Paroxysmal nocturnal hemoglobinuria clones in children with acquired aplastic anemia: A multicentre study

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

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Article numbere101948
JournalPLoS One
Issue number7
Publication statusPublished - Jul 9 2014

ASJC Scopus subject areas

  • Agricultural and Biological Sciences(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Medicine(all)


Dive into the research topics of 'Paroxysmal nocturnal hemoglobinuria clones in children with acquired aplastic anemia: A multicentre study'. Together they form a unique fingerprint.

Cite this