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Your search keyword '"Prealbumin therapeutic use"' showing total 2 results
Start Over Descriptor "Prealbumin therapeutic use" Publisher munksgaard
2 results on '"Prealbumin therapeutic use"'

1. Treatment of acquired transthyretin amyloidosis in domino liver transplantation.

Author

Tsamis KI, Mytilinaios D, Heneghan M, Gillmore JD, Gilbertson JA, Giannopoulos S, Sarmas I, and Konitsiotis S

Subjects
Humans, Prealbumin genetics, Prealbumin metabolism, Prealbumin therapeutic use, Amyloid Neuropathies, Familial etiology, Amyloid Neuropathies, Familial surgery, Liver Transplantation adverse effects
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 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., (© 2022 The Authors. Clinical Transplantation published by John Wiley & Sons Ltd.)

Published
2023
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2. Heart transplantation in Val142Ile mutation in the modern era: A single center experience.

Author

Lyle MA, Brown MT, Morris AA, Gupta D, Vega JD, Cole RT, and Bhatt KN

Subjects
Humans, Retrospective Studies, Mutation, Prealbumin genetics, Prealbumin therapeutic use, Amyloid Neuropathies, Familial genetics, Amyloid Neuropathies, Familial surgery, Amyloid Neuropathies, Familial complications, Heart Transplantation
Abstract

Little is known about the post heart transplantation management of extra cardiac manifestations in patients with hereditary transthyretin amyloid cardiomyopathy (hATTR-CM) in the new era of disease modifying treatment for ATTR amyloidosis. This is a retrospective study of all patients with hATTR-CM associated with the Val142Ile variant who underwent heart transplantation (HT) from January 2014 to February 2022. All 10 patients with the Val142Ile mutation were successfully transplanted, with a 1 year survival post heart transplantation (HT) of 90%, comparable to an age, sex, and race matched cohort of patients transplanted for non-amyloid indications. However, 4 (40%) of these patients developed progressive extracardiac manifestations requiring initiation of TTR silencer therapy with the small interfering RNA (siRNA) drug patisiran, which was well tolerated with no significant side effects in this population. We recommend formal neurologic evaluation and assessment of extracardiac manifestations annually as part of routine post-transplant care, and disease modifying therapy, aimed at TTR stabilization or silencing, should be initiated in the context of previously untreated extracardiac manifestations or evidence of subclinical neuropathy to prevent progression., (© 2022 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.)

Published
2022
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