title: 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.affiliation: Service d'Hématologie-Greffe, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris (APHP), Paris, France; Université Paris Diderot, Institut Universitaire d'Hématologie, Sorbonne Paris Cité, Paris, France; Centre de Référence Aplasie Médullaire, APHP, Paris, France; and Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation, Leiden, The Netherlands.
authors.forename: Régis
authors.initials: R
authors.lastname: Peffault de Latour
mesh_terms: D064591:Allografts; D000741:Anemia, Aplastic / Q000628:therapy*; D002908:Chronic Disease; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006457:Hemoglobinuria, Paroxysmal / Q000517:prevention & control; D006801:Humans; D011788:Quality of Life; D061349:Unrelated Donors