Last marrow standing: bone marrow transplantation for acquired bone marrow failure conditions

Curr Hematol Malig Rep. 2012 Dec;7(4):292-9. doi: 10.1007/s11899-012-0138-x.

Abstract

Paroxysmal nocturnal hemoglobinuria, aplastic anemia, and myelodysplastic syndrome are a spectrum of acquired marrow failure, having a common pathologic thread of both immune dysregulation and the development of abnormal hematopoiesis. Allogeneic hematopoietic cell transplantation plays a critical role in the treatment of these disorders and, for many patients, is the only treatment modality with demonstrated curative potential. In recent years, there have been many breakthroughs in the understanding of the pathogenesis of these uncommon disorders. The subsequent advances in non-transplant therapies, along with concurrent improvement in outcomes after hematopoietic cell transplantation, necessitate continual appraisal of the indications, timing, and approaches to transplantation for acquired marrow failure syndromes. We review here contemporary and critical new findings driving current treatment decisions.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Anemia, Aplastic / drug therapy
  • Anemia, Aplastic / surgery*
  • Antibodies, Monoclonal, Humanized / therapeutic use
  • Bone Marrow Transplantation / methods*
  • Combined Modality Therapy
  • Hemoglobinuria, Paroxysmal / drug therapy
  • Hemoglobinuria, Paroxysmal / surgery*
  • Humans
  • Myelodysplastic Syndromes / drug therapy
  • Myelodysplastic Syndromes / surgery*
  • Transplantation Conditioning / methods

Substances

  • Antibodies, Monoclonal, Humanized
  • eculizumab