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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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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). |
format | Online Article Text |
id | pubmed-8146901 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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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