Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina

Light chain (AL) amyloidosis is a form of systemic amyloidosis, causing organ dysfunction, mainly affecting the heart and kidney. Patient-tailored and risk-adapted decision making is critical in AL amyloidosis management. There is limited real-world evidence data from Argentina and Latin America reg...

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Autores principales: Posadas-Martinez, Maria Lourdes, Aguirre, María Adela, Brulc, Erika, Saez, Maria Soledad, Sorroche, Patricia, Machnicki, Gerardo, Fernandez, Mariana, Nucifora, Elsa Mercedes
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9612475/
https://www.ncbi.nlm.nih.gov/pubmed/36301970
http://dx.doi.org/10.1371/journal.pone.0274578
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author Posadas-Martinez, Maria Lourdes
Aguirre, María Adela
Brulc, Erika
Saez, Maria Soledad
Sorroche, Patricia
Machnicki, Gerardo
Fernandez, Mariana
Nucifora, Elsa Mercedes
author_facet Posadas-Martinez, Maria Lourdes
Aguirre, María Adela
Brulc, Erika
Saez, Maria Soledad
Sorroche, Patricia
Machnicki, Gerardo
Fernandez, Mariana
Nucifora, Elsa Mercedes
author_sort Posadas-Martinez, Maria Lourdes
collection PubMed
description Light chain (AL) amyloidosis is a form of systemic amyloidosis, causing organ dysfunction, mainly affecting the heart and kidney. Patient-tailored and risk-adapted decision making is critical in AL amyloidosis management. There is limited real-world evidence data from Argentina and Latin America regarding the treatment approaches for AL amyloidosis. This retrospective cohort study aimed to describe the treatment patterns and outcomes in adult patients (>18 years) diagnosed with AL amyloidosis at the Hospital Italiano in Buenos Aires, Argentina, using a 10-yearfollow-up data (June 1, 2010 to May 31, 2019) from the institutional registry of amyloidosis (IRA). The study population had a mean age of 63 years and 54.4% weremale. Heart and kidney were the most frequently affected organs. Of the 90 eligible patients included in the study, 70underwent treatment. Bortezomib-based regimen was the preferred first-line treatment (75.7% patients). Overall,54.4% of the patients presented a deep response (complete or very good partial response). Median overall survival (OS) was 5years, the 1-year OS and progression free survival rates were 80% (95% confidence interval [CI]: 68–87) and 80% (95%CI 68–87)), respectively. This study provides vital real-world evidence for the long-term treatment patterns and survival in a large cohort of AL amyloidosis patients in Argentina.
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spelling pubmed-96124752022-10-28 Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina Posadas-Martinez, Maria Lourdes Aguirre, María Adela Brulc, Erika Saez, Maria Soledad Sorroche, Patricia Machnicki, Gerardo Fernandez, Mariana Nucifora, Elsa Mercedes PLoS One Research Article Light chain (AL) amyloidosis is a form of systemic amyloidosis, causing organ dysfunction, mainly affecting the heart and kidney. Patient-tailored and risk-adapted decision making is critical in AL amyloidosis management. There is limited real-world evidence data from Argentina and Latin America regarding the treatment approaches for AL amyloidosis. This retrospective cohort study aimed to describe the treatment patterns and outcomes in adult patients (>18 years) diagnosed with AL amyloidosis at the Hospital Italiano in Buenos Aires, Argentina, using a 10-yearfollow-up data (June 1, 2010 to May 31, 2019) from the institutional registry of amyloidosis (IRA). The study population had a mean age of 63 years and 54.4% weremale. Heart and kidney were the most frequently affected organs. Of the 90 eligible patients included in the study, 70underwent treatment. Bortezomib-based regimen was the preferred first-line treatment (75.7% patients). Overall,54.4% of the patients presented a deep response (complete or very good partial response). Median overall survival (OS) was 5years, the 1-year OS and progression free survival rates were 80% (95% confidence interval [CI]: 68–87) and 80% (95%CI 68–87)), respectively. This study provides vital real-world evidence for the long-term treatment patterns and survival in a large cohort of AL amyloidosis patients in Argentina. Public Library of Science 2022-10-27 /pmc/articles/PMC9612475/ /pubmed/36301970 http://dx.doi.org/10.1371/journal.pone.0274578 Text en © 2022 Posadas-Martinez et al https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
spellingShingle Research Article
Posadas-Martinez, Maria Lourdes
Aguirre, María Adela
Brulc, Erika
Saez, Maria Soledad
Sorroche, Patricia
Machnicki, Gerardo
Fernandez, Mariana
Nucifora, Elsa Mercedes
Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina
title Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina
title_full Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina
title_fullStr Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina
title_full_unstemmed Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina
title_short Treatment patterns and outcomes in light chain amyloidosis: An institutional registry of amyloidosis report in Argentina
title_sort treatment patterns and outcomes in light chain amyloidosis: an institutional registry of amyloidosis report in argentina
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9612475/
https://www.ncbi.nlm.nih.gov/pubmed/36301970
http://dx.doi.org/10.1371/journal.pone.0274578
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