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比较高剂量马法兰和干细胞移植治疗 AL 淀粉样变性后的血液学反应的测量值。

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

机构信息

Amyloidosis Center, Boston University School of Medicine, Stem Cell Transplantation Program in the Section of Hematology and Medical Oncology, Boston Medical Center, Boston, MA, USA.

Department of Biostatistics, Boston University School of Public Health, Boston, MA, USA.

出版信息

Blood Cancer J. 2020 Sep 1;10(8):88. doi: 10.1038/s41408-020-00354-7.

Abstract

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

在 AL 淀粉样变性中,血液学完全缓解(hemCR)需要免疫固定电泳(IFE)中不存在单克隆蛋白和正常的血清游离轻链比(FLCR)。最近的文献表明,受累游离轻链(iFLC)<20mg/L 或游离轻链差值(dFLC)<10mg/L 可能更能准确预测治疗后的结果。我们评估了 340 例接受高剂量美法仑和干细胞移植(SCT)治疗的患者的总生存率。在 305 例可评估 SCT 后 6 个月的患者中,90 例(30%)达到 hemCR,132 例(43%)dFLC<10mg/L,118 例(39%)iFLC<20mg/L,176 例(58%)FLCR 正常。在 215 例未达到 hemCR 的患者中,65 例(30%)dFLC<10mg/L,86 例(40%)FLCR 正常。在未达到 hemCR 但达到 dFLC<10mg/L 或 FLCR 正常的患者中,总生存率(OS)低于达到 hemCR 的患者(p=0.013 和 p=0.001)。在未达到 hemCR 但 iFLC<20mg/L 的患者中,OS 与达到 hemCR 的患者无显著差异(p=0.243)。在达到 hemCR 的患者中,如果同时达到 dFLC<10mg/L,OS 并未显著改善(p=0.852),但如果同时达到 hemCR 和 iFLC<20mg/L,OS 则得到改善(p=0.009)。多变量分析表明,IFE 中无单克隆蛋白和 iFLC<20mg/L 是生存的独立预测因素。达到 hemCR 仍然是治疗目标,尽管达到 iFLC<20mg/L 也可能预测 OS 改善。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2cd5/7462849/5ae492e4e4fc/41408_2020_354_Fig1_HTML.jpg

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