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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doaj-d06a6d05cde24c73bef923f786e5d24c2020-11-24T21:24:26ZengPublic Library of Science (PLoS)PLoS ONE1932-62032014-01-0197e10194810.1371/journal.pone.0101948Paroxysmal nocturnal hemoglobinuria clones in children with acquired aplastic anemia: a multicentre study.Fabio TimeusNicoletta CrescenzioDaniela LongoniAlessandra DoriaLuiselda FogliaSara PaglianoStefano ValleroValentina DecimiJohanna SvahnGiuseppe PalumboAntonio RuggieroBaldassarre MartireMarta PillonNicoletta MarraCarlo DufourUgo RamenghiPaola SaraccoA 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.http://europepmc.org/articles/PMC4090189?pdf=render |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Fabio Timeus Nicoletta Crescenzio Daniela Longoni Alessandra Doria Luiselda Foglia Sara Pagliano Stefano Vallero Valentina Decimi Johanna Svahn Giuseppe Palumbo Antonio Ruggiero Baldassarre Martire Marta Pillon Nicoletta Marra Carlo Dufour Ugo Ramenghi Paola Saracco |
spellingShingle |
Fabio Timeus Nicoletta Crescenzio Daniela Longoni Alessandra Doria Luiselda Foglia Sara Pagliano Stefano Vallero Valentina Decimi Johanna Svahn Giuseppe Palumbo Antonio Ruggiero Baldassarre Martire Marta Pillon Nicoletta Marra Carlo Dufour Ugo Ramenghi Paola Saracco Paroxysmal nocturnal hemoglobinuria clones in children with acquired aplastic anemia: a multicentre study. PLoS ONE |
author_facet |
Fabio Timeus Nicoletta Crescenzio Daniela Longoni Alessandra Doria Luiselda Foglia Sara Pagliano Stefano Vallero Valentina Decimi Johanna Svahn Giuseppe Palumbo Antonio Ruggiero Baldassarre Martire Marta Pillon Nicoletta Marra Carlo Dufour Ugo Ramenghi Paola Saracco |
author_sort |
Fabio Timeus |
title |
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_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_sort |
paroxysmal nocturnal hemoglobinuria clones in children with acquired aplastic anemia: a multicentre study. |
publisher |
Public Library of Science (PLoS) |
series |
PLoS ONE |
issn |
1932-6203 |
publishDate |
2014-01-01 |
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. |
url |
http://europepmc.org/articles/PMC4090189?pdf=render |
work_keys_str_mv |
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