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Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area

Background: Over the past decade, three new drugs have been approved for the treatment of hereditary amyloid transthyretin (ATTRv) polyneuropathy. The aim of this work was to analyze whether current therapies prolong survival for patients affected by ATTRv amyloidosis. Methods: The study was conduct...

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Autores principales: Russo, Massimo, Gentile, Luca, Di Stefano, Vincenzo, Di Bella, Gianluca, Minutoli, Fabio, Toscano, Antonio, Brighina, Filippo, Vita, Giuseppe, Mazzeo, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8146901/
https://www.ncbi.nlm.nih.gov/pubmed/33925301
http://dx.doi.org/10.3390/brainsci11050545
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author Russo, Massimo
Gentile, Luca
Di Stefano, Vincenzo
Di Bella, Gianluca
Minutoli, Fabio
Toscano, Antonio
Brighina, Filippo
Vita, Giuseppe
Mazzeo, Anna
author_facet Russo, Massimo
Gentile, Luca
Di Stefano, Vincenzo
Di Bella, Gianluca
Minutoli, Fabio
Toscano, Antonio
Brighina, Filippo
Vita, Giuseppe
Mazzeo, Anna
author_sort Russo, Massimo
collection PubMed
description Background: Over the past decade, three new drugs have been approved for the treatment of hereditary amyloid transthyretin (ATTRv) polyneuropathy. The aim of this work was to analyze whether current therapies prolong survival for patients affected by ATTRv amyloidosis. Methods: The study was conducted retrospectively, analyzing the medical records of 105 patients with genetic diagnoses of familial amyloidotic polyneuropathy followed at the two referral centers for the disease in Sicily, Italy. Of these, 71 received disease-modifying therapy, while 34 received only symptomatic treatment or no therapy. Results: The most used treatment in our patient cohort was tafamidis, followed by liver transplantation, patisiran, inotersen, and diflunisal. The median survival was significantly longer for treated vs. untreated patients (12 years vs. 8 years). In the 71 patients who received disease-modifying treatment, the presence of cardiac involvement, weight loss, or autonomic dysfunction at diagnosis was not related to survival. Conversely, patients diagnosed in the early stage of the disease (PND 1) had significantly longer survival than those diagnosed in the late stage (PND 2–4).
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spelling pubmed-81469012021-05-26 Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area Russo, Massimo Gentile, Luca Di Stefano, Vincenzo Di Bella, Gianluca Minutoli, Fabio Toscano, Antonio Brighina, Filippo Vita, Giuseppe Mazzeo, Anna Brain Sci Article Background: Over the past decade, three new drugs have been approved for the treatment of hereditary amyloid transthyretin (ATTRv) polyneuropathy. The aim of this work was to analyze whether current therapies prolong survival for patients affected by ATTRv amyloidosis. Methods: The study was conducted retrospectively, analyzing the medical records of 105 patients with genetic diagnoses of familial amyloidotic polyneuropathy followed at the two referral centers for the disease in Sicily, Italy. Of these, 71 received disease-modifying therapy, while 34 received only symptomatic treatment or no therapy. Results: The most used treatment in our patient cohort was tafamidis, followed by liver transplantation, patisiran, inotersen, and diflunisal. The median survival was significantly longer for treated vs. untreated patients (12 years vs. 8 years). In the 71 patients who received disease-modifying treatment, the presence of cardiac involvement, weight loss, or autonomic dysfunction at diagnosis was not related to survival. Conversely, patients diagnosed in the early stage of the disease (PND 1) had significantly longer survival than those diagnosed in the late stage (PND 2–4). MDPI 2021-04-27 /pmc/articles/PMC8146901/ /pubmed/33925301 http://dx.doi.org/10.3390/brainsci11050545 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
Russo, Massimo
Gentile, Luca
Di Stefano, Vincenzo
Di Bella, Gianluca
Minutoli, Fabio
Toscano, Antonio
Brighina, Filippo
Vita, Giuseppe
Mazzeo, Anna
Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area
title Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area
title_full Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area
title_fullStr Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area
title_full_unstemmed Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area
title_short Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area
title_sort use of drugs for attrv amyloidosis in the real world: how therapy is changing survival in a non-endemic area
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8146901/
https://www.ncbi.nlm.nih.gov/pubmed/33925301
http://dx.doi.org/10.3390/brainsci11050545
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