Aplastic anemia

Curr Opin Hematol. 2008 May;15(3):162-8. doi: 10.1097/MOH.0b013e3282fa7470.


Purpose of review: Most acquired aplastic anemia is the result of immune-mediated destruction of hematopoietic stem cells causing pancytopenia and an empty bone marrow, which can be successfully treated with either immunosuppressive therapy or hematopoietic stem-cell transplantation.

Recent findings: In aplastic anemia, oligoclonally expanded cytotoxic T cells induce apoptosis of hematopoietic progenitors. T-bet, a transcription factor that binds to the interferon-gamma promoter region, is upregulated in aplastic anemia T cells. Regulatory T cells are significantly reduced in patients' peripheral blood and in an aplastic anemia murine model, infusion of regulatory T cells ameliorates disease progression. In a minority of cases, loss-of-function mutations in telomerase complex genes may underlie disease development. Long-term survival, once strongly linked to response to immunosuppressive therapy, can now be achieved even among nonresponders due to significant advances in supportive care and better salvage treatments.

Summary: Evidence has accumulated in the recent years further corroborating an immune-mediated process underlying aplastic anemia pathogenesis. Hematopoietic stem-cell transplantation from a matched sibling donor is preferred for children and young adults with severe aplastic anemia, and immunosuppressive therapy is employed when hematopoietic stem-cell transplantation is not feasible due to age, lack of a histocompatible sibling, co-morbidities, or by patient choice.

Publication types

  • Review

MeSH terms

  • Anemia, Aplastic / physiopathology*
  • Anemia, Aplastic / therapy
  • Antilymphocyte Serum / therapeutic use
  • Hematopoiesis / immunology*
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Immunosuppressive Agents / therapeutic use


  • Antilymphocyte Serum
  • Immunosuppressive Agents