文章：

AL淀粉样变性中深度血液学反应的指标：完全缓解仍是治疗目标

Indicators of profound hematologic response in AL amyloidosis: complete response remains the goal of therapy

原文发布日期：2020-09-01

DOI: 10.1038/s41408-020-00355-6

类型: Article

开放获取: 是

英文摘要：

In AL amyloidosis complete response (aCR) is defined as negative serum and urine immunofixation with normalized free light chain ratio (FLCR). However, achievement of low levels of involved FLC (iFLC) or difference between iFLC and uninvolved FLC (dFLC) are also relevant endpoints for treatment. We divided 434 consecutive patients with AL amyloidosis into five groups according to response 6 months after treatment initiation: aCR, iFLC <20 mg/L, normalized-iFLC, dFLC <10 mg/L, and normalized FLC ratio. Overall survival (OS) was similar (median not reached) in patients in aCR and in those who reached iFLC <20 mg/L, while it was inferior in all other groups (medians ranging from 79 to 91 months). Time to next therapy or death (TNTD) was longer in subjects attaining aCR (median 69 months) than in subjects reaching any FLC endpoint (medians ranging from 18 to 39 months). The ability of discriminating patients who survived more than 2 years among all responders was greater for current definition of aCR compared to combination of negative serum and urine immunofixation with any low-FLC endpoint. Complete response predicts best outcomes in AL amyloidosis and should be the goal of therapy if tolerability allows.
 

摘要翻译： 

在AL型淀粉样变性病中，完全缓解（aCR）定义为血清和尿免疫固定电泳阴性且游离轻链比率（FLCR）恢复正常。然而，达到较低水平的受累游离轻链（iFLC）或受累与非受累游离轻链差值（dFLC）也是治疗相关的终点。我们根据治疗开始后6个月的治疗反应，将434例连续入组的AL型淀粉样变性病患者分为五组：完全缓解组、iFLC＜20 mg/L组、iFLC正常化组、dFLC＜10 mg/L组以及游离轻链比率正常化组。完全缓解组与iFLC＜20 mg/L组患者的总生存期相近（中位数均未达到），而其他所有组别的总生存期均较差（中位生存期范围在79至91个月之间）。达到完全缓解的患者至下次治疗或死亡的时间（中位69个月）比达到任何游离轻链终点的患者更长（中位时间范围在18至39个月）。在区分所有应答者中生存超过2年的患者方面，当前完全缓解的定义相较于血清和尿免疫固定电泳阴性结合任何低游离轻链终点的定义具有更强的鉴别能力。完全缓解预示着AL型淀粉样变性病的最佳预后，若耐受性允许，应作为治疗目标。

原文链接：

Indicators of profound hematologic response in AL amyloidosis: complete response remains the goal of therapy