[de novo hATTR amyloidosis after domino transplantation of a donor's liver: a case report for the use of Patisiran]. / de novo hATTR Amyloidose nach Domino-Lebertransplantation: Ein Fallbeispiel für den Einsatz von Patisiran.
Z Gastroenterol
; 60(11): 1659-1664, 2022 Nov.
Article
em De
| MEDLINE
| ID: mdl-35533685
ABSTRACT
Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare, rapidly progressing, and potentially life-threatening disease caused by one of more than 120 mutations in the transthyretin (TTR) gene. As a result of the cumulative amyloid deposits, especially in the peripheral nerves and the heart, the majority of patients develop progressive, peripheral sensorimotor polyneuropathy and biventricular cardiomyopathy over time.Since TTR - and its amyloidogenic variants too - is predominantly synthesized in the liver, early, orthotopic liver transplantation (LTx) is a treatment option that can be used to potentially stop the progression of hATTR amyloidosis.The actual case shows a patient with hepatocellular carcinoma who received the organ of a patient with hATTR as part of a domino liver transplantation. After approximately 10 years, the patient started to develop the characteristic symptoms of the metabolic disorder. Because of a further progression of the amyloidosis, therapy with the RNA interference therapeutic patisiran was initiated, which temporarily halted the progression.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Polineuropatias
/
Neuropatias Amiloides Familiares
Tipo de estudo:
Diagnostic_studies
/
Etiology_studies
Limite:
Humans
Idioma:
De
Ano de publicação:
2022
Tipo de documento:
Article