EHA 2025 Insights: CARTITUDE-1 Trial Updates on Long-Term Efficacy and Safety of CAR T in Late R/R MM

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The CARTITUDE-1 trial 5-year follow-up data presented at the European Hematology Association (EHA) 2025 Congress demonstrate remarkable long-term efficacy for CAR T-cell therapy in patients with relapsed/refractory (R/R) MM. Approximately one-third of patients remained drug free and myeloma free at 5 years post treatment, with all 12 patients from one institution maintaining minimal residual disease negativity. These patients had received an average of 6 prior therapy lines, with some exceeding 10 previous treatments, highlighting CAR T’s effectiveness in heavily pretreated populations.

Safety management has significantly improved through enhanced patient selection protocols and refined toxicity mitigation strategies. Physicians now implement aggressive bridging therapy protocols, with some patients receiving up to 4 bridging treatments to optimize their condition before CAR T infusion. Advanced monitoring techniques, including ALK surveillance as a surrogate marker for T-cell expansion, enable early intervention with steroids to prevent severe toxicities. Although rare adverse events such as immune-mediated colitis continue to emerge as more patients receive treatment, these occur in fewer than 1 in 1000 cases.

The treatment-related mortality rate remains at approximately 5% between 2 to 4 months post infusion, though individual institutions report rates below 2%. Neurotoxicity, although delayed compared with other CAR T products, typically occurs around day 7 and lasts a median of 2 days. Comprehensive patient counseling addresses concerns about rare complications, including secondary malignancies and the widely publicized case of T-cell lymphoma, emphasizing the importance of informed consent and shared decision-making in CAR T patient selection.