Abstract

Introduction

The assessment of AL amyloidosis response is based on serum free light chains (sFLC) levels, and serum and urine monoclonal protein investigations. Recently, difference between involved and uninvolved free light chains (dFLC), involved free light chain (iFLC) and complete response (CR) has been reported as independent predictor of survival and a refinement of the hematological response criteria has been proposed by several groups.

Methods

In the current study, all consecutive newly diagnosed symptomatic AL amyloidosis patients were evaluated. The primary objective of the study was to assess hematological and organ response after first line of treatment.

Results

A cohort of 76 cases with upfront treatment was used for this analysis. After a median of 3 months post-therapy, hematological response was seen in 88% of cases including CR in 26.3%, VGPR in 38.2% and PR in 23.7%. Median OS was longer in patients with dFLC < 10 mg/L at 3 months, iFLC <20 mg/L at 1 and 3 months, and those achieving CR. Multivariate analysis showed presence of CR as the most important independent prognostic factors for survival.

Conclusions

Our study suggests that maximal sFLC response and CR are potential endpoints to define clinical outcomes. Large collaborative studies are required to validate and optimize response criteria.

中文翻译：

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一组新诊断的 AL 淀粉样变性的治疗反应测量和生存结果

摘要

介绍

AL 淀粉样变性反应的评估基于血清游离轻链 (sFLC) 水平以及血清和尿液单克隆蛋白研究。最近，参与和未参与的游离轻链 (dFLC)、参与的游离轻链 (iFLC) 和完全反应 (CR) 之间的差异已被报道为生存的独立预测因子，并且已由多个小组提出了对血液学反应标准的改进。

方法

在目前的研究中，评估了所有连续新诊断的有症状的 AL 淀粉样变性患者。该研究的主要目的是评估一线治疗后的血液学和器官反应。

结果

该分析使用了一组 76 例接受前期治疗的病例。治疗后中位 3 个月后，88% 的病例出现血液学反应，包括 26.3% 的 CR、38.2% 的 VGPR 和 23.7% 的 PR。3 个月时 dFLC < 10 mg/L、1 个月和 3 个月时 iFLC <20 mg/L 以及达到 CR 的患者中位 OS 更长。多变量分析显示 CR 是生存最重要的独立预后因素。

结论

我们的研究表明，最大 sFLC 反应和 CR 是定义临床结果的潜在终点。需要大型合作研究来验证和优化响应标准。