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- Title
Paroxysmal Nocturnal Hemoglobinuria Clones in Children with Acquired Aplastic Anemia: A Multicentre Study.
- Authors
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
- Abstract
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.
- Subjects
CLONING; PAROXYSMAL hemoglobinuria; APLASTIC anemia; PHOSPHATIDYL inositol; JUVENILE diseases; IMMUNOSUPPRESSION
- Publication
PLoS ONE, 2014, Vol 9, Issue 7, p1
- ISSN
1932-6203
- Publication type
Article
- DOI
10.1371/journal.pone.0101948