Transplantation for bone marrow failure: current issues.

Abstract

The preferred treatment of idiopathic aplastic anemia (AA) is allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-identical sibling donor. Transplantation from a well-matched unrelated donor (MUD) may be considered for patients without a sibling donor after failure of immunosuppressive therapy, as may alternative transplantation (mismatched, cord blood or haplo-identical HSCT) for patients without a MUD. HSCT may also be contemplated for congenital disorders in cases of pancytopenia or severe isolated cytopenia. Currently, HSCT aims are not only to cure patients but also to avoid long-term complications, notably chronic graft-versus-host disease (GVHD), essential for a good quality of life long term. This paper summarizes recent advances in HSCT for idiopathic and inherited AA disorders. The effect of age on current transplantation outcomes, the role of transplantation in paroxysmal nocturnal hemoglobinuria, and the prevention of GVHD are also discussed. Emerging strategies regarding the role of up-front unrelated donor and alternative donor HSCT in idiopathic AA, along with advances in the treatment of clonal evolution in Fanconi anemia, are also examined.

• Authors: Régis Peffault de Latour
• Journal: Hematology. American Society of Hematology. Education Program (Hematology Am Soc Hematol Educ Program) Vol. 2016 Issue 1 Pg. 90-98 (Dec 02 2016) ISSN: 1520-4383 [Electronic] United States
• PMID: 27913467 (Publication Type: Journal Article, Review)
• Copyright: © 2016 by The American Society of Hematology. All rights reserved.

Topics

• Allografts
• Anemia, Aplastic (therapy)
• Chronic Disease
• Graft vs Host Disease (prevention & control)
• Hematopoietic Stem Cell Transplantation
• Hemoglobinuria, Paroxysmal (prevention & control)
• Humans
• Quality of Life
• Unrelated Donors