Department of Internal Medicine, Cleveland Clinic, Cleveland, Ohio, USA.
Cleveland Clinic Taussig Cancer Center, Cleveland Clinic, Cleveland, Ohio, USA.
Eur J Haematol. 2024 Dec;113(6):817-823. doi: 10.1111/ejh.14293. Epub 2024 Aug 27.
Clinical trials evaluating chimeric antigen receptor (CAR) T-cell therapy in relapsed/refractory multiple myeloma (RRMM) have typically excluded patients with AL amyloidosis. As a result, there are limited data on the safety and efficacy of CAR T-cell therapy in this patient population. We retrospectively reviewed eight consecutive patients with RRMM and AL amyloidosis who were treated with standard of care CAR T-cell therapy. Cytokine release syndrome was seen in 75% of patients (grade ≥3: 0%) and immune effector cell-associated neurotoxicity syndrome (grade 1) in only one patient. Low-grade cytopenias were common (any grade/grade ≥3: neutropenia 62.5%/37.5%, anemia 37.5%/0%, thrombocytopenia 25%/0%). CAR T-cell therapy led to rapid and deep responses with a median time to best response of 43 days and a hematologic very good partial response or better rate of 62.5%. Overall, we found that commercial CAR T-cell therapy was feasible, and effective in patients with RRMM and concurrent AL amyloidosis.
临床研究评估嵌合抗原受体 (CAR) T 细胞疗法在复发/难治性多发性骨髓瘤 (RRMM) 中的疗效，通常排除了有 AL 淀粉样变性的患者。因此，针对该患者人群，CAR T 细胞疗法的安全性和疗效数据有限。我们回顾性分析了 8 例连续接受标准 CARE CAR T 细胞疗法治疗的 RRMM 合并 AL 淀粉样变性患者。75%的患者出现细胞因子释放综合征（≥3 级：0%），仅 1 例出现免疫效应细胞相关神经毒性综合征（1 级）。常见低级别血细胞减少症（任何级别/≥3 级：中性粒细胞减少症 62.5%/37.5%、贫血症 37.5%/0%、血小板减少症 25%/0%）。CAR T 细胞疗法可迅速产生深度应答，最佳反应中位时间为 43 天，血液学非常好的部分缓解或更好的缓解率为 62.5%。总体而言，我们发现商业 CAR T 细胞疗法对 RRMM 合并 AL 淀粉样变性患者是可行且有效的。
