Systemic amyloidoses are characterized by the unrelenting deposition of autologous proteins as highly ordered fibrils in target organs. The ensuing, potentially fatal organ dysfunction is the result of the combined damage caused by the proteotoxic effect of prefibrillar species and by the cytotoxicity and the structural alterations produced by the amyloid fibrils. Current therapy is focused on eliminating the amyloid protein, thus extinguishing the amyloid cascade at its origin. While this approach may end the cell damage caused by prefibrillar aggregates and prevent further amyloid accumulation, the noxious effects of the amyloid fibrils persist and may hamper the recovery of organ function, which is the ultimate goal of therapy as it is necessary to improve the quality of life and extend survival. Preclinical studies indicate that the clearance of amyloid deposits can be accelerated by specific antibodies targeting amyloid fibrils that activate complement-mediated macrophages and giant cell phagocytosis, possibly promoting the recovery of organ function. Measuring the therapeutic effect of anti-amyloid agents is still a matter of research. In recent years, several monoclonal antibodies targeting amyloid deposits have been tested in clinical trials with mixed outcomes. Recent encouraging results from phase I/II trials, new anti-amyloid agents, and new antibody engineering offer hope that effective amyloid removal will be accomplished in the near future, accelerating organ recovery and improving quality of life and survival.

系统性淀粉样变性的特征是自体蛋白作为高度有序的原纤维在靶器官中不断沉积。随之而来的、可能致命的器官功能障碍是前原纤维种类的蛋白毒性作用和淀粉样蛋白原纤维产生的细胞毒性和结构改变引起的联合损伤的结果。目前的治疗重点是消除淀粉样蛋白，从而在其起源处消除淀粉样蛋白级联反应。虽然这种方法可能会结束由原纤维前聚集体引起的细胞损伤并防止淀粉样蛋白进一步积累，但淀粉样蛋白原纤维的有害作用持续存在并可能阻碍器官功能的恢复，这是治疗的最终目标，因为有必要提高质量的生命和延长生存期。临床前研究表明，针对淀粉样蛋白原纤维的特异性抗体可以加速淀粉样蛋白沉积物的清除，这些抗体激活补体介导的巨噬细胞和巨细胞吞噬作用，可能促进器官功能的恢复。测量抗淀粉样蛋白药物的治疗效果仍然是一个研究问题。近年来，几种针对淀粉样蛋白沉积物的单克隆抗体已经在临床试验中进行了测试，结果喜忧参半。最近来自 I/II 期试验、新的抗淀粉样蛋白药物和新的抗体工程的令人鼓舞的结果为在不久的将来实现有效去除淀粉样蛋白、加速器官恢复并改善生活质量和生存提供了希望。