxicity syndrome (36%), which are known side effects of immune therapies, were common but not life-threatening.
- 75% of patients required a planned or urgent hospitalization at the time of or within several weeks of treatment
- 40% of patients experienced a clinically significant infection.
- There were five deaths: two due to disease progression, two due to infection, and one due to multiple causes.
Conclusions:
This is the second real-world study showing that patients with CLL treated with liso-cel (CAR-T) have response rates that are just as good or even higher than those seen in clinical trials. Another analysis of real-world data found that patients with CLL treated with liso-cel had very similar overall response rates and complete response rates to those seen here. This is surprising given that clinical trial results are usually better than those seen in the real world, and this suggests that CAR-T is an effective therapy for relapsed / refractory CLL. It is heartening that there is now an effective therapy for those who have failed all available targeted therapies, although the toxicity of CAR-T remains a challenge. Real-world results over a longer period of follow-up and among older or less independent patients remain uncertain.
Links and Resources:
You can read the actual ASCO abstract here: Real-world outcomes of lisocabtagene maraleucel (liso-cel) in patients (pt) with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL): First results from CIBMTR
Take care of yourself first.
Ann Liu, PhD
