In a recent study published in Blood, researchers analyzed outcomes from the use of ciltacabtagene autoleucel (cilta-cel), a chimeric antigen receptor (CAR)-T therapy for multiple myeloma, in real-world settings. The study included 236 patients who received cilta-cel infusions at 16 U.S. medical centers in 2022. Results showed that 89% of patients responded to the treatment, with 70% achieving a complete response. These response rates were comparable to those seen in the phase II CARTITUDE-1 trial that led to cilta-cel’s FDA approval. More importantly, over half of the patients in the new study would have been ineligible for CARTITUDE-1, highlighting the therapy’s effectiveness in a broader patient population.
Multiple myeloma is a cancer that affects plasma cells, and the prognosis is poor for patients who are refractory to standard treatments or have relapsed disease. CAR-T therapies have shown promise in these patients, and cilta-cel was approved in 2022 for heavily pre-treated patients. The study focused on patients who had undergone multiple lines of therapy without lasting responses. Of the 255 patients who started receiving cilta-cel, 236 completed treatment. Researchers found that patients who met FDA quality standards had higher response rates compared to those who did not, suggesting the importance of adherence to treatment protocols.
In addition to evaluating response rates, researchers examined outcomes in subgroups of patients. Those who had received prior therapies targeting B cell maturation antigen (BCMA) showed lower response rates, especially if the prior therapy was more recent. This indicates that further studies are needed to understand how the timing of cilta-cel and other BCMA-targeted therapies may impact outcomes. The study also identified patient and disease characteristics associated with lower survival rates or disease progression, providing insights for personalized treatment approaches.
The study reported rates of serious side effects comparable to previous clinical trials, with most patients experiencing cytokine release syndrome (CRS). However, severe CRS events were rare, and rates of neurotoxicity were relatively low. Delayed neurotoxicity, specific to cilta-cel, was observed in 10% of patients, highlighting the need for continued monitoring and management of side effects. Additionally, there was a relatively high rate of non-cancer-related deaths, mainly due to infections or CRS, suggesting opportunities for improvement in infection prevention and CRS management.
As a retrospective, real-world study, the research did not include a control group, and there might have been variations in outcomes assessment among the contributing centers. The findings indicated the need for further studies to optimize treatment protocols and reduce serious side effects. The researchers also suggested exploring the potential benefits of using cilta-cel earlier in the course of treatment to minimize toxicity risks. Overall, the study provided valuable insights into the real-world outcomes of cilta-cel therapy for multiple myeloma patients, demonstrating the therapy’s effectiveness and safety in a broader patient population.
