Adverse events reported to the U.S. Food and Drug Administration Adverse Event Reporting System for tisagenlecleucel

Abstract The U.S. Food and Drug Administration (FDA) approved the first chimeric antigen receptor T‐cell therapy, tisagenlecleucel, in August 2017. We sought to describe adverse events (AEs) reported FDA Adverse Event Reporting System (FAERS) for tisagenlecleucel post‐marketing period. searched FAERS reports identify patients treated with between 30, 2017‐August 31, 2019. reviewed individual reports, calculated AE frequencies reporting rates (RRs), used Empirical Bayesian Geometric Mean methods disproportionate reporting. identified 646 de‐duplicated a median age at of 18 (interquartile range: 11–56) years. overall RR was 81.0%, more than 95% described serious outcome. Cytokine release syndrome (CRS) most frequently (51.1%) 41.4%; neurotoxicity less (21.2%), 17.2%. Most disproportionately AEs were listed on package insert or confounded by indication. 13 subsequent neoplasms (SPN), majority occurring within 6 months administration, none evidence insertional mutagenesis. A total 165 (26%) death outcome; deaths occurred >30 days after treatment. (64%) due progression underlying lymphoid neoplasm, few (<5%) attributed CRS neurotoxicity. did not new safety concerns lower prelicensure clinical trials.