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Detection of paroxysmal nocturnal hemoglobinuria clones to exclude inherited bone marrow failure syndromes.

AbstractBACKGROUND:
Inherited bone marrow failure syndromes (IBMFS) and acquired aplastic anemia (AA) are life-threatening marrow failure disorders. These entities can be difficult to distinguish because they present similarly. Correct diagnosis is imperative for proper therapy.
DESIGN AND METHODS:
This is a retrospective, single-center study of patients <40 yr of age, evaluated for bone marrow failure, and assayed for the presence of a PNH clone in the pediatric or adult hematology/oncology clinics from 2001 to present. Patients were also evaluated for IBMFS.
RESULTS:
We present results from 156 patients with marrow failure, 20 of whom have IBMFS. None of the IBMSF patients had paroxysmal nocturnal hemoglobinuria (PNH) clones.
CONCLUSIONS:
Although further studies are needed, our results suggest that the detection of a PNH clone can be a useful diagnostic tool to exclude the diagnosis of IBMFS and focus the work-up and treatment on an acquired form of marrow failure.
AuthorsAmy E DeZern, Heather J Symons, Linda S Resar, Michael J Borowitz, Mary Y Armanios, Robert A Brodsky
JournalEuropean journal of haematology (Eur J Haematol) Vol. 92 Issue 6 Pg. 467-70 (Jun 2014) ISSN: 1600-0609 [Electronic] England
PMID24612308 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
Copyright© 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
Topics
  • Adolescent
  • Adult
  • Age Factors
  • Anemia, Aplastic
  • Bone Marrow (pathology)
  • Bone Marrow Diseases
  • Bone Marrow Failure Disorders
  • Child
  • Child, Preschool
  • Clonal Evolution
  • Diagnosis, Differential
  • Female
  • Hemoglobinuria, Paroxysmal (diagnosis, genetics, pathology)
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Retrospective Studies
  • Young Adult

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