Lewis Ellen, Jimenez-Zepeda Victor Hugo
Arthur Child Comprehensive Cancer Centre, Calgary, AB T2N 4N2, Canada.
Division of Hematology, Department of Medicine, University of Calgary, Calgary, AB T2N 4N1, Canada.
Curr Oncol. 2025 Jul 25;32(8):418. doi: 10.3390/curroncol32080418.
Light-chain (AL) amyloidosis is a rare clonal plasma cell disorder that, if left untreated, carries a high risk of organ damage and mortality. Due to the rarity of the disease and the vulnerability of affected organ systems, treatment requires significant caution and nuance. As a plasma cell dyscrasia, AL amyloidosis treatment regimens are often adapted from those used for related disorders, particularly multiple myeloma. Despite substantial progress in research and drug development, optimal treatment strategies for relapsed/refractory (RR) AL amyloidosis remain unclear, and no FDA-approved therapies currently exist for this setting. B-cell maturation antigen (BCMA) has emerged as a promising immunotherapy target, with associated drug classes including antibody-drug conjugates, bispecific antibodies, and CAR-T cell therapies. These therapies have been extensively studied in relapsed/refractory multiple myeloma (RRMM) and are now being explored in the context of RR AL amyloidosis. This review summarizes the current literature on the efficacy and tolerability of BCMA-directed therapies in AL amyloidosis, with a particular emphasis on CAR-T cell therapy and offers comparisons to outcomes observed in RRMM.
轻链(AL)淀粉样变性是一种罕见的克隆性浆细胞疾病,如果不进行治疗,会有很高的器官损害和死亡风险。由于该疾病的罕见性以及受累器官系统的脆弱性,治疗需要格外谨慎并注重细节。作为一种浆细胞发育异常疾病,AL淀粉样变性的治疗方案通常借鉴用于相关疾病(尤其是多发性骨髓瘤)的方案。尽管在研究和药物开发方面取得了重大进展,但复发/难治性(RR)AL淀粉样变性的最佳治疗策略仍不明确,目前尚无FDA批准的针对这种情况的疗法。B细胞成熟抗原(BCMA)已成为一种有前景的免疫治疗靶点,相关药物类别包括抗体药物偶联物、双特异性抗体和嵌合抗原受体T细胞(CAR-T)疗法。这些疗法已在复发/难治性多发性骨髓瘤(RRMM)中得到广泛研究,目前正在RR AL淀粉样变性的背景下进行探索。本综述总结了目前关于BCMA导向疗法在AL淀粉样变性中的疗效和耐受性的文献,特别强调了CAR-T细胞疗法,并与RRMM中观察到的结果进行了比较。