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Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study

Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of cytopenias, with limited data on management and outcome. We describe a large multicentric series of 259 patients, focusing on clinical and molecular features, treatment, evolution,...

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Autores principales: Fattizzo, Bruno, Gurnari, Carmelo, Cassanello, Giulio, Bortolotti, Marta, Awada, Hussein, Giammarco, Sabrina, Consonni, Dario, Sica, Simona, Gandhi, Shreyans, Trikha, Roochi, Large, Joanna, Salter, Sarah, Maciejewski, Jaroslaw P., Barcellini, Wilma, Kulasekararaj, Austin G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10681892/
https://www.ncbi.nlm.nih.gov/pubmed/37794100
http://dx.doi.org/10.1038/s41375-023-02047-z
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author Fattizzo, Bruno
Gurnari, Carmelo
Cassanello, Giulio
Bortolotti, Marta
Awada, Hussein
Giammarco, Sabrina
Consonni, Dario
Sica, Simona
Gandhi, Shreyans
Trikha, Roochi
Large, Joanna
Salter, Sarah
Maciejewski, Jaroslaw P.
Barcellini, Wilma
Kulasekararaj, Austin G.
author_facet Fattizzo, Bruno
Gurnari, Carmelo
Cassanello, Giulio
Bortolotti, Marta
Awada, Hussein
Giammarco, Sabrina
Consonni, Dario
Sica, Simona
Gandhi, Shreyans
Trikha, Roochi
Large, Joanna
Salter, Sarah
Maciejewski, Jaroslaw P.
Barcellini, Wilma
Kulasekararaj, Austin G.
author_sort Fattizzo, Bruno
collection PubMed
description Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of cytopenias, with limited data on management and outcome. We describe a large multicentric series of 259 patients, focusing on clinical and molecular features, treatment, evolution, and survival. The majority required treatment with cyclosporine (CyA) alone (N = 84) or in combination with anti-thymocyte globulin (ATG,44) or eltrombopag (20), eltrombopag alone (10), or others (25) including androgens. Similar outcomes were observed across different strategies, with a 6-month overall response rate of 73% for CyA, 74% for ATG plus CyA, 68% for CyA plus eltrombopag, 87% for eltrombopag, and 79% for others. Notably, 56 patients (39%), mainly receiving CyA plus eltrombopag, achieved a trilineage response (p = 0.02). Progression to myeloid neoplasms was limited (8%) and not related to mutational status. Hemolytic PNH developed in 10% of cases, being predicted by detection of small clones at diagnosis. Survival was negatively impacted by age, male gender, LDH, platelets/erythrocyte transfusion need, and somatic mutations by NGS, and positively by higher neutrophils at diagnosis, PNH clones, and trilineage response at 6 and 12 months. Multivariable analysis confirmed the detrimental role of age and the favorable association with PNH clone and trilineage response at 6 months.
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spelling pubmed-106818922023-11-30 Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study Fattizzo, Bruno Gurnari, Carmelo Cassanello, Giulio Bortolotti, Marta Awada, Hussein Giammarco, Sabrina Consonni, Dario Sica, Simona Gandhi, Shreyans Trikha, Roochi Large, Joanna Salter, Sarah Maciejewski, Jaroslaw P. Barcellini, Wilma Kulasekararaj, Austin G. Leukemia Article Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of cytopenias, with limited data on management and outcome. We describe a large multicentric series of 259 patients, focusing on clinical and molecular features, treatment, evolution, and survival. The majority required treatment with cyclosporine (CyA) alone (N = 84) or in combination with anti-thymocyte globulin (ATG,44) or eltrombopag (20), eltrombopag alone (10), or others (25) including androgens. Similar outcomes were observed across different strategies, with a 6-month overall response rate of 73% for CyA, 74% for ATG plus CyA, 68% for CyA plus eltrombopag, 87% for eltrombopag, and 79% for others. Notably, 56 patients (39%), mainly receiving CyA plus eltrombopag, achieved a trilineage response (p = 0.02). Progression to myeloid neoplasms was limited (8%) and not related to mutational status. Hemolytic PNH developed in 10% of cases, being predicted by detection of small clones at diagnosis. Survival was negatively impacted by age, male gender, LDH, platelets/erythrocyte transfusion need, and somatic mutations by NGS, and positively by higher neutrophils at diagnosis, PNH clones, and trilineage response at 6 and 12 months. Multivariable analysis confirmed the detrimental role of age and the favorable association with PNH clone and trilineage response at 6 months. Nature Publishing Group UK 2023-10-04 2023 /pmc/articles/PMC10681892/ /pubmed/37794100 http://dx.doi.org/10.1038/s41375-023-02047-z Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) .
spellingShingle Article
Fattizzo, Bruno
Gurnari, Carmelo
Cassanello, Giulio
Bortolotti, Marta
Awada, Hussein
Giammarco, Sabrina
Consonni, Dario
Sica, Simona
Gandhi, Shreyans
Trikha, Roochi
Large, Joanna
Salter, Sarah
Maciejewski, Jaroslaw P.
Barcellini, Wilma
Kulasekararaj, Austin G.
Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study
title Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study
title_full Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study
title_fullStr Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study
title_full_unstemmed Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study
title_short Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study
title_sort deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10681892/
https://www.ncbi.nlm.nih.gov/pubmed/37794100
http://dx.doi.org/10.1038/s41375-023-02047-z
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