PMID- 27000737
OWN - NLM
STAT- MEDLINE
DCOM- 20160912
LR  - 20181202
IS  - 1532-8686 (Electronic)
IS  - 0037-1963 (Linking)
VI  - 53
IP  - 2
DP  - 2016 Apr
TI  - Alternative donor allogeneic hematopoietic cell transplantation for 
      hemoglobinopathies.
PG  - 120-8
LID - S0037-1963(16)00002-0 [pii]
LID - 10.1053/j.seminhematol.2016.01.001 [doi]
AB  - Allogeneic hematopoietic stem cell transplantation (HSCT) offers a curative 
      therapy for patients with hemoglobinopathies, mainly severe sickle cell disease 
      (SCD) and thalassemia (TM). However, the applicability of HSCT has been limited 
      mainly by donor availability, with a less than 25%-30% of eligible patients 
      having human leukocyte antigen (HLA)-matched sibling donors. Previous outcomes 
      using alternate donor options have been markedly inferior due to increased 
      regimen-related toxicity, transplant-related mortality, graft failure, and 
      graft-versus-host disease (GVHD). Advances in transplant technology, including 
      high-resolution HLA typing, improved GVHD prophylactic approaches with tolerance 
      induction, and better supportive care over the last decade, are addressing these 
      historical challenges, resulting in increasing donor options. Herein, we review 
      alternate donor HSCT approaches for severe SCD and TM using unrelated donors, 
      umbilical cord blood units, or related haploidentical donors. Though this is an 
      emerging field, early results are promising and in selected patients, this may be 
      the preferred option to mitigate against the age-related morbidity and early 
      mortality associated with these disorders.
CI  - Copyright (c) 2016 Elsevier Inc. All rights reserved.
FAU - Alfraih, Feras
AU  - Alfraih F
AD  - Adult Hematology and Hematopoietic Stem Cell Transplantation, King Faisal 
      Hospital and Research Centre, Riyadh, Saudi Arabia. Electronic address: 
      Falfraih@kfshrc.edu.sa.
FAU - Aljurf, Mahmoud
AU  - Aljurf M
AD  - Molecular and Clinical Hematology Branch, NHLBI, NIH, Bethesda, MD, USA.
FAU - Fitzhugh, Courtney D
AU  - Fitzhugh CD
AD  - Division of Hematology and Oncology, Department of Medicine and Vanderbilt- 
      Meharry Center for Excellence in Sickle Cell Disease, Vanderbilt University 
      Medical Center, Nashville, TN, USA.
FAU - Kassim, Adetola A
AU  - Kassim AA
AD  - Division of Hematology and Oncology, Department of Medicine and Vanderbilt- 
      Meharry Center for Excellence in Sickle Cell Disease, Vanderbilt University 
      Medical Center, Nashville, TN, USA.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20160115
PL  - United States
TA  - Semin Hematol
JT  - Seminars in hematology
JID - 0404514
SB  - IM
MH  - Graft vs Host Disease/etiology
MH  - Haploidy
MH  - *Hematopoietic Stem Cell Transplantation/adverse effects
MH  - Hemoglobinopathies/*therapy
MH  - Histocompatibility Testing
MH  - Humans
MH  - Transplantation, Homologous
OTO - NOTNLM
OT  - Allogeneic hematopoietic stem cell transplantation
OT  - Alternative donor
OT  - Hemoglobinopathies
OT  - Sickle cell disease
OT  - Thalassemia
EDAT- 2016/03/24 06:00
MHDA- 2016/09/13 06:00
CRDT- 2016/03/23 06:00
PHST- 2016/03/23 06:00 [entrez]
PHST- 2016/03/24 06:00 [pubmed]
PHST- 2016/09/13 06:00 [medline]
AID - S0037-1963(16)00002-0 [pii]
AID - 10.1053/j.seminhematol.2016.01.001 [doi]
PST - ppublish
SO  - Semin Hematol. 2016 Apr;53(2):120-8. doi: 10.1053/j.seminhematol.2016.01.001. 
      Epub 2016 Jan 15.