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B 细胞成熟抗原靶向 CAR-T 细胞治疗在复发/难治性多发性骨髓瘤合并轻链淀粉样变性患者中的安全性与疗效

英文原题:Safety and efficacy of B cell maturation antigen-directed CAR T-cell therapy in patients with relapsed/refractory multiple myeloma and concurrent light chain amyloidosis.

查看英文原题

Safety and efficacy of B cell maturation antigen-directed CAR T-cell therapy in patients with relapsed/refractory multiple myeloma and concurrent light chain amyloidosis.

PubMed 2024/08/27(内容时间) Eur J Haematol Q2 · IF 2.6(JCR 2025)

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中文摘要

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)中的临床试验通常排除了AL淀粉样变性患者。因此,关于CAR-T 细胞疗法在该患者群体中的安全性和疗效的数据有限。我们回顾性分析了8例连续接受标准治疗CAR-T 细胞疗法的RRMM合并AL淀粉样变性患者。细胞因子释放综合征见于75%的患者(3级:0%),免疫效应细胞相关神经毒性综合征(1级)仅见于1例患者。低级别血细胞减少常见(任何级别/3级:中性粒细胞减少62.5%/37.5%,贫血37.5%/0%,血小板减少25%/0%)。CAR-T 细胞疗法带来了快速且深度的缓解,中位最佳缓解时间为43天,血液学非常好的部分缓解或更好率为62.5%。总体而言,我们发现商业化CAR-T 细胞疗法在RRMM合并AL淀粉样变性患者中是可行的且有效的。

展开英文摘要原文

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.

论文信息

作者
Goel U、Dima D、Davis J、Ahmed N、Shaikh H、Lochner J、Abdallah AO、Khouri J
第一作者单位
Department of Internal Medicine, Cleveland Clinic, Cleveland, Ohio, USA.United States
通讯作者单位
Cleveland Clinic Taussig Cancer Center, Cleveland Clinic, Cleveland, Ohio, USA.United States
期刊
European journal of haematology2024 Dec
原文标识
PubMed 39189919 · DOI 10.1111/ejh.14293