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Trajectories of Kidney Function in Patients with ATTRv Treated with Gene Silencers
Hereditary transthyretin amyloidosis (ATTRv; v for “variant”) is the most common form of hereditary amyloidosis, with an autosomal dominant inheritance and a variable penetrance. This disease has a significant variability in clinical presentation and multiorgan involvement. While kidney involvement...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9777815/ https://www.ncbi.nlm.nih.gov/pubmed/36553503 http://dx.doi.org/10.3390/genes13122236 |
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author | Luigetti, Marco Guglielmino, Valeria Romano, Angela Sciarrone, Maria Ausilia Vitali, Francesca D’Ambrosio, Viola Ferraro, Pietro Manuel |
author_facet | Luigetti, Marco Guglielmino, Valeria Romano, Angela Sciarrone, Maria Ausilia Vitali, Francesca D’Ambrosio, Viola Ferraro, Pietro Manuel |
author_sort | Luigetti, Marco |
collection | PubMed |
description | Hereditary transthyretin amyloidosis (ATTRv; v for “variant”) is the most common form of hereditary amyloidosis, with an autosomal dominant inheritance and a variable penetrance. This disease has a significant variability in clinical presentation and multiorgan involvement. While kidney involvement in early-onset ATTRv has been reported in one-third of patients, in late-onset ATTRv it has generally been considered rare. In the present study, we describe trajectories of kidney function over time before and after treatment with gene silencing therapies in a cohort of 17 ATTRv patients with different mutations, coming from Italy (nine subjects treated with inotersen and eight patients treated with patisiran). The analysis of estimated glomerular filtration rate (eGFR) slopes revealed that the average change in eGFR was 0.01 mL/min/1.73 m(2) per month before initiation and −0.23 mL/min/1.73 m(2) per month during follow-up for inotersen and −0.62 mL/min/1.73 m(2) per month before initiation and −0.20 mL/min/1.73 m(2) per month during follow-up for patisiran. In conclusion, we did not observe any significant difference either between the two groups of treatment or within-group before and after therapy, so gene-silencing therapies may be considered safe for renal function in ATTRv and are not associated with a worsening of eGFR slope. |
format | Online Article Text |
id | pubmed-9777815 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-97778152022-12-23 Trajectories of Kidney Function in Patients with ATTRv Treated with Gene Silencers Luigetti, Marco Guglielmino, Valeria Romano, Angela Sciarrone, Maria Ausilia Vitali, Francesca D’Ambrosio, Viola Ferraro, Pietro Manuel Genes (Basel) Communication Hereditary transthyretin amyloidosis (ATTRv; v for “variant”) is the most common form of hereditary amyloidosis, with an autosomal dominant inheritance and a variable penetrance. This disease has a significant variability in clinical presentation and multiorgan involvement. While kidney involvement in early-onset ATTRv has been reported in one-third of patients, in late-onset ATTRv it has generally been considered rare. In the present study, we describe trajectories of kidney function over time before and after treatment with gene silencing therapies in a cohort of 17 ATTRv patients with different mutations, coming from Italy (nine subjects treated with inotersen and eight patients treated with patisiran). The analysis of estimated glomerular filtration rate (eGFR) slopes revealed that the average change in eGFR was 0.01 mL/min/1.73 m(2) per month before initiation and −0.23 mL/min/1.73 m(2) per month during follow-up for inotersen and −0.62 mL/min/1.73 m(2) per month before initiation and −0.20 mL/min/1.73 m(2) per month during follow-up for patisiran. In conclusion, we did not observe any significant difference either between the two groups of treatment or within-group before and after therapy, so gene-silencing therapies may be considered safe for renal function in ATTRv and are not associated with a worsening of eGFR slope. MDPI 2022-11-29 /pmc/articles/PMC9777815/ /pubmed/36553503 http://dx.doi.org/10.3390/genes13122236 Text en © 2022 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 | Communication Luigetti, Marco Guglielmino, Valeria Romano, Angela Sciarrone, Maria Ausilia Vitali, Francesca D’Ambrosio, Viola Ferraro, Pietro Manuel Trajectories of Kidney Function in Patients with ATTRv Treated with Gene Silencers |
title | Trajectories of Kidney Function in Patients with ATTRv Treated with Gene Silencers |
title_full | Trajectories of Kidney Function in Patients with ATTRv Treated with Gene Silencers |
title_fullStr | Trajectories of Kidney Function in Patients with ATTRv Treated with Gene Silencers |
title_full_unstemmed | Trajectories of Kidney Function in Patients with ATTRv Treated with Gene Silencers |
title_short | Trajectories of Kidney Function in Patients with ATTRv Treated with Gene Silencers |
title_sort | trajectories of kidney function in patients with attrv treated with gene silencers |
topic | Communication |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9777815/ https://www.ncbi.nlm.nih.gov/pubmed/36553503 http://dx.doi.org/10.3390/genes13122236 |
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