Abstract
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.
Highlights
Light chain (AL) amyloidosis is caused by a small plasma cell clone producing light chains that form amyloid deposit while causing organ dysfunction and damage[1]
Four-hundred thirty-four patients were included and distributed in five groups (Table 1): (a) patients in Amyloid complete response (aCR), and subjects who did not qualify for aCR, namely (b) patients whose post-treatment involved free light chain (FLC) (iFLC) was
The present study is based on the largest patient population (1378 consecutive subjects) systematically searched for individuals with profound hematologic response (N = 434 patients)
Summary
Light chain (AL) amyloidosis is caused by a small plasma cell clone producing light chains that form amyloid deposit while causing organ dysfunction and damage[1]. Chemotherapy targeting the plasma cell clone aims at prolonging survival by obtaining deep reductions of the amyloid light chain. Amyloid complete response (aCR) was defined as negative serum and urine immunofixation and normalized free light chain (FLC) ratio[2]. This predicted the longest OS in the testing and in the validation cohorts[2]. Other response categories were very good partial response (VGPR), Several groups reported that in patients with low-FLC burden (a baseline dFLC
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