文章：

比较AL型淀粉样变性患者接受大剂量马法兰和干细胞移植后的血液学反应指标

Comparing measures of hematologic response after high-dose melphalan and stem cell transplantation in AL amyloidosis

原文发布日期：2020-09-01

DOI: 10.1038/s41408-020-00354-7

类型: Article

开放获取: 是

英文摘要：

Hematologic complete response (hemCR) in AL amyloidosis requires absence of monoclonal protein by immunofixation electrophoreses (IFE) and normal serum free light chain ratio (FLCR). Recent literature suggests that an involved free light chain (iFLC) <20 mg/L or difference in free light chains (dFLC) <10 mg/L may more accurately predict outcomes after treatment. We evaluated overall survival in 340 patients treated with high-dose melphalan and stem cell transplantation (SCT). Of 305 patients evaluable 6 months after SCT, 90 (30%) achieved hemCR, 132 (43%) dFLC <10 mg/L, 118 (39%) iFLC <20 mg/L, and 176 (58%) normal FLCR. Of 215 patients without hemCR, 65 (30%) had dFLC <10 mg/L and 86 (40%) had normal FLCR. Overall survival (OS) in those achieving dFLC <10 mg/L or normal FLCR without hemCR was inferior to those achieving hemCR (p = 0.013 and p = 0.001). OS was not significantly different in patients achieving iFLC <20 mg/L without hemCR compared with hemCR (p = 0.243). Of those with hemCR, OS was not significantly improved if dFLC <10 mg/L was also achieved (p = 0.852), but OS was improved for those with hemCR who also attained iFLC <20 mg/L (p = 0.009). Multivariate analysis demonstrated absence of monoclonal protein in IFE and iFLC <20 mg/L as independent predictors of survival. Attainment of hemCR remains a treatment goal, although achieving iFLC <20 mg/L may also predict improved OS.
 

摘要翻译： 

AL淀粉样变性的血液学完全缓解（hemCR）需满足免疫固定电泳（IFE）检测不到单克隆蛋白且血清游离轻链比率（FLCR）正常。近期文献指出，受累游离轻链（iFLC）<20 mg/L或游离轻链差值（dFLC）<10 mg/L可能更准确预测治疗后结局。我们评估了340例接受大剂量美法仑和干细胞移植（SCT）患者的总生存期。在SCT后6个月可评估的305例患者中，90例（30%）达到hemCR，132例（43%）实现dFLC<10 mg/L，118例（39%）实现iFLC<20 mg/L，176例（58%）FLCR恢复正常。在215例未达到hemCR的患者中，65例（30%）实现dFLC<10 mg/L，86例（40%）FLCR正常。达到dFLC<10 mg/L或FLCR正常但未实现hemCR者的总生存期（OS）劣于达到hemCR者（p=0.013和p=0.001）。实现iFLC<20 mg/L但未达到hemCR者与达到hemCR者的OS无显著差异（p=0.243）。在达到hemCR的患者中，额外实现dFLC<10 mg/L并未显著改善OS（p=0.852），但额外实现iFLC<20 mg/L可改善OS（p=0.009）。多变量分析显示IFE中单克隆蛋白消失和iFLC<20 mg/L是生存的独立预测因子。实现hemCR仍是治疗目标，而达到iFLC<20 mg/L也可能预示OS改善。

原文链接：

Comparing measures of hematologic response after high-dose melphalan and stem cell transplantation in AL amyloidosis