PDF(Pubmed)
Abstract：
Immunoglobulin light chain (AL) amyloidosis is an incurable plasma cell disorder characterized by deposition of fibrils of misfolded immunoglobulin free light chains (FLC) in target organs, leading to failure. Cardiac involvement is common in AL amyloidosis and represents the single most adverse prognostic feature. A high index of clinical suspicion with rapid tissue diagnosis and commencement of combinatorial, highly effective cytoreductive therapy is crucial to arrest the process of amyloid deposition and preserve organ function. The clinical use of molecularly targeted drugs, such as proteasome inhibitors and immunomodulatory agents, monoclonal antibodies such as daratumumab, and risk-adjusted autologous stem cell transplant in eligible patients, has radically changed the natural history of AL amyloidosis. Here, we review the state-of-the-art treatment landscape in AL amyloidosis with an eye toward future therapeutic venues to impact the outcome of this devastating illness.
摘要：
免疫球蛋白轻链（AL）淀粉样变性是一种无法治愈的浆细胞疾病，其特征是错误折叠的免疫球蛋白游离轻链（FLC）的原纤维在靶器官中沉积，导致失败。心脏受累在AL淀粉样变性中很常见，并且是最不利的预后特征。具有快速组织诊断和组合开始的高临床怀疑指数，高效的细胞减灭剂治疗对于阻止淀粉样蛋白沉积过程和保持器官功能至关重要。分子靶向药物的临床使用,如蛋白酶体抑制剂和免疫调节剂，单克隆抗体，如daratumumab，对符合条件的患者进行风险调整后的自体干细胞移植，从根本上改变了AL淀粉样变性的自然史。这里,我们回顾了AL淀粉样变性最先进的治疗方案,着眼于未来的治疗场所,以影响这一毁灭性疾病的结局.
