PMID- 27913467
OWN - NLM
STAT- MEDLINE
DCOM- 20170529
LR  - 20230928
IS  - 1520-4383 (Electronic)
IS  - 1520-4391 (Print)
IS  - 1520-4383 (Linking)
VI  - 2016
IP  - 1
DP  - 2016 Dec 2
TI  - Transplantation for bone marrow failure: current issues.
PG  - 90-98
AB  - 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.
CI  - (c) 2016 by The American Society of Hematology. All rights reserved.
FAU - Peffault de Latour, Regis
AU  - Peffault de Latour R
AD  - Service d'Hematologie-Greffe, Hopital Saint-Louis, Assistance Publique-Hopitaux 
      de Paris (APHP), Paris, France; Universite Paris Diderot, Institut Universitaire 
      d'Hematologie, Sorbonne Paris Cite, Paris, France; Centre de Reference Aplasie 
      Medullaire, APHP, Paris, France; and Severe Aplastic Anemia Working Party of the 
      European Group for Blood and Marrow Transplantation, Leiden, The Netherlands.
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Hematology Am Soc Hematol Educ Program
JT  - Hematology. American Society of Hematology. Education Program
JID - 100890099
SB  - IM
MH  - Allografts
MH  - Anemia, Aplastic/*therapy
MH  - Chronic Disease
MH  - Graft vs Host Disease/prevention & control
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Hemoglobinuria, Paroxysmal/prevention & control
MH  - Humans
MH  - Quality of Life
MH  - *Unrelated Donors
PMC - PMC6142500
COIS- Conflict-of-interest disclosure: The author has received research funding from, 
      consulted for, and received honoraria from Alexion, Pfizer, and Novartis, and 
      received research funding from Amgen.
EDAT- 2016/12/04 06:00
MHDA- 2017/05/30 06:00
PMCR- 2017/12/02
CRDT- 2016/12/04 06:00
PHST- 2016/12/04 06:00 [entrez]
PHST- 2016/12/04 06:00 [pubmed]
PHST- 2017/05/30 06:00 [medline]
PHST- 2017/12/02 00:00 [pmc-release]
AID - 2016/1/90 [pii]
AID - 088397 [pii]
AID - 10.1182/asheducation-2016.1.90 [doi]
PST - ppublish
SO  - Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):90-98. doi: 
      10.1182/asheducation-2016.1.90.