Tsamis, Konstantinos I;
Mytilinaios, Dimitrios;
Heneghan, Michael;
Gillmore, Julian D;
Gilbertson, Janet A;
Giannopoulos, Sotirios;
Sarmas, Ioannis;
(2023)
Treatment of Acquired Transthyretin Amyloidosis in Domino Liver Transplantation.
Clinical Transplantation
, 37
(1)
, Article e14822. 10.1111/ctr.14822.
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Abstract
BACKGROUND: Domino liver transplantation (DLT) has been commonly used during the last two decades to partly meet the high need for liver transplants. However, the recipients of grafts from patients with noncirrhotic inherited metabolic disorders may ultimately develop metabolic syndrome, and management is usually intricate, being complicated by the underlying initial disorder, other comorbidities, and post-transplantation conditions. CASE: We report here the management and the outcome in a patient with acquired transthyretin amyloidosis after DLT and significant comorbidities. Final treatment with a transthyretin gene silencing agent, patisiran, was well tolerated and resulted in remission of the aggravating neurological deficits in a follow-up period of two years. CONCLUSIONS: The case presented here supports the concept that patisiran can target the hepatocytes producing the mutated transthyretin in acquired transthyretin amyloidosis, as efficiently as in hereditary transthyretin amyloidosis, and can be used to treat patients with transthyretin amyloidosis after DLT. This article is protected by copyright. All rights reserved.
Type: | Article |
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Title: | Treatment of Acquired Transthyretin Amyloidosis in Domino Liver Transplantation |
Location: | Denmark |
Open access status: | An open access version is available from UCL Discovery |
DOI: | 10.1111/ctr.14822 |
Publisher version: | https://doi.org/10.1111/ctr.14822 |
Language: | English |
Additional information: | © 2022 The Authors. Clinical Transplantation published by John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License (http://creativecommons.org/licenses/by-nc/4.0/). |
Keywords: | RNA interference, amyloidosis, liver transplant, patisiran, transthyretin |
UCL classification: | UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine |
URI: | https://discovery.ucl.ac.uk/id/eprint/10156858 |
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