PMID- 29883237
OWN - NLM
STAT- MEDLINE
DCOM- 20190228
LR  - 20230921
IS  - 1747-4094 (Electronic)
IS  - 1747-4086 (Print)
IS  - 1747-4094 (Linking)
VI  - 11
IP  - 7
DP  - 2018 Jul
TI  - Stem cell transplantation in sickle cell disease: therapeutic potential and 
      challenges faced.
PG  - 547-565
LID - 10.1080/17474086.2018.1486703 [doi]
AB  - Sickle cell disease (SCD) is the most common inherited hemoglobinopathy 
      worldwide, and is a life-limiting disease with limited therapeutic options to 
      reduce disease severity. Despite being a monogenic disorder, the clinical 
      phenotypes of SCD are variable, with few reliable predictors of disease severity 
      easily identifying patients where the benefits of curative therapy outweigh the 
      risks. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only 
      curative option, though significant advances in gene therapy raise the promise 
      for additional curative methods. Areas covered: Allogeneic transplantation in SCD 
      has evolved and improved over the last two decades, now offering a standard of 
      care curative option using a human leukocyte antigen (HLA)-matched sibling donor. 
      Many of the seminal transplantation studies are reviewed here, demonstrating how 
      initial failures and successes have influenced and led to current HSCT 
      strategies. Such strategies aim to overcome setbacks and limitations, and focus 
      on conditioning regimens, immune suppression methods, the use alternative donor 
      sources, and gene therapy approaches. Expert commentary: SCD is a curable 
      disease. Each dedicated effort to refine transplantation methods, expand the 
      donor pool, and bring gene therapy models to fruition will make enormous impacts 
      reducing disease burden and improving outcomes and quality of life for patients 
      with SCD.
FAU - Leonard, Alexis
AU  - Leonard A
AD  - a Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood 
      Institute , National Institutes of Health , Bethesda , MD , USA.
AD  - b Center for Cancer and Blood Disorders , Children's National Health System , 
      Washington , DC , USA.
FAU - Tisdale, John F
AU  - Tisdale JF
AD  - a Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood 
      Institute , National Institutes of Health , Bethesda , MD , USA.
LA  - eng
GR  - Z99 HL999999/ImNIH/Intramural NIH HHS/United States
PT  - Journal Article
PT  - Review
DEP - 20180620
PL  - England
TA  - Expert Rev Hematol
JT  - Expert review of hematology
JID - 101485942
SB  - IM
MH  - Allografts
MH  - Anemia, Sickle Cell/genetics/metabolism/pathology/*therapy
MH  - Genetic Therapy/methods
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Histocompatibility Testing
MH  - Humans
MH  - Siblings
PMC - PMC8459571
MID - NIHMS1036456
OTO - NOTNLM
OT  - Allogeneic transplantation
OT  - autologous transplantation
OT  - gene therapy
OT  - hematopoietic stem cell transplantation
OT  - sickle cell disease
EDAT- 2018/06/09 06:00
MHDA- 2019/03/01 06:00
PMCR- 2021/09/23
CRDT- 2018/06/09 06:00
PHST- 2018/06/09 06:00 [pubmed]
PHST- 2019/03/01 06:00 [medline]
PHST- 2018/06/09 06:00 [entrez]
PHST- 2021/09/23 00:00 [pmc-release]
AID - 10.1080/17474086.2018.1486703 [doi]
PST - ppublish
SO  - Expert Rev Hematol. 2018 Jul;11(7):547-565. doi: 10.1080/17474086.2018.1486703. 
      Epub 2018 Jun 20.