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 TRANSCENT 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 adult patients 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, all had experienced treatment failure of a BTK inhibitor, and 70 had also experienced venetoclax failure. The complete response rate was found to be statistically significant at 18% (n = 9; 95% CI: 9-32; p = 0.0006) for those patients who received the higher dose level of 100 x 106 of liso-cel. 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.