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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...
Autores principales: | , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2014
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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. |
format | Online Article Text |
id | pubmed-4090189 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2014 |
publisher | Public Library of Science |
record_format | MEDLINE/PubMed |
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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