Sher Taimur, Gertz Morie A
Department of Medicine, Mayo Clinic, Jacksonville, Florida.
Department of Medicine, Mayo Clinic, Rochester, Minnesota.
Curr Probl Cancer. 2017 Mar-Apr;41(2):129-137. doi: 10.1016/j.currproblcancer.2017.03.001. Epub 2017 Mar 16.
Systemic chemotherapy aimed at eradicating transformed plasma cells is the mainstay of treatment for immunoglobulin light chain amyloidosis (AL). Autologous stem cell transplantation (SCT) is a highly effective treatment for AL and can lead to long term survival in excess of 10 years in patients who achieve complete remission. Since AL is a unique disease characterized by multiple organ dysfunction, SCT poses unique challenges in this disease. Morbidity and mortality of SCT has remarkably improved over time primarily due to careful selection of patients and evolution of predictive and prognostic models based on serum immunoglobulin light chains and cardiac biomarkers. In this review we focus on the historical evolution of SCT as a treatment for AL and unique challenges it poses in the management of this rare disease and provide guidelines for managing these challenges.
旨在根除转化浆细胞的全身化疗是免疫球蛋白轻链淀粉样变性(AL)治疗的主要手段。自体干细胞移植(SCT)是治疗AL的一种高效疗法,对于实现完全缓解的患者,可使其长期存活超过10年。由于AL是一种以多器官功能障碍为特征的独特疾病,SCT在该疾病中带来了独特的挑战。随着时间推移,SCT的发病率和死亡率已显著改善,这主要归功于对患者的精心挑选以及基于血清免疫球蛋白轻链和心脏生物标志物的预测和预后模型的发展。在本综述中,我们聚焦于SCT作为AL治疗方法的历史演变及其在这种罕见疾病管理中所带来的独特挑战,并提供应对这些挑战的指导方针。