Wechalekar Ashutosh D, Hawkins Philip N, Gillmore Julian D
National Amyloidosis Centre, Centre for Amyloidosis & Acute Phase Proteins, Department of Medicine, Royal Free and University College Medical School, London, UK.
Br J Haematol. 2008 Feb;140(4):365-77. doi: 10.1111/j.1365-2141.2007.06936.x. Epub 2007 Dec 19.
Light chain (AL) amyloidosis is the most frequently diagnosed form of systemic amyloid in the western world. The historically poor prognosis of AL amyloidosis appears to be improving with currently reported median survival of c. 40 months compared to 13 months in the early 1990s when low-dose oral melphalan was the mainstay of treatment. Autologous stem cell transplantation (ASCT) achieves the highest rates of complete clonal response but is confounded by substantial treatment-related mortality in AL amyloidosis unless it is restricted to highly selected patients. Newer chemotherapy regimens appear to have a balance of better safety and respectable efficacy with overall outcomes nearly similar to ASCT, but which may be used more widely. There are few data comparing durability, depth of clonal response, rate of organ response and overall survival following ASCT or chemotherapy, but a recent small, randomized trial did not suggest superiority of ASCT to oral melphalan and dexamethasone. There is a compelling need for further and larger randomized trials in this context. At the same time, various new specific anti-amyloid drugs have shown (in early phase studies or animal models) some very promising results. This review attempts to highlight the challenges, controversies and progress in AL amyloidosis.
轻链（AL）淀粉样变性是西方世界最常被诊断出的系统性淀粉样变性形式。与20世纪90年代初低剂量口服美法仑作为主要治疗手段时的13个月中位生存期相比，目前报道的AL淀粉样变性中位生存期约为40个月，其既往较差的预后似乎正在改善。自体干细胞移植（ASCT）实现完全克隆反应的比例最高，但在AL淀粉样变性中存在大量与治疗相关的死亡率，除非仅限于高度选择的患者。较新的化疗方案似乎在安全性和疗效方面取得了较好的平衡，总体结果与ASCT几乎相似，但可能应用更广泛。关于ASCT或化疗后的缓解持续时间、克隆反应深度、器官反应率和总生存期的比较数据很少，但最近一项小型随机试验并未表明ASCT优于口服美法仑和地塞米松。在这种情况下，迫切需要进一步开展更大规模的随机试验。与此同时，各种新型特异性抗淀粉样药物（在早期研究或动物模型中）已显示出一些非常有前景的结果。本综述试图突出AL淀粉样变性中的挑战、争议和进展。
