Strong Real-World Outcomes Reported for Ide-Cel in Relapsed or Refractory MM

Currently, idecabtagene vicleucel (ide-cel) is the standard of care for multiple myeloma (MM), having been the first FDA-approved chimeric antigen receptor T-cell (CAR-T) therapy for this disease in 2021. However, patients with complex conditions or significant comorbidities are often ineligible for studies due to selectivity and eligibility parameters. To evaluate the safety and effectiveness of ide-cel in practice, investigators analyzed data from the Center for International Blood and Marrow Transplant Research registry.

In the largest real-world study of ide-cel for treatment of relapsed or refractory MM to date, Surbhi Sidana, MD, of Stanford University School of Medicine and colleagues evaluated 821 patients receiving ide-cel, with a median follow-up of 11.6 months. Within this cohort, the median age was 66 years (33% aged ≥70 years). Fifteen percent were Black and 7% were Hispanic. At least one major comorbidity was present in 77% of patients. The participants had received a median of seven prior lines of therapy; 15% had previous B-cell maturation antigen (BCMA)–directed treatment. Extramedullary disease was present in 17%, and 27% had high-risk cytogenetics (eg, t(4;14), t(14;16), del(17p)).

The overall response rate was 73%, with a complete response rate of 25 % and median progression-free survival (PFS) of 8.8 months. Treatment-related mortality was reported in 6%, cytokine release syndrome (CRS) in 80% (3% grade ≥3), immune effector cell–associated neurotoxicity syndrome (ICANS) in 28% (5% grade ≥3; no parkinsonism cases). Clinically significant infections were present in 45%, and second primary malignancies, in 4% (1% myeloid).

Patients with controlled disease had higher rates of very good partial response or better at infusion, despite 54% requiring bridging therapy linked to more aggressive disease features, such as a higher International Staging System (ISS) stage or cytopenias. Standard fludarabine/cyclophosphamide lymphodepletion outperformed bendamustine, which was associated with poorer PFS. Any prior BCMA therapy reduced the efficacy of ide-cel.

Ide-cel showed safety and efficacy in this patient population, with real-world outcomes closely aligning with clinical trial data. Findings from this analysis suggest ide-cel’s potential to bridge gaps between clinical trials and clinical practice for patients with heavily pretreated MM, offering robust data to guide individualized treatment decisions in complex cases, particularly when lymphodepletion and disease control must be optimized before infusion.