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Treatment of acquired transthyretin amyloidosis in domino liver transplantation.
Tsamis, Konstantinos I; Mytilinaios, Dimitrios; Heneghan, Michael; Gillmore, Julian D; Gilbertson, Janet A; Giannopoulos, Sotirios; Sarmas, Ioannis; Konitsiotis, Spyridon.
Afiliación
  • Tsamis KI; Faculty of Medicine, Department of Physiology, School of Health Sciences, University of Ioannina, Ioannina, Greece.
  • Mytilinaios D; Department of Neurology, University Hospital of Ioannina, University of Ioannina, Ioannina, Greece.
  • Heneghan M; Kenhub GmbH, Leipzig, Germany.
  • Gillmore JD; Hepatology Department, Institute of Liver Studies, King's College Hospital, London, UK.
  • Gilbertson JA; National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Campus, London, UK.
  • Giannopoulos S; National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Campus, London, UK.
  • Sarmas I; Department of Neurology, University Hospital of Ioannina, University of Ioannina, Ioannina, Greece.
  • Konitsiotis S; Second Department of Neurology, National & Kapodistrian University of Athens, School of Medicine, "Attikon" University Hospital, Athens, Greece.
Clin Transplant ; 37(1): e14822, 2023 Jan.
Article en En | MEDLINE | ID: mdl-36128766
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 2 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 (hATTR), and can be used to treat patients with transthyretin amyloidosis after DLT.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Trasplante de Hígado / Neuropatías Amiloides Familiares Límite: Humans Idioma: En Revista: Clin Transplant Asunto de la revista: TRANSPLANTE Año: 2023 Tipo del documento: Article País de afiliación: Grecia Pais de publicación: Dinamarca
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Trasplante de Hígado / Neuropatías Amiloides Familiares Límite: Humans Idioma: En Revista: Clin Transplant Asunto de la revista: TRANSPLANTE Año: 2023 Tipo del documento: Article País de afiliación: Grecia Pais de publicación: Dinamarca