PMID- 18991653
OWN - NLM
STAT- MEDLINE
DCOM- 20090204
LR  - 20190923
IS  - 1566-5240 (Print)
IS  - 1566-5240 (Linking)
VI  - 8
IP  - 7
DP  - 2008 Nov
TI  - Recent advances in bone marrow transplantation in hemoglobinopathies.
PG  - 675-89
AB  - Allogeneic hematopoietic cell transplantation (HCT) is currently the only 
      treatment with curative potential for sickle cell disease (SCD) and 
      beta-thalassemia. HCT was first used to treat SCD and thalassemia more than two 
      decades ago, and with increasing experience this treatment modality has shifted 
      from being an experimental intervention to one in which selected patient 
      populations are targeted for treatment. Recent multicenter clinical studies show 
      an event-free survival (EFS) of 85% after human leukocyte antigen (HLA)-identical 
      sibling transplantation for SCD, using conventional myeloablative conditioning 
      with a backbone of busulfan (BU) and cyclophosphamide (CY) [1-3]. Results of HCT 
      for thalassemia show very similar outcomes, with EFS probabilities that range 
      from 81%-87% [4,5]. However, the risk of graft failure, recurrent disease, 
      graft-versus-host-disease (GVHD), infections, and long-term sequelae of chronic 
      GVHD and endocrinopathies related to Fe overload and myeloablative BU limit 
      broader application of this therapy. Non-myeloablative conditioning regimens may 
      offer a lower risk of toxicity, and investigations to identify a regimen that is 
      sufficiently immunosuppressive to ensure stable engraftment of donor cells are 
      ongoing. Alternative sources of donor hematopoietic cells that include 
      HLA-matched unrelated donor (URD) and umbilical cord blood (UCB), are being 
      pursued for hemoglobinopathies, with promising initial results. This review 
      discusses the successes, challenges and future direction of HCT for SCD and 
      thalassemia.
FAU - Michlitsch, Jennifer G
AU  - Michlitsch JG
AD  - Children's Hospital & Research Center, Oakland, Oakland, CA, USA.
FAU - Walters, Mark C
AU  - Walters MC
LA  - eng
PT  - Journal Article
PT  - Review
PL  - Netherlands
TA  - Curr Mol Med
JT  - Current molecular medicine
JID - 101093076
SB  - IM
MH  - Follow-Up Studies
MH  - Graft Rejection/etiology
MH  - Graft vs Host Disease/etiology
MH  - Hematopoietic Stem Cell Transplantation/adverse effects/*trends
MH  - Hemoglobinopathies/complications/mortality/*therapy
MH  - Humans
MH  - Transplantation Chimera
RF  - 101
EDAT- 2008/11/11 09:00
MHDA- 2009/02/05 09:00
CRDT- 2008/11/11 09:00
PHST- 2008/11/11 09:00 [pubmed]
PHST- 2009/02/05 09:00 [medline]
PHST- 2008/11/11 09:00 [entrez]
AID - 10.2174/156652408786241393 [doi]
PST - ppublish
SO  - Curr Mol Med. 2008 Nov;8(7):675-89. doi: 10.2174/156652408786241393.