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

Jeffrey R. Andolina, Ariel L. Reinish, Razia Akhtar, Suzie Noronha, Jessica C. Shand, Angela Girvin, David N. Korones, Lauren B. Bruckner, Craig A. Mullen, Kevin J. Curran, Farid Boulad

Research output: Contribution to journalArticlepeer-review

4 Scopus citations

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.

Original languageEnglish
Article numbere27218
JournalPediatric Blood and Cancer
Volume65
Issue number8
DOIs
StatePublished - Aug 2018
Externally publishedYes

Keywords

  • paroxysmal nocturnal hemoglobinuria
  • pediatric
  • stem cell transplantation

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