Mahadevia Himil, Ponvilawan Ben, Sharma Parth, Al-Obaidi Ammar, Qasim Hana, Koyi Jagadish, Anwer Faiz, Raza Shahzad
Department of Internal Medicine, University of Missouri-Kansas City, 2411 Holmes St, Kansas City, MO 64108, USA.
Department of Hematology/Oncology, University of Missouri-Kansas City, 2411 Holmes St, Kansas City, MO 64108, USA.
Crit Rev Oncol Hematol. 2023 Mar;183:103917. doi: 10.1016/j.critrevonc.2023.103917. Epub 2023 Jan 22.
Light-chain (AL) amyloidosis is a type of plasma cell neoplasm with abnormal monoclonal immunoglobulin light-chain production and their subsequent deposition in tissues causing end-organ damage. In addition to existing treatments including autologous stem cell transplantation, there is a need for other approaches for eradicating abnormal plasma cells and amyloid tissue deposits. Treatment strategies of AL amyloidosis are mostly based on medications that are effective in multiple myeloma due to similar cell of origin. Daratumumab along with proteasome inhibitors and corticosteroids has become standard of care for AL amyloidosis. Another appealing approach is disassembling amyloid deposits with hope to potentially reverse the damage done by the disease. This was met with promising results for CAEL-101 and birtamimab. Although still in early stages, novel treatment options in pipeline, including antibody-drug conjugates, bispecific T-cell engagers, and chimeric antigen receptor T cell therapy may diversify the treatment armamentarium of AL amyloidosis in the future.
轻链（AL）淀粉样变性是一种浆细胞肿瘤，其产生异常单克隆免疫球蛋白轻链，并随后沉积在组织中导致终末器官损伤。除了包括自体干细胞移植在内的现有治疗方法外，还需要其他方法来根除异常浆细胞和淀粉样组织沉积物。由于起源细胞相似，AL淀粉样变性的治疗策略大多基于对多发性骨髓瘤有效的药物。达雷妥尤单抗联合蛋白酶体抑制剂和皮质类固醇已成为AL淀粉样变性的标准治疗方案。另一种有吸引力的方法是拆解淀粉样沉积物，以期有可能逆转该疾病造成的损害。CAEL-101和比他米单抗在这方面取得了令人鼓舞的结果。尽管仍处于早期阶段，但正在研发的新型治疗选择，包括抗体药物偶联物、双特异性T细胞衔接器和嵌合抗原受体T细胞疗法，未来可能会使AL淀粉样变性的治疗手段更加多样化。
