期刊
BLOOD
卷 128, 期 2, 页码 159-168出版社
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2016-01-629790
关键词
-
类别
资金
- Associazione Italiana per la Ricerca sul Cancro-Special Program Molecular Clinical Oncology [9965]
- Cassa di Risparmio delle Provincie Lombarde (CARIPLO) Structure-function relation of amyloid: understanding the molecular bases of protein misfolding diseases to design new treatments [2013-0964]
- CARIPLO Molecular mechanisms of Ig toxicity in age-related plasma cell dyscrasias [2015-0591]
Light chain (AL) amyloidosis is caused by a usually small plasma cell clone producing a misfolded light chain that deposits in tissues. Survival is mostly determined by the severity of heart involvement. Recent studies are clarifying themechanisms of cardiac damage, pointing to a toxic effect of amyloidogenic light chains and offering new potential therapeutic targets. The diagnosis requires adequate technology, available at referral centers, for amyloid typing. Late diagnosis results in approximately 30% of patients presenting with advanced, irreversible organ involvement and dying in a few months despite modern treatments. The availability of accurate biomarkers of clonal and organ disease is reshaping the approach to patients with AL amyloidosis. Screening of early organ damage based on biomarkers can help identify patients with monoclonal gammopathy of undetermined significance who are developing AL amyloidosis before they become symptomatic. Staging systems and response assessment based on biomarkers facilitate the design and conduction of clinical trials, guide the therapeutic strategy, and allow the timely identification of refractory patients to be switched to rescue therapy. Treatment should be risk-adapted. Recent studies are linking specific characteristics of the plasma cell clone to response to different types of treatment, moving toward patient-tailored therapy. In addition, novel anti-amyloid treatments are being developed that might be combined with anti-plasma cell chemotherapy.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据