Division of Hematology and Oncology, Department of Medicine, O'Neal Comprehensive Cancer Center, University of Alabama at Birmingham, Birmingham, AL.
Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.
Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):682-688. doi: 10.1182/hematology.2021000305.
Systemic light chain (AL) amyloidosis is a protein misfolding disorder characterized by the deposition of abnormal immunoglobulin light chains in fibrillary aggregates, resulting in end-organ damage. Several unique challenges face treating physicians, including delayed diagnosis, advanced vital organ involvement, and morbidity with treatment. Aggressive supportive care and risk-adapted application of plasma cell-directed therapies are the cornerstones of management. The therapeutic revolution in multiple myeloma will likely further expand the arsenal against plasma cells. Careful investigation of these agents will be critical to establish their role in this fragile population. The promise of fibril-directed therapies to restore organ function remains despite early disappointments. In this review, we discuss new therapies to tackle AL amyloidosis using a case-based approach.
系统性轻链(AL)淀粉样变性是一种蛋白质错误折叠疾病,其特征是异常免疫球蛋白轻链在纤维状聚集物中沉积,导致终末器官损伤。治疗医生面临着几个独特的挑战,包括诊断延迟、重要生命器官受累和治疗带来的发病率。积极的支持性护理和针对浆细胞的治疗方法的风险适应是管理的基石。多发性骨髓瘤的治疗革命可能会进一步扩大针对浆细胞的武器库。仔细研究这些药物对于确定它们在这个脆弱人群中的作用至关重要。尽管早期令人失望,但纤维定向治疗恢复器官功能的前景依然存在。在这篇综述中,我们采用病例为基础的方法讨论了使用新的疗法来治疗 AL 淀粉样变性。