Year: 2026
Type: article
Study design: Observational Study
Abstract: 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.
Source: Haematologica
Authors: Eli Muchtar, Giovanni Palladini, Stefan Schonland, Susan Geyer, Katharine E. Dooley, Angela Dispenzieri, Brendan Wisniowski, Giampaolo Merlini, Paolo Milani, Ute Hegenbart, Tobias Dittrich, Efstathios Kastritis, Meletios A. Dimopoulos, Vaishali Sanchorawala, Raphael Szalat, Michaela Liedtke, Mani Gupta, Heather Landau, Suzanne Lentzsch, Michael S. Hughes, Maria Teresa Cibeira, Joan Blade, Shaji K. Kumar, Ashutosh Wechalekar, Morie A. Gertz
Institutions: Mayo Clinic, University of Pavia, Policlinico San Matteo Fondazione, Heidelberg University, The Royal Free Hospital, University College London, National and Kapodistrian University of Athens, Boston University, Boston Medical Center, Stanford Medicine, Stanford University, Memorial Sloan Kettering Cancer Center, Columbia University Irving Medical Center, Hospital Clínic de Barcelona, Consorci Institut D'Investigacions Biomediques August Pi I Sunyer
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $2,157
OpenAlex ID: W7124306659