Division of Internal Medicine, Mayo Clinic, Rochester, Minnesota 55905, USA.
Cancer. 2012 Dec 15;118(24):6105-9. doi: 10.1002/cncr.27660. Epub 2012 Jun 15.
The current study was conducted to determine characteristics distinguishing the 10-year survivor group in patients with systemic immunoglobulin light chain (AL) amyloidosis who underwent autologous stem cell transplantation (SCT).
The study group included all 74 patients with AL amyloidosis who underwent high-dose melphalan treatment supported by autologous SCT since the beginning of the Mayo Clinic's SCT program until prior to August 2001.
A total of 32 patients (43%) patients survived for > 10 years. Statistically significant baseline differences in the 10-year survivor group included: 1) the number of organs involved; 2) septal thickness; 3) total cholesterol; and 4) urine total protein. The number of organs involved was the only predictor found on multivariable analysis. Depth of the response to therapy, as measured by the lowest posttransplantation serum free light chain level, was found to be the most significant indicator of durability of response.
Autologous SCT can offer durable benefit for patients with AL amyloidosis. The number of organs involved offers the greatest pretreatment prognostic value, whereas the lowest posttransplantation serum free light chain level offers the best posttreatment prognostic value.
本研究旨在确定接受自体干细胞移植（SCT）的系统性免疫球蛋白轻链（AL）淀粉样变性患者中，10 年幸存者的特征。
研究组包括自梅奥诊所 SCT 项目开始至 2001 年 8 月前接受大剂量美法仑治疗并支持自体 SCT 的所有 74 例 AL 淀粉样变性患者。
共有 32 例（43%）患者存活时间>10 年。10 年幸存者组在基线水平上存在统计学显著差异，包括：1）受累器官数量；2）间隔厚度；3）总胆固醇；和 4）尿总蛋白。受累器官数量是多变量分析中唯一发现的预测因素。治疗反应的深度，如移植后血清游离轻链水平的最低值所示，是反应持续时间的最重要指标。
自体 SCT 可为 AL 淀粉样变性患者提供持久的益处。受累器官数量提供了最大的预处理预后价值，而移植后血清游离轻链水平的最低值提供了最佳的治疗后预后价值。
