Lisocabtagene maraleucel in chronic lymphocytic leukaemia and small lymphocytic lymphoma (TRANSCEND CLL 004): a multicentre, open-label single-arm, phase 1-2 study

Primary analysis results for the TRANSCEND CLL 004 were presented at the 2023 ASCO Annual Meeting, with the full report published in the Lancet. This study examined two dose levels of lisocabtagene maraleucel (liso-cel), a CAR-T cell therapy, for adults with relapsed or refractory CLL who had experienced treatment failure with a BTK inhibitor and one other therapy, such as venetoclax. Of the 117 patients in the trial, 70 had experienced treatment failure with both BTKi and venetoclax. Higher dose level of liso-cel CAR-T cell therapy demonstrated best results, with a complete response rate of 18% (n = 9; 95% CI: 9-32; p = 0.0006 compared to lower dose liso-cel CAR-T). There were 51 total deaths: 43 after liso-cel infusion, of which five were within 90 days of infusion and due to treatment-emergent adverse events, and one due due macrophage activation syndrome-haemophagocytic lymphohistiocytosis. A total of 10 patients experienced grade 3 cytokine release syndrome, and 21 patients experienced grade 3 neurologic events.