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Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study

A multicentre study evaluating the presence of glycosil phosphatidyl-inositol (GPI)-negative populations was performed in 85 children with acquired aplastic anemia (AA). A GPI-negative population was observed in 41% of patients at diagnosis, 48% during immune-suppressive therapy (IST), and 45% in pa...

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Autores principales: Timeus, Fabio, Crescenzio, Nicoletta, Longoni, Daniela, Doria, Alessandra, Foglia, Luiselda, Pagliano, Sara, Vallero, Stefano, Decimi, Valentina, Svahn, Johanna, Palumbo, Giuseppe, Ruggiero, Antonio, Martire, Baldassarre, Pillon, Marta, Marra, Nicoletta, Dufour, Carlo, Ramenghi, Ugo, Saracco, Paola
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4090189/
https://www.ncbi.nlm.nih.gov/pubmed/25007335
http://dx.doi.org/10.1371/journal.pone.0101948
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author Timeus, Fabio
Crescenzio, Nicoletta
Longoni, Daniela
Doria, Alessandra
Foglia, Luiselda
Pagliano, Sara
Vallero, Stefano
Decimi, Valentina
Svahn, Johanna
Palumbo, Giuseppe
Ruggiero, Antonio
Martire, Baldassarre
Pillon, Marta
Marra, Nicoletta
Dufour, Carlo
Ramenghi, Ugo
Saracco, Paola
author_facet Timeus, Fabio
Crescenzio, Nicoletta
Longoni, Daniela
Doria, Alessandra
Foglia, Luiselda
Pagliano, Sara
Vallero, Stefano
Decimi, Valentina
Svahn, Johanna
Palumbo, Giuseppe
Ruggiero, Antonio
Martire, Baldassarre
Pillon, Marta
Marra, Nicoletta
Dufour, Carlo
Ramenghi, Ugo
Saracco, Paola
author_sort Timeus, Fabio
collection PubMed
description A multicentre study evaluating the presence of glycosil phosphatidyl-inositol (GPI)-negative populations was performed in 85 children with acquired aplastic anemia (AA). A GPI-negative population was observed in 41% of patients at diagnosis, 48% during immune-suppressive therapy (IST), and 45% in patients off-therapy. No association was found between the presence of a GPI-negative population at diagnosis and the response to IST. In addition, the response rate to IST did not differ between the patients who were GPI-positive at diagnosis and later developed GPI-negative populations and the 11 patients who remained GPI-positive. Two patients with a GPI-negative population >10%, and laboratory signs of hemolysis without hemoglobinuria were considered affected by paroxysmal nocturnal hemoglobinuria (PNH) secondary to AA; no thrombotic event was reported. Excluding the 2 patients with a GPI-negative population greater than 10%, we did not observe a significant correlation between LDH levels and GPI-negative population size. In this study monitoring for laboratory signs of hemolysis was sufficient to diagnose PNH in AA patients. The presence of minor GPI-negative populations at diagnosis in our series did not influence the therapeutic response. As occasionally the appearance of a GPI-negative population was observed at cyclosporine (CSA) tapering or AA relapse, a possible role of GPI-negative population monitoring during IST modulation may need further investigation.
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spelling pubmed-40901892014-07-14 Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study Timeus, Fabio Crescenzio, Nicoletta Longoni, Daniela Doria, Alessandra Foglia, Luiselda Pagliano, Sara Vallero, Stefano Decimi, Valentina Svahn, Johanna Palumbo, Giuseppe Ruggiero, Antonio Martire, Baldassarre Pillon, Marta Marra, Nicoletta Dufour, Carlo Ramenghi, Ugo Saracco, Paola PLoS One Research Article A multicentre study evaluating the presence of glycosil phosphatidyl-inositol (GPI)-negative populations was performed in 85 children with acquired aplastic anemia (AA). A GPI-negative population was observed in 41% of patients at diagnosis, 48% during immune-suppressive therapy (IST), and 45% in patients off-therapy. No association was found between the presence of a GPI-negative population at diagnosis and the response to IST. In addition, the response rate to IST did not differ between the patients who were GPI-positive at diagnosis and later developed GPI-negative populations and the 11 patients who remained GPI-positive. Two patients with a GPI-negative population >10%, and laboratory signs of hemolysis without hemoglobinuria were considered affected by paroxysmal nocturnal hemoglobinuria (PNH) secondary to AA; no thrombotic event was reported. Excluding the 2 patients with a GPI-negative population greater than 10%, we did not observe a significant correlation between LDH levels and GPI-negative population size. In this study monitoring for laboratory signs of hemolysis was sufficient to diagnose PNH in AA patients. The presence of minor GPI-negative populations at diagnosis in our series did not influence the therapeutic response. As occasionally the appearance of a GPI-negative population was observed at cyclosporine (CSA) tapering or AA relapse, a possible role of GPI-negative population monitoring during IST modulation may need further investigation. Public Library of Science 2014-07-09 /pmc/articles/PMC4090189/ /pubmed/25007335 http://dx.doi.org/10.1371/journal.pone.0101948 Text en © 2014 Timeus et al http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are properly credited.
spellingShingle Research Article
Timeus, Fabio
Crescenzio, Nicoletta
Longoni, Daniela
Doria, Alessandra
Foglia, Luiselda
Pagliano, Sara
Vallero, Stefano
Decimi, Valentina
Svahn, Johanna
Palumbo, Giuseppe
Ruggiero, Antonio
Martire, Baldassarre
Pillon, Marta
Marra, Nicoletta
Dufour, Carlo
Ramenghi, Ugo
Saracco, Paola
Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study
title Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study
title_full Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study
title_fullStr Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study
title_full_unstemmed Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study
title_short Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study
title_sort paroxysmal nocturnal hemoglobinuria clones in children with acquired aplastic anemia: a multicentre study
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4090189/
https://www.ncbi.nlm.nih.gov/pubmed/25007335
http://dx.doi.org/10.1371/journal.pone.0101948
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