A prospective multicenter study of paroxysmal nocturnal hemoglobinuria cells in patients with bone marrow failure
Article first published online: 12 NOV 2013
© 2013 The Authors. Published by Wiley Periodicals, Inc.
This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.
Cytometry Part B: Clinical Cytometry
Volume 86, Issue 3, pages 175–182, May 2014
How to Cite
How to cite this article: A Prospective Multicenter Study of Paroxysmal Nocturnal Hemoglobinuria Cells in Patients with Bone Marrow Failure. Cytometry Part B 2014:86B:175–182., , , , , , , , .
- Issue published online: 9 APR 2014
- Article first published online: 12 NOV 2013
- Manuscript Accepted: 8 OCT 2013
- Manuscript Revised: 22 JUL 2013
- Manuscript Received: 7 MAY 2013
- Alexion Pharmaceuticals
- paroxysmal nocturnal hemoglobinuria;
- bone marrow dysfunction;
- clinical trial;
- flow cytometry;
Paroxysmal nocturnal hemoglobinuria (PNH), a rare clonal hematopoietic stem cell disorder, is characterized by chronic, uncontrolled complement activation leading to intravascular hemolysis and an inflammatory prothrombotic state. The EXPLORE study aimed to determine the prevalence of undiagnosed PNH in patients with aplastic anemia (AA), myelodysplastic syndrome (MDS), and/or other bone marrow failure (BMF) syndromes and the effect of PNH clone size on hemolysis.
Patients, selected from medical office chart reviews, had blood samples collected for hematologic panel testing and for flow cytometry detection of PNH clones.
Granulocyte PNH clones ≥ 1% were detected in 199 of all 5,398 patients (3.7%), 93 of 503 AA patients (18.5%), 50 of 4,401 MDS patients (1.1%), and 3 of 130 other BMF patients (2.3%). Higher-sensitivity analyses detected PNH clones ≥ 0.01% in 167 of 1,746 patients from all groups (9.6%) and in 22 of 1,225 MDS patients (1.8%), 116 of 294 AA patients (39.5%), and four of 54 other BMF patients (7.8%). Among patients with PNH clones ≥ 1%, median clone size was smaller in patients with AA (5.1%) than in those with MDS (17.6%) or other BMF (24.4%), and the percentage of patients with lactate dehydrogenase levels (a marker for intravascular hemolysis) ≥ 1.5 × upper limit of normal was smaller in patients with AA (18.3%) than in those with MDS (42.0%).
These results confirm the presence of PNH clones in high-risk patient groups and suggest that screening of such patients may facilitate patient management and care. © 2013 The Authors. Published by Wiley Periodicals, Inc.