法国早期全国性 idecabtagene vicleucelCAR-T 细胞治疗复发/难治性多发性骨髓瘤患者经验（FENIX）：来自 DESCAR-T 注册登记的一项 IFM 真实世界研究

Idecabtagene vicleucel (ide-cel), a chimeric antigen receptor T-cell therapy targeting B-cell maturation antigen (BCMA), received early access program (EAP) authorization in France in April 2021 for relapsed/refractory multiple myeloma (RRMM).

We conducted a real-world registry-based multicentre observational study in 11 French hospitals to evaluate ide-cel outcomes. Data from 176 RRMM patients who underwent apheresis between June 2021 and November 2022 were collected from the French national DESCAR-T registry. Of these, 159 patients (90%) received ide-cel. Cytokine release syndrome occurred in 90% with 2% grade ≥3, and neurotoxicity occurred in 12% with 3% grade ≥3. Over the first 6 months, the best overall response and ≥complete response rates were 88% and 47% respectively.

The median progression-free survival (PFS) from the ide-cel infusion was 12. 5 months, the median overall survival (OS) was 20. 8 months and the estimated OS rate at 12 months was 73. 3%. Patients with extra-medullary disease (EMD) had impaired PFS (6. 2 months vs. 14. 8 months). On multivariable analysis, EMD and previous exposure to BCMA-targeted immunoconjugate or T-cell-redirecting GPRC5D bispecific antibody were associated with inferior PFS.

Our study supports ide-cel's feasibility, safety and efficacy in real-life settings, emphasizing the importance of screening for EMD and considering prior treatments to optimize patient selection.