文章：

通过下一代流式细胞术检测到的微小残留病阴性，与AL型淀粉样变性患者器官反应的改善相关

Minimal residual disease negativity by next-generation flow cytometry is associated with improved organ response in AL amyloidosis

原文发布日期：2021-02-16

DOI: 10.1038/s41408-021-00428-0

类型: Article

开放获取: 是

英文摘要：

Light chain (AL) amyloidosis is caused by a small B-cell clone producing light chains that form amyloid deposits and cause organ dysfunction. Chemotherapy aims at suppressing the production of the toxic light chain (LC) and restore organ function. However, even complete hematologic response (CR), defined as negative serum and urine immunofixation and normalized free LC ratio, does not always translate into organ response. Next-generation flow (NGF) cytometry is used to detect minimal residual disease (MRD) in multiple myeloma. We evaluated MRD by NGF in 92 AL amyloidosis patients in CR. Fifty-four percent had persistent MRD (median 0.03% abnormal plasma cells). There were no differences in baseline clinical variables in patients with or without detectable MRD. Undetectable MRD was associated with higher rates of renal (90% vs 62%, p = 0.006) and cardiac response (95% vs 75%, p = 0.023). Hematologic progression was more frequent in MRD positive (0 vs 25% at 1 year, p = 0.001). Altogether, NGF can detect MRD in approximately half the AL amyloidosis patients in CR, and persistent MRD can explain persistent organ dysfunction. Thus, this study supports testing MRD in CR patients, especially if not accompanied by organ response. In case MRD persists, further treatment could be considered, carefully balancing residual organ damage, patient frailty, and possible toxicity.
 

摘要翻译： 

轻链淀粉样变性由小B细胞克隆产生轻链引起，这些轻链形成淀粉样沉积并导致器官功能障碍。化疗旨在抑制毒性轻链的产生并恢复器官功能。然而，即使是完全血液学缓解（定义为血清和尿免疫固定电泳阴性且游离轻链比值恢复正常），也并不总能转化为器官缓解。二代流式细胞术常用于多发性骨髓瘤的微小残留病检测。本研究通过二代流式细胞术对92例达到完全缓解的轻链淀粉样变性患者进行了微小残留病评估。54%的患者存在持续微小残留病（异常浆细胞中位数为0.03%）。无论是否检测到微小残留病，患者的基线临床特征均无差异。未检测到微小残留病与较高的肾脏缓解率（90% vs 62%，p=0.006）和心脏缓解率（95% vs 75%，p=0.023）相关。微小残留病阳性患者的血液学进展更为常见（1年时0% vs 25%，p=0.001）。总体而言，二代流式细胞术可在约半数达到完全缓解的轻链淀粉样变性患者中检测到微小残留病，持续存在的微小残留病可以解释持续的器官功能障碍。因此，本研究支持对完全缓解患者进行微小残留病检测，特别是未伴随器官缓解时。若微小残留病持续存在，可考虑进一步治疗，同时需谨慎权衡残留器官损伤、患者虚弱程度及潜在治疗毒性。

原文链接：

Minimal residual disease negativity by next-generation flow cytometry is associated with improved organ response in AL amyloidosis