DP119 | REAL-WORLD SAFETY AND EFFICACY OF CAR-T CELLS THERAPIES FOR RELAPSED/REFRACTORY MULTIPLE MYELOMA: THE CAR-T ITALIAN OBSERVATIONAL STUDY

The treatment of RRMM witnessed the introduction of chimeric antigen receptor T-cell (CAR-T) therapies targeting the B Cell Maturation Antigen (BCMA). However, their efficacy and safety profile stemmed from controlled clinical trials, whose patients’ (pts) characteristics may not adequately mirror the real-life populations. Moreover, pts’ selection and the sequencing of previous and subsequent therapies are not an entirely elucidated process. Thus, real-word evidence validation is warranted. We designed a multicentre, observational, ambispective national study, to explore CAR-T efficacy, safety, and feasibility in the Italian healthcare system. The study is supported by AIL. The overall response rate (ORR) will be the primary study objective. Key secondary objectives will include the rates of complete response (CR), minimal residual disease negativity rates, progression-free and overall survival, manufacturing failures, drop-outs after leukapheresis, as well as the validation of clinical and biological markers predictive of outcomes and toxicities. Safety outcomes including the rates of cytokine release syndrome (CRS), immune effector T cells associated neurotoxicity syndrome (ICANS), cytopenias, infections, and secondary primary malignancies will be recorded. Efficacy of bridging and subsequent therapies will be examined, to inform treatment sequencing. Immunological and molecular predictors of response, toxicity, and resistance will be assessed as exploratory endpoints. As more CAR-T products and extended indications will be approved, their respective efficacy and safety profiles will be compared. Herein, we anticipate an early glance at 20 pts who underwent leukapheresis for standard care idecabtagene vicleucel. Pts’s features are listed in Table 1. Infusion rate was 90% (1 manufacturing failure, 1 clinical deterioration). Best ORR/CR rates were 94% and 50%, respectively. At a median follow up of 5 months (mos), survival data are still immature. Any grade/≥ grade 3 CRS or ICANS occurred in 100%/5% and 11%/0% of pts, respectively; tocilizumab and steroids were required in 94% and 39% of pts, respectively. At one month from infusion, 66% of pts had ≥ grade 3 cytopenia, that resolved by 3 mos in 99% of them. One ≥ grade 3 infection occurred. Our study aims to inform the real-life impact of CAR-T therapies for RRMM. A larger series of patients with extended follow up and granular data will be presented at the meeting.