CARTITUDE-1 患者与既往暴露于蛋白酶体抑制剂、免疫调节药物和抗 CD38 抗体的多发性骨髓瘤患者之间的结局调整比较：来自前瞻性、多国 LocoMMotion 真实世界临床实践研究

Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor T-cell therapy studied in patients with multiple myeloma exposed to three classes of treatment in the single-arm CARTITUDE-1 study. To assess the effectiveness of cilta-cel compared to real-world clinical practice (RWCP), we performed adjusted comparisons using individual patients' data from CARTITUDE-1 and LocoMMotion, a prospective, multinational study of patients with multiple myeloma triple-class exposed of treatment. Comparisons were performed using inverse probability weighting. In CARTITUDE-1, 113 patients were enrolled, and 97 patients were infused with cilta-cel. In LocoMMotion, 248 patients were enrolled, and 170 patients were included in the comparisons versus infused patients. Ninety-two unique regimens were used in LocoMMotion, most frequently carfilzomib-dexamethasone (13. 7%), pomalidomide-cyclophosphamide-dexamethasone (13. 3%) and pomalidomidedexamethasone (11. 3%). Adjusted comparisons showed that patients treated with cilta-cel were 3. 12-fold more likely to respond to treatment than those managed by RWCP (response rate, 3.

12, 95% confidence interval [95% CI]: 2. 24-4. 00), had their risk of progression or death reduced to by 85% (progression-free survival hazard ratio=0. 15, 95% CI: 0. 08-0. 29), and a risk of death lowered by 80% (overall survival hazard ratio HR=0. 20, 95% CI: 0. 09-0. 41). The incremental improvement in healthrelated quality of life from baseline for cilta-cel versus RWCP at week 52, as measured by EORTC QLQ-C30 Global Health Status, was 13. 4 (95% CI: 3. 5-23. 6) and increased to 30. 8 (95% CI: 21. 8-39. 8) when including death as additional information regarding patients' health status.

Patients treated with cilta-cel experienced more adverse events than those managed with RWCP (any grade: 100% vs. 83. 5%). The results from this study demonstrate improved efficacy outcomes of cilta-cel versus RWCP and highlight its potential as a novel and effective treatment option for patients with multiple myeloma triple-class exposed of antimyeloma treatment. CARTITUDE-1 is registered with clinicaltrials gov. Identifier: NCT03548207. LocoMMotion is registered with clinicaltrials gov. Identifier: NCT04035226.