Hematopoietic stem cell transplantation for acquired aplastic anemia

George E. Georges*, Rainer Storb

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

22 Scopus citations


Purpose of review There has been a steady improvement in outcomes with allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA), because of progress in optimization of the conditioning regimens, donor hematopoietic cell source, and supportive care. Here, we review recently published data that highlight the improvements and current issues in the treatment of SAA. Recent findings Approximately one-third of aplastic anemia patients treated with immune suppressive therapy (IST) have acquired mutations in myeloid cancer candidate genes. Because of the greater probability for eventual failure of IST, human leukocyte antigen (HLA)-matched sibling donor BMT is the first-line of treatment for SAA. HLA-matched unrelated donor (URD) BMT is generally recommended for patients who have failed IST. However, in younger patients for whom a 10/10-HLA-allele matched URD can be rapidly identified, there is a strong rationale to proceed with URD BMT as first-line therapy. HLA-haploidentical BMT using posttransplant cyclophosphamide conditioning regimens is now a reasonable second-line treatment for patients who failed IST. Summary Improved outcomes have led to an increased first-line role of BMT for treatment of SAA. The optimal cell source from an HLA-matched donor is bone marrow. Additional studies are needed to determine the optimal conditioning regimen for HLA-haploidentical donors.

Original languageEnglish (US)
Pages (from-to)495-500
Number of pages6
JournalCurrent opinion in hematology
Issue number6
StatePublished - Nov 1 2016


  • allogeneic bone marrow transplantation
  • antithymocyte globulin
  • severe aplastic anemia

ASJC Scopus subject areas

  • Hematology


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