Liver Transplantation for Hereditary Transthyretin Amyloidosis. Issue 9 (September 2015)

• Record Type: Journal Article
• Authors: Ericzon, Bo-Göran; Wilczek, Henryk E.; Larsson, Marie; Wijayatunga, Priyantha; Stangou, Arie; Pena, João Rodrigues; Furtado, Emanuel; Barroso, Eduardo; Daniel, Jorge; Samuel, Didier; Adam, Rene; Karam, Vincent; Poterucha, John; Lewis, David; Ferraz-Neto, Ben-Hur; Cruz, Márcia Waddington; Munar-Ques, Miguel; Fabregat, Juan; Ikeda, Shu-ichi; Ando, Yukio; Heaton, Nigel; Otto, Gerd; Suhr, Ole
• Is Part Of: Transplantation. Volume 99:Issue 9(2015)
• Publication Date: 2015-09
• Subjects: Transplantation of organs, tissues, etc -- Periodicals; Transplantation immunology -- Periodicals; 617.95
• DOI: 10.1097/TP.0000000000000574
• Languages: English
• ISSNs: 0041-1337

Abstract

<abstract> <title> <x xml:space="preserve">Abstract</x> </title> <sec> <title>Background</title> <p>Until recently, liver transplantation (Ltx) was the only available treatment for hereditary transthyretin (TTR) amyloidosis; today, however, several pharmacotherapies are tested. Herein, we present survival data from the largest available database on transplanted hereditary TTR patients to serve as a base for comparison.</p> </sec> <sec> <title>Methods</title> <p>Liver transplantation was evaluated in a 20-year retrospective analysis of the Familial Amyloidosis Polyneuropathy World Transplant Registry.</p> </sec> <sec> <title>Results</title> <p>From April 1990 until December 2010, data were accumulated from 77 liver transplant centers. The Registry contains 1940 patients, and 1379 are alive. Eighty-eight Ltx were performed in combination with a heart and/or kidney transplantation. Overall, 20-year survival after Ltx was 55.3%. Multivariate analysis revealed modified body mass index, early onset of disease (<50 years of age), disease duration before Ltx, and TTR Val30Met versus non-TTR Val30Met mutations as independent significant survival factors. Early-onset patients had an expected mortality rate of 38% that of the late-onset group (<italic>P</italic> < 0.001). Furthermore, Val30Met patients had an expected mortality rate of 61% that of non-TTR Val30Met patients (<italic>P</italic> < 0.001). With each year of duration of disease before Ltx, expected mortality increased by 11% (<italic>P</italic> < 0.001). With each 100-unit increase in modified body mass index at Ltx, the expected mortality decreased to 89% of the expected mortality (<italic>P</italic> < 0.001). Cardiovascular death was markedly more common than that observed in patients undergoing Ltx for end-stage liver disease.</p> </sec> <sec> <title>Conclusions</title> <p>Long-term survival after Ltx, especially for early-onset TTR Val30Met patients, is excellent. The risk of delaying Ltx by testing alternative treatments, especially in early-onset TTR Val30Met patients, requires consideration.</p> </sec> </abstract>