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Development of hemolytic paroxysmal nocturnal hemoglobinuria without graft loss following hematopoietic stem cell transplantation for acquired aplastic anemia.

Authors
  • Oved, Joseph H1, 2, 3
  • Stanley, Natasha3
  • Babushok, Daria V3, 4
  • Huang, Yanping5
  • Duke, Jamie L5
  • Monos, Dimitrios S5
  • Teachey, David T2
  • Olson, Timothy S1, 2, 3
  • 1 Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
  • 2 Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
  • 3 Comprehensive Bone Marrow Failure Center, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
  • 4 Division of Hematology-Oncology, Hospital of University of Pennsylvania, Philadelphia, Pennsylvania.
  • 5 Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Type
Published Article
Journal
Pediatric Transplantation
Publisher
Wiley (Blackwell Publishing)
Publication Date
Jun 01, 2019
Volume
23
Issue
4
Identifiers
DOI: 10.1111/petr.13393
PMID: 30900367
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

PNH is the most common clonal hematopoietic disorder arising in patients with aAA. PNH is caused by mutations in PIGA, a gene that encodes the catalytic subunit of an enzyme involved in the biosynthesis of GPI anchors, transmembrane glycolipids required for cell surface expression of many proteins. PNH clones likely arise as immune escape mechanisms in aAA by preventing CD1D-restricted T-cell recognition of GPI anchors and GPI-linked autoantigens. Though many patients with aAA treated with IST will develop subclinical PNH clones, only a subset will develop PNH disease, characterized by increased thrombosis, intravascular hemolysis, and potential for severe organ dysfunction. In contrast to IST, allogeneic HSCT for patients with aAA is thought to cure bone marrow aplasia and prevent hematopoietic clonal evolution to PNH. Herein, we present a phenomenon of host-derived PNH disease arising in a patient with aAA many years following MSD-BMT, highlighting the importance of monitoring for this clonal disease in aAA patients with stable mixed donor/recipient chimerism after HSCT. We also provide a literature review for similar occurrences of PNH arising after HSCT. © 2019 Wiley Periodicals, Inc.

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