Medically reviewed by Dr. Shani S. Saks, D.O. on August 2, 2023
Ide-cel therapy significantly improved response and progression-free survival compared to standard regimens in triple-class-exposed relapsed and refractory multiple myeloma patients.
Relapsed and refractory multiple myeloma (MM) patients with triple-class exposure display suboptimal responses to standard therapies, leading to poor survival outcomes. Idecabtagene vicleucel (ide-cel), a B-cell maturation antigen-directed chimeric antigen receptor (CAR) T-cell therapy, demonstrated deep and durable responses in triple-class-exposed, heavily pretreated relapsed and refractory MM patients.
A phase III study in The New England Journal of Medicine compared the effectiveness of ide-cel therapy with standard regimens in treating triple-class-exposed relapsed and refractory MM.
Study Population
A total of 225 patients received ide-cel infusion therapy, and 126 received the standard regimen. Efficacy evaluation in the intention-to-treat population included 29 patients from the ide-cel groups and 6 patients from the standard-regimen group who did not receive the assigned therapy. Baseline patient characteristics were comparable between the groups, except for Black patients, of whom 7% were assigned to the ide-cel group and 14% were assigned to the standard regimen group.
Prolonged Progression-Free Survival With Ide-Cel Treatment
At a median follow-up of 18.6 months, 242 events of disease progression or death were observed. In the intention-to-treat population, progression-free survival (PFS) was significantly longer in the ide-cel group than in the standard-regimen group (P<0.001). The 6-month PFS was 73% in the ide-cel group and 40% in the standard-regimen group, while the 12-month PFS was 55% and 30%, respectively. PFS benefit with ide-cel was observed in all subgroups.
High Response Rates With Ide-Cel Therapy
A significantly higher percentage of patients demonstrated a response with ide-cel therapy compared to standard regimens. A total of 71% of patients in the ide-cel group and 42% in the standard-regimen group had a partial response or better (P<0.001). Complete response or stringent complete response was also higher with ide-cel than standard regimens (39% vs. 5%, respectively). The median duration of response was 14.8 months in the ide-cel group and 9.7 months in the standard regimen group.
High Incidence of Adverse Events in Both Treatment Groups
The safety profile of ide-cel was consistent with previous reports. Adverse events were reported in 99% of patients in the ide-cel group and 98% in the standard-regimen group, with grade 3 or 4 events reported in 93% and 75%, respectively, and grade 5 events in 14% and 6%, respectively. Hematologic adverse events were more frequent in the ide-cel group (78%) than the standard-regimen group (44%). Serious adverse events were reported at 52% in the ide-cel group and 38% in the standard-regimen group. Among the 225 patients who received ide-cel, cytokine release syndrome occurred in 88%, with mostly grade 1 or 2 and 5% having a grade 3 or higher event, and neurotoxic effects were observed in 15%, with 3% being grade 3 or higher.
Source:
Rodriguez-Otero, P., Ailawadhi, S., Arnulf, B., Patel, K., Cavo, M., Nooka, A. K., Manier, S., Callander, N. S., Costa, L. J., Vij, R., Bahlis, N. J., Moreau, P., Solomon, S. R., Delforge, M., Berdeja, J. G., Truppel-Hartmann, A., Yang, Z., Favre-Kontula, L., Wang, F., . . . Giralt, S. (2023). Ide-CEL or standard regimens in relapsed and refractory multiple myeloma. The New England Journal of Medicine, 388(11), 1002–1014. https://doi.org/10.1056/nejmoa2213614
