Real world adverse events of ciltacabtagene autoleucel, a B-cell maturation antigen-directed chimeric antigen receptor T-cell therapy.

Abstract

e19552 Background: Ciltacabtagene autoleucel, a B-cell maturation antigen-directed chimeric antigen receptor T-cell therapy was approved by the FDA in February 2022 for the treatment of adult patients with relapsed or refractory multiple myeloma. The real-world adverse event data about this therapy is limited. The FDA Adverse Event Reporting System (FAERS) is a publicly accessible pharmacovigilance tool developed by FDA to collect adverse event reports from the United States and other countries, offering valuable real-world data. Methods: FAERS was searched for all the adverse events reported for Ciltacabtagene autoleucel and all the reports were analyzed in detail. Results: A total of 1714 adverse events related to ciltacabtagene autoleucel were reported to FAERS from inception through January 2025, with the majority originating from the United States. Of these, 1257 were classified as serious adverse events. Reports increased over time, with 10% submitted in 2022, 35% in 2023, and 54% in 2024. Healthcare professionals accounted for 56.6% of the reports, while 43.4% were submitted by consumers. In terms of patient demographics, 433 adverse events were reported in females, 582 in males, and 699 cases lacked sex specification. The highest number of adverse events occurred in the nervous system disorders category (443 events), followed by immune system disorders (337 events) and infectious diseases (266 events). The most commonly reported adverse event was cytokine release syndrome (296 cases, 17%), followed by pyrexia (124 cases, 7.2%), immune effector cell-associated neurotoxicity syndrome (ICANS) (98 cases, 5.7%), parkinsonism (68 cases, 4%), and neurotoxicity (3.5%). Additionally, a total of 226 cases resulted in death. Conclusions: Cytokine release syndrome, pyrexia, neurotoxicity, and parkinsonism are the most common reported adverse events of Ciltacabtagene autoleucel. While the data highlights patterns of adverse events, long-term studies are necessary to comprehensively understand the safety profile of this therapy in real-world settings. Most common reported adverse events of ciltacabtagene autoleucel. Adverse Event Number of Cases (Total 1714) Percentage 1 Cytokine Release Syndrome 296 17.27% 2 Pyrexia 124 7.23% 3 Immune Effector Cell-Associated Neurotoxicity Syndrome 98 5.72% 4 Parkinsonism 68 3.97% 5 Neurotoxicity 61 3.56% 6 Bell's Palsy 48 2.80% 7 Facial Paralysis 44 2.57% 8 Haemophagocytic Lymphohistiocytosis 44 2.57% 9 Pneumonia 39 2.28%