Hematology Department, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Hospital Clinic de Barcelona, Barcelona, Spain.
Amyloidosis and Multiple Myeloma Unit, Hospital Clinic de Barcelona, Barcelona, Spain.
J Immunother Cancer. 2021 Dec;9(12). doi: 10.1136/jitc-2021-003783.
Multiple myeloma (MM) remains incurable despite the number of novel therapies that have become available in recent years. Occasionally, a patient with MM will develop an amyloid light-chain (AL) amyloidosis with organ dysfunction. Chimeric antigen receptor T-cell (CART) therapy has become a promising approach in treating hematological malignancies. Our institution has developed a second-generation B-cell maturation antigen (BCMA)-CART which is currently being tested in a clinical trial for relapsed/refractory MM.We present the first reported case, to our knowledge, of a patient with AL amyloidosis and renal involvement in the course of an MM, successfully treated with CART therapy targeting BCMA. The patient received a fractioned dose of 3×10/kg BCMA-CARTs after lymphodepletion. At 3 months from infusion, the patient had already obtained a deep hematological response with negative measurable residual disease by flow cytometry in the bone marrow. After 12 months, the patient remains in hematological stringent complete remission and has achieved an organ renal response with a decrease of 70% of proteinuria.This case suggests that concomitant AL amyloidosis in the setting of MM can benefit from CART therapy, even in patients in which predominant symptoms at the time of treating are caused by AL amyloidosis.
多发性骨髓瘤（MM）尽管近年来出现了许多新的治疗方法，但仍然无法治愈。偶尔，MM 患者会出现伴有器官功能障碍的淀粉样轻链（AL）淀粉样变性。嵌合抗原受体 T 细胞（CART）疗法已成为治疗血液恶性肿瘤的一种有前途的方法。我们机构已经开发出第二代 B 细胞成熟抗原（BCMA）-CART，目前正在临床试验中用于治疗复发/难治性 MM。我们报告了首例已知的 MM 患者在疾病过程中发生 AL 淀粉样变性和肾脏受累，该患者成功接受了针对 BCMA 的 CART 治疗。患者在淋巴耗竭后接受了 3×10/kg 的分次剂量 BCMA-CART。输注后 3 个月，患者已经在骨髓中通过流式细胞术获得了阴性可测量残留疾病的深度血液学反应。12 个月后，患者仍处于血液学严格完全缓解状态，并实现了肾脏器官反应，蛋白尿减少了 70%。该病例表明，即使在治疗时主要症状由 AL 淀粉样变性引起的情况下，MM 合并 AL 淀粉样变性也可以从 CART 治疗中获益。
