Penn Amyloidosis Program, Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA; and.
The John C. Davis Myeloma and Amyloid Program, Tufts Medical Center, Boston, MA.
Blood. 2016 May 12;127(19):2275-80. doi: 10.1182/blood-2015-11-681650. Epub 2016 Feb 23.
Systemic immunoglobulin light chain (LC) amyloidosis (AL) is a potentially fatal disease caused by immunoglobulin LC produced by clonal plasma cells. These LC form both toxic oligomers and amyloid deposits disrupting vital organ function. Despite reduction of LC by chemotherapy, the restoration of organ function is highly variable and often incomplete. Organ damage remains the major source of mortality and morbidity in AL. This review focuses on the challenges posed by emerging therapies that may limit the toxicity of LC and improve organ function by accelerating the resorption of amyloid deposits.
系统性免疫球蛋白轻链 (LC) 淀粉样变性 (AL) 是一种由克隆性浆细胞产生的免疫球蛋白 LC 引起的潜在致命疾病。这些 LC 形成有毒的低聚物和淀粉样沉积物，破坏重要器官的功能。尽管通过化疗减少了 LC，但器官功能的恢复差异很大，且常常不完整。器官损伤仍然是 AL 患者死亡和发病的主要原因。这篇综述重点介绍了新兴治疗方法带来的挑战，这些方法可能通过加速淀粉样沉积物的吸收来限制 LC 的毒性并改善器官功能。
