Organ response is key to improving outcomes in light chain (AL) amyloidosis. We investigated factors associated hepatic response (HepR) in a large cohort of patients with hepatic AL amyloidosis.
Methods: Retrospective study of newly-diagnosed AL amyloidosis patients (n = 130) with liver involvement evaluated at the Mayo Clinic between 2000-2021. Patients were eligible if they had documented liver involvement and baseline alkaline phosphatase (ALP)≥1.5x upper limit of normal (ULN). HepR was defined as >50% reduction in ALP from baseline or ALP normalization. HepRs were assessed at 6, 12, 24 month after treatment initiation and the best HepR at any time point.
Results: The median baseline ALP was 2.88-fold the ULN (ALP:ULN, IQR: 2.15-4.41), and the median bilirubin was 0.7 mg/dL. HepR rates increased with time from 28% at 6 months, 36% at 12 months and 48% at 24 months. The median time to HepR was 21.5 months (95%CI = 15.4-29.5). Baseline ALP ≥ 4xULN consistently predicted HepR across all time points. Hematological response (HemR) also independently predicted HepR at 12, 24 months and best response. At best hepatic response, kappa isotype, and front-line ASCT were further independent predictors of HepR.
Conclusions: The degree of baseline ALP elevation and HemR are reliable predictors of HepR.
Keywords: Light-chain amyloidosis; liver amyloidosis; organ response prediction.