Hepatic response criteria in light chain amyloidosis: a multicenter validation study

Liver involvement in light chain amyloidosis (AL) is seen in 10-20% of patients and is associated with poor prognosis. The goal of this study was to assess the prognostic impact of the hepatic response criteria. AL patients diagnosed between 2010 and 2015 with liver involvement [serum alkaline phosphatase (AP) >1.5 upper reference limit (URL)] who achieved hematological response were included. Hepatic response was defined as >50% reduction (or normalization) of AP from baseline. Hepatic response was assessed at 6, 12, and 24-months after therapy initiation and at best response. Overall survival (OS) was assessed from time of therapy initiation. Hepatic response was evaluated in 116 patients. The median baseline serum AP was X2.6 URL. Hematological very good partial response (VGPR) or better was achieved in 69% of patients. AP decreased with time, with a median reduction of 22%, 34%, and 53% at 6-, 12-, and 24-months, respectively, and a median AP reduction of 56% at the time of best response. The median time to hepatic response was 13.3 months and was longer for patients undergoing autologous stem cell transplantation. Achievement of hepatic response, particularly as early as 12 months, and at best response, was associated with improved survival, independent of other prognostic factors. Predictors of hepatic response include higher baseline AP level, lower total bilirubin, hematological ≥VGPR, and cardiac and renal responses, when applicable. Hepatic response measured by the change in alkaline phosphatase is a prognostic factor in patients with AL amyloidosis.