The presence of extramedullary disease (EMD) has been associated with poor outcomes in patients with relapsed-refractory multiple myeloma (RRMM). Herein, we report the outcomes of RRMM patients who were treated with standard-of-care (SOC) chimeric antigen receptor (CAR) T-cell therapy and had active extraosseous EMD before the infusion. Data were retrospectively collected from patients at three US institutions with the intent to receive SOC CAR T. Responses were assessed per the International Myeloma Working Group criteria. A total of 152 patients proceeded with infusion, of whom 47 (31%) had EMD (EMD group) and 105 (69%) did not (non-EMD group). Baseline patient characteristics were comparable between the two groups. The EMD group had a higher incidence of high-grade CRS, steroid and anakinra use, and thrombocytopenia on day +30 compared to the non-EMD group. In addition, the EMD group had an inferior overall response rate (58% vs 96%, p < 0.00001), median progression-free survival (PFS) (5.1 vs 12.4 months; p < 0.0001), and overall survival (OS) (12.2 vs 27.5 months; p = 0.00058) compared to the non-EMD group. We further subdivided the non-EMD patients into those with paramedullary disease (PMD-only group, n = 26 [17%]) and those with neither EMD nor PMD (bone marrow-contained group or BM-only group, n = 79 [52%]). Patients with PMD-only had similar median PFS (11.2 vs 13.6 months, p = 0.3798) and OS (not reached [NR] vs 27.5 months, p = 0.6446) compared to patients with BM-only disease. However, patients with EMD exhibited inferior median PFS (5.1 vs 13.6 months, p < 0.0001) and OS (12.2 vs 27.5, p = 0.0008) compared to patients in the BM-only group. Treatment with SOC CAR T yielded meaningful clinical outcomes in real-world RRMM patients with extraosseous EMD, though responses and survival outcomes were suboptimal compared to patients without EMD. The presence of only EMD but not PMD was associated with significantly worse survival outcomes following the CAR T infusion.
髓外疾病（EMD）的存在与复发难治性多发性骨髓瘤（RRMM）患者的不良预后相关。本文报告了接受标准护理（SOC）嵌合抗原受体（CAR）T细胞治疗且在输注前存在活动性骨外EMD的RRMM患者的预后情况。数据回顾性收集自美国三家计划接受SOC CAR T治疗机构的患者，疗效评估依据国际骨髓瘤工作组标准。共152例患者接受了输注，其中47例（31%）存在EMD（EMD组），105例（69%）无EMD（非EMD组）。两组患者的基线特征具有可比性。与非EMD组相比，EMD组高级别细胞因子释放综合征发生率更高，需使用类固醇和阿那白滞素的情况更多，且第+30天血小板减少症发生率更高。此外，EMD组的总缓解率（58% vs 96%，p < 0.00001）、中位无进展生存期（PFS）（5.1个月 vs 12.4个月；p < 0.0001）和总生存期（OS）（12.2个月 vs 27.5个月；p = 0.00058）均劣于非EMD组。我们进一步将非EMD患者分为仅伴旁髓疾病组（仅PMD组，n = 26[17%]）以及既无EMD也无PMD组（骨髓局限组或仅BM组，n = 79[52%]）。与仅BM组患者相比，仅PMD组患者的中位PFS（11.2个月 vs 13.6个月，p = 0.3798）和OS（未达到[NR] vs 27.5个月，p = 0.6446）相似。然而，与仅BM组患者相比，EMD患者的中位PFS（5.1个月 vs 13.6个月，p < 0.0001）和OS（12.2个月 vs 27.5个月，p = 0.0008）更差。SOC CAR T治疗为真实世界中伴有骨外EMD的RRMM患者带来了有意义的临床结局，但与无EMD患者相比，其缓解率和生存结局欠佳。仅存在EMD（而非PMD）与CAR T输注后生存结局显著更差相关。
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