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. Mazyar Shadman from the Fred Hutch Cancer Center and colleagues presented the results at the American Society for Hematology (ASH) Annual Meeting in 2025.
Background:
Chimeric antigen receptor T-cell (CAR-T) therapy reprograms your body’s own immune cells to recognize and destroy cancer cells. Lisocabtagene maraleucel (liso-cel) is the only CAR-T therapy that the FDA approves for treating chronic lymphocytic leukemia (CLL) / small lymphocytic lymphoma (SLL). While clinical trials are critical for testing new therapies and getting them approved, they often have strict inclusion and exclusion criteria, which may not be representative of the general population of CLL / SLL patients. In this study, researchers used real-world data to look at the efficacy and safety of liso-cel in patients with CLL.
Methods and Participants:
This clinical trial was a retrospective study using real-world data from multiple institutions. It included patients with CLL who received liso-cel in the United States. Patients with Richter transformation were excluded.
Results:
- A total of 30 patients were included in the study, and 67% had deletion 17p or TP53 mutation.
- Patients had received a median of six prior lines of therapy, and 80% had previously been treated with chemotherapy.
- All patients have previously been treated with a covalent BTK inhibitor and a BCL2 inhibitor.
- 60% of patients were treated with pirtobrutinib as their last line of treatment before liso-cel.
- The overall response rate (how many patients had their cancer shrink) was 83%, and the complete response rate (how many patients had no detectable signs of cancer) was 60%.
- Patients who had pirtobrutinib as their last line of therapy were more likely to achieve a complete response (72% vs 28%).
Conclusions:
In the real world, patients with CLL treated with liso-cel have response rates that are as good as or even higher than those seen in clinical trials. Using BTK inhibitors before and even after CAR-T infusion appears to improve efficacy. While the follow-up time in this analysis was short, researchers will continue to follow patients to determine the durability of these responses. There are two big takeaways from this research:
- CAR-T results were surprisingly as good and maybe better in the real world compared to in clinical trials. The reverse is almost always true with the patients in trials doing the best. This suggests CAR-T is an efficcacous and probably underused treatment in R/R CLL in the clinic.
- The combination of BTKi with CAR-T significantly improves outcomes for CLL patients, suggesting that most patients should use this combination rather than CAR-T monotherapy.
Links and Resources:
Watch the interview on the abstract here:
You can read the actual ASH abstract here: Superior real-world outcomes of lisocabtagene maraleucel in chronic lymphocytic leukemia
