PMID- 31731790
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20200928
IS  - 2077-0383 (Print)
IS  - 2077-0383 (Electronic)
IS  - 2077-0383 (Linking)
VI  - 8
IP  - 11
DP  - 2019 Nov 15
TI  - Choice of Donor Source and Conditioning Regimen for Hematopoietic Stem Cell 
      Transplantation in Sickle Cell Disease.
LID - 10.3390/jcm8111997 [doi]
LID - 1997
AB  - In the United States, one out of every 500 African American children have sickle 
      cell disease (SCD), and SCD affects approximately 100,000 Americans. Significant 
      advances in the treatment of this monogenetic disorder have failed to 
      substantially extend the life expectancy of adults with SCD over the past two 
      decades. Hematopoietic stem cell transplantation (HSCT) remains the only curative 
      option for patients with SCD. While human leukocyte antigen (HLA) matched sibling 
      HSCT has been successful, its availability is extremely limited. This review 
      summarizes various conditioning regimens that are currently available. We explore 
      recent efforts to expand the availability of allogeneic HSCT, including matched 
      unrelated, umbilical cord blood, and haploidentical stem cell sources. We 
      consider the use of nonmyeloablative conditioning and haploidentical donor 
      sources as emerging strategies to expand transplant availability, particularly 
      for SCD patients with complications and comorbidities who can undergo neither 
      matched related transplant nor myeloablative conditioning. Finally, we show that 
      improved conditioning agents have improved success rates not only in the 
      HLA-matched sibling setting but also alternative donor settings.
FAU - Limerick, Emily
AU  - Limerick E
AD  - Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood 
      Institute, Bethesda, MD 20892, USA.
FAU - Fitzhugh, Courtney
AU  - Fitzhugh C
AD  - Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood 
      Institute, Bethesda, MD 20892, USA.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20191115
PL  - Switzerland
TA  - J Clin Med
JT  - Journal of clinical medicine
JID - 101606588
PMC - PMC6912427
OTO - NOTNLM
OT  - allogeneic hematopoietic stem cell transplantation
OT  - alternative donor
OT  - graft-versus-host disease (GVHD)
OT  - haploidentical transplantation
OT  - human leukocyte antigen (HLA) matched sibling
OT  - sickle cell disease
COIS- The authors declare no conflict of interest.
EDAT- 2019/11/17 06:00
MHDA- 2019/11/17 06:01
PMCR- 2019/11/15
CRDT- 2019/11/17 06:00
PHST- 2019/09/28 00:00 [received]
PHST- 2019/11/07 00:00 [revised]
PHST- 2019/11/08 00:00 [accepted]
PHST- 2019/11/17 06:00 [entrez]
PHST- 2019/11/17 06:00 [pubmed]
PHST- 2019/11/17 06:01 [medline]
PHST- 2019/11/15 00:00 [pmc-release]
AID - jcm8111997 [pii]
AID - jcm-08-01997 [pii]
AID - 10.3390/jcm8111997 [doi]
PST - epublish
SO  - J Clin Med. 2019 Nov 15;8(11):1997. doi: 10.3390/jcm8111997.