Hereditary fibrinogen A alpha-chain amyloidosis: clinical phenotype and role of liver transplantation.

摘要

Having had the opportunity to follow up 71 patients with hereditary fibrinogen A alpha-chain amyloidosis (AFib) at the United Kingdom National Amyloidosis Centre, our perspective on this disease differs substantially in several important respects from that of Dr Stangou and colleagues, despite our series including 20 of the 21 patients reported in their recent Blood paper. Our experience is that AFib is, for all intents and purposes, a renal disease that is neither associated with cardiac amyloidosis nor autonomic neuropathy in any clinically meaningful way. Furthermore, we do not believe that the authors' preliminary findings justify their concluding statement encouraging preemptive solitary orthotopic liver transplantation (OLT).