Introduction The liver is frequently involved in systemic AL amyloidosis in association with B-cell dyscrasias, and some of the hereditary forms mainly apolipoprotein ApoAI amyloidosis. Extensive hepatic amyloid infiltrate can lead to liver failure. We report here the features, disease course, management and outcome of 28 patients with end-stage hepatic amyloidosis.
Methods Twenty-eight patients with amyloid related liver failure, median age 53 (28–70) years. All patients had hepatomegaly, 14 (50%) had jaundice, ten had ascites, four patients had encephalopathy, and three were in renal failure. The amyloid type was determined by immunohistochemistry, serum/urine electrophoresis and DNA analysis. All patients had comprehensive systemic, cardiac and neurological evaluation.
Results 22 patients had AL amyloidosis due to B-cell dyscrasias; none were eligible for chemotherapy. Six cases had hereditary amyloid due to variant fibrinogen A α chain, AFib (1) and apolipoprotein ApoAI (5). Extrahepatic amyloid deposits were present in all cases. 11/22 patients with AL liver failure were listed for liver transplantation (LT). All six patients with ApoAI or AFib amyloidosis who had liver and renal failure were considered for combined liver/kidney transplant (LKT).
Nine AL patients received LT as a rescue procedure and two died with complications of hepatic rupture or multi-organ failure on the waiting list. The remaining 11 AL cases all died within 2–16 weeks with progressive cholestasis and subacute liver failure. Actuarial 1-year survival post LT in AL was 75%, 2-year 50%. Early mortality post LT was due to septic episodes, nephrotic syndrome and renal failure, and medium-term due to progressive amyloidosis and multiple organ falure. Only two AL patients exhibited over 5 year survival. Both had predominantly liver and splenic amyloid with no cardiac or renal involvement and were treated with stem cell transplant (SCT) after LT. Three ApoAI cases had successful LKT but two died with complications of liver failure on the wait list. The AFib patient has normal graft function at 13 years post LKT (cumulative LKT survival for ApoAI and AFib 100%).
Conclusion LT is life saving for liver failure due to AL amyloidosis, but the outcome is ultimately determined by severity of systemic disease at the time of LT and eligibility for efficient treatment for AL after LT. Sequential SCT was associated with long-term remission in two LT recipients. The excellent outcome of LT in both ApoAI and AFib hereditary systemic amyloidosis strongly supports its use for these indications.
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