Successful reduced-intensity conditioning hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria with aplastic anemia in two children Journal Article


Authors: Andolina, J. R.; Reinish, A. L.; Akhtar, R.; Noronha, S.; Shand, J. C.; Girvin, A.; Korones, D. N.; Bruckner, L. B.; Mullen, C. A.; Curran, K. J.; Boulad, F.
Article Title: Successful reduced-intensity conditioning hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria with aplastic anemia in two children
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare cause of bone marrow failure in children. We report two children who presented with pancytopenia, and were diagnosed with PNH with severe aplastic anemia. Both children underwent upfront, successful hematopoietic stem cell transplantation with reduced-intensity conditioning. One patient had a syngeneic donor, and one patient had a 10/10 matched unrelated donor. Neither patient developed graft versus host disease, infections, or recurrent PNH. Reduced-intensity conditioning hematopoietic stem cell transplantation is a reasonable therapy for PNH with marrow failure in children. © 2018 Wiley Periodicals, Inc.
Keywords: stem cell transplantation; pediatric; paroxysmal nocturnal hemoglobinuria
Journal Title: Pediatric Blood and Cancer
Volume: 65
Issue: 8
ISSN: 1545-5009
Publisher: Wiley Periodicals, Inc  
Date Published: 2018-08-01
Start Page: e27218
Language: English
DOI: 10.1002/pbc.27218
PROVIDER: scopus
PUBMED: 29722478
DOI/URL:
Notes: Article -- Export Date: 1 August 2018 -- Source: Scopus
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MSK Authors
  1. Farid Boulad
    254 Boulad
  2. Kevin Joseph Curran
    58 Curran