淀粉样变治疗学研究的最新进展

Recent progress of research on therapeutics of amyloidosis

既往10-15年中系统性淀粉样变新治疗的发展取得了实质性进展。这些进展改善了患者的转归,但亦带来新的临床问题。例如,由于淀粉样轻链(或)(AL)淀粉样变对自体造血干细胞移植的作用不太肯定,为此正在开发广泛途径的治疗研究和提供最佳支持治疗。现认为淀粉样前体和成熟原纤维促使淀粉样变相关的器官病变,显示完全清除AL淀粉样变患者促淀粉样(amyloidogenic)轻链生成的重要性,同时治疗前后需作淀粉样前体蛋白和器官病变标记的监测。

During the past 10 to 15 years, there has been substantial progress in developing new treatments for the systemic amyloidosis. These advances have improved patient outcomes but have also raised new questions with direct clinical implications. For example, development of less intensive treatments for AL amyloidosis has made less certain the role of autologous stem cell transplantation, and new quantitative assays should now allow determination of the im- portance of fully eliminating amyloidogenic light chain production in AL disease. Additionally, observations from a clinical trial in AA amyloidosis have generated hypotheses about the relative contributions of amyloid precursors and mature fibrils to amyloidosis associated kidney disease.