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

Area of Science:

• Hematology
• Oncology
• Gastroenterology

Background:

• Liver involvement in light chain amyloidosis (AL) affects 10-20% of patients and is linked to a poor prognosis.
• Assessing liver function and response is crucial for managing AL amyloidosis patients.

Purpose of the Study:

• To evaluate the prognostic significance of hepatic response criteria in AL amyloidosis patients with liver involvement.
• To determine if specific markers of liver recovery correlate with improved overall survival.

Main Methods:

• A cohort of 116 AL amyloidosis patients diagnosed between 2010-2015 with liver involvement (serum alkaline phosphatase [AP] >1.5 upper reference limit [URL]) and achieving hematological response were analyzed.
• Hepatic response was defined as >50% reduction or normalization of AP from baseline, assessed at multiple time points (6, 12, 24 months, and best response).
• Overall survival (OS) was calculated from therapy initiation.

Main Results:

• A median baseline serum AP of 2.6 URL was observed. Hematological response (very good partial response [VGPR] or better) was achieved in 69% of patients.
• Hepatic response, defined by AP reduction, was observed in a significant proportion of patients, with a median reduction of 56% at best response.
• Achievement of hepatic response, especially by 12 months and at best response, was independently associated with improved OS.

Conclusions:

• Hepatic response, measured by changes in alkaline phosphatase, is a significant independent prognostic factor in AL amyloidosis.
• Early and sustained reduction in AP levels indicates a favorable prognosis and improved survival in these patients.
• Predictors of hepatic response include higher baseline AP, lower bilirubin, and achievement of hematological, cardiac, and renal responses.