Ciltacabtagene Autoleucel Improves Long-Term Outcomes in Standard-Risk Relapsed/Refractory Multiple Myeloma

Key Clinical Summary:

• Design/Population: A subgroup analysis of the phase 3 CARTITUDE-4 trial evaluated 59 patients with cytogenetically standard-risk relapsed/refractory multiple myeloma (±1q abnormalities) treated with ciltacabtagene autoleucel in the second- to fourth-line setting, with comparisons to later-line outcomes from CARTITUDE-1.
• Key Outcomes: Cilta-cel demonstrated high response rates with durable progression-free and overall survival, outperforming historical outcomes seen in later-line CAR T-cell therapy.
• Clinical Relevance: Earlier use of cilta-cel in standard-risk myeloma yields deep, durable responses with prolonged survival, supporting its role as a potential one-time, definitive therapy and reinforcing the benefit of earlier CAR T-cell intervention in eligible patients.

Luciano Costa, MD, University of Alabama, Birmingham, Alabama, discusses long-term results from CARTITUDE-4 trial evaluating ciltacabtagene autoleucel in patients with standard-risk multiple myeloma. 

Results demonstrated that ciltacabtagene autoleucel provides profound and durable benefit when used earlier in the treatment course, supporting its role as a potential one-time, disease-controlling therapy in this population.

Dr Costa presented these results at the 2026 Tandem Meetings in Orlando, Florida. 

Transcript:

Hello, my name is Luciano Costa, I'm a myeloma investigator at University of Alabama at Birmingham. At Tandem Meetings in 2026, I had the privilege of presenting on behalf of my coinvestigators, the long-term progression-free survival benefit of ciltacabtagene autoleucel (cilta-cel) in standard risk relapsed/refractory myeloma.

Based on the CARTITUDE-4 trial, we know that cilta-cel provides better progression-free survival and overall survival than the standard of care traditional triplets, namely PVd or DVd, in patients with 1 to 3 prior lines of therapy and lenalidomide-refractory multiple myeloma. This has led to the approval of cilta-cel in as early as second-line therapy in the United States and other countries. As is often the case, we tend to prioritize patients who are perceived as having higher risk disease.

The idea of this presentation was to shed some light on the outcomes of patients with cytogenetically defined standard-risk myeloma treated with cilta-cel. We know from a previously presented subgroup analysis that the standard risk patients had an improvement in progression-face survival and overall survival when treated with cilta-cel as opposed to standard of care as previously presented. Here we want to focus on the patients who actually receive therapy and compare with those patients with the patients who received cilta-cel in later lines of therapy as displayed in the CARTITUDE-1 trial.

There was overall 59 patients with standard risk disease who received cilta-cel. Because the CARTITUDE-1 trial for patients with 3 or more prior lines of therapy included the patients with 1q gain as a single extra abnormality as part of a standard risk, we also want to look at the outcomes of this expanded standard risk with the traditional definition plus 1q. What we saw was that regardless of the definition, the response rate was 100%. A complete response rate was 90% or 92%, whether or not you include the 1q– that looks very favorable compared to what we see in later lines of therapy with 94% response and 82% complete response.

When you look at PFS, the 30 months, 2 and a half years, PFS for standard-risk myeloma treated on CAR-T4 trial was 80.5 months, if we include the patients with 1q abnormalities, that is 72%. The overall survival is 87% and 86% at 2 and a half years. This compares very favorably to patients with standard risk disease treated in later lines therapy where the 2 and a half years progression-free survival is 60% and the 30 months overall survival is 71%.

We also did an interesting analysis looking at the patients who survived the first year, which is the vast majority of the patients, and look at how they fare long-term. In the 2 and a half years, 93% of those patients are progression-free and 94% of those patients are alive. Of the patients who were evaluable for MRD and were MRD-negative, which was the vast majority at the 1-year mark, which is 81%, all of those patients were progression-free and alive at 2 and a half years.

In summary, this data provides very robust evidence of the long-term benefit for cilta-cel being used in earlier therapy, particularly in patients with standard risk disease, for which it gives the possibility of a 1-time definitive therapy in at least some of those patients.

Source:

Costa LJ, Oriol A, Dytfeld D, et al. Long-term progression-free survival benefit with ciltacabtagene autoleucel in standard-risk relapsed/refractory multiple myeloma. Presented at Tandem Meetings; Salt Lake City, Utah. February 4-7, 2026. Abstract 191.