Acquired Aplastic Anemia: What Have We Learned and What Is in the Horizon?

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Acquired aplastic anemia (aAA) characterized by peripheral pancytopenia and bone marrow aplasia is a rare and serious disorder. Differential diagnosis includes constitutional bone marrow failure syndromes and myelodysplastic disorders. Autoimmune reaction to altered hematopoietic stem cells highlights the underlying mechanism. Matched related donor allogeneic hematopoietic stem cell transplantation is the ideal pediatric treatment; alternative approaches include immunosuppressive therapy and use of eltrombopag. Progression to clonal disorders can occur. Recently, alternative donor hematopoietic stem cell transplantation outcomes have significantly improved. Despite advances, aAA continues to be a challenge for hematologists.

Original languageEnglish
Pages (from-to)597-606
Number of pages10
JournalPediatric Clinics of North America
Issue number3
StatePublished - Jun 2018


  • Acquired aplastic anemia
  • Autoimmunity
  • Clonality
  • Hematopoietic stem cell
  • Hematopoietic stem cell transplantation
  • Immunosuppressive therapy
  • Myelodysplastic syndrome


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