High-dose cyclophosphamide for graft-versus-host disease prevention

Research output: Contribution to journalReview articlepeer-review

66 Scopus citations


Purpose of Review: Administration of high-dose cyclophosphamide after transplantation inhibits both graft rejection and graft-versus-host disease (GvHD) in mouse models of allogeneic blood or marrow transplantation (alloBMT). This strategy has recently been adapted to human transplantation. Recent Findings: The safety and efficacy of high-dose posttransplantation cyclophosphamide, when given in combination with tacrolimus and mycophenolate mofetil, was first demonstrated after nonmyeloablative conditioning and allografting using human leukocyte antigen (HLA)-mismatched related donors. Further analysis shows that increasing HLA disparity does not worsen overall outcome. High-dose posttransplantation cyclophosphamide was also found to be effective as sole prophylaxis of acute and chronic GvHD after HLA-matched alloBMT. Summary: Taking advantage of the differential susceptibility of proliferating, alloreactive T cells over nonproliferating, nonalloreactive T cells to high-dose cyclophosphamide, and owing to the drug's stem cell sparing effects, this novel strategy provides a unique opportunity to optimize GvHD prophylaxis after HLA-matched alloBMT and increase the use of HLA-mismatched related donors. Well tolerated and effective mismatched related alloBMT provides access to essentially everyone, such as patients with sickle cell anemia, in need of the procedure.

Original languageEnglish (US)
Pages (from-to)493-499
Number of pages7
JournalCurrent opinion in hematology
Issue number6
StatePublished - Nov 2010


  • allogeneic blood or marrow transplantation
  • graft-versus-host disease
  • posttransplantation cyclophosphamide
  • transplantation tolerance

ASJC Scopus subject areas

  • Hematology


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