PMID- 31258425
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20201001
IS  - 1524-5012 (Print)
IS  - 1524-5012 (Linking)
VI  - 19
IP  - 2
DP  - 2019 Summer
TI  - Curative Therapies for Sickle Cell Disease.
PG  - 131-137
LID - 10.31486/toj.18.0044 [doi]
AB  - Background: Sickle cell disease (SCD) is an inherited hemoglobinopathy associated 
      with severe morbidity, impaired quality of life, and premature mortality. 
      Hematopoietic stem cell transplantation (HSCT) is the only curative treatment 
      available for patients with SCD and has a >90% event-free survival when a matched 
      related donor is used. However, availability of human leukocyte antigen 
      (HLA)-identical sibling donors for the SCD population is limited. The use of 
      HLA-matched unrelated donors or related haploidentical donors has the potential 
      to expand the donor pool. Methods: We reviewed the current literature on the 
      indications for SCD transplantation, donor options, and the emerging use of gene 
      therapy as a treatment option. Google Scholar and PubMed were searched using the 
      terms SCD, bone marrow transplantation, donor sources, gene therapy, HSCT, and 
      HLA matching. Additional articles were identified from the bibliographies of 
      retrieved articles. All articles were reviewed for pertinent information related 
      to SCD and transplantation. Results: HSCT has the potential to establish 
      donor-derived normal erythropoiesis with stable long-term engraftment, 
      amelioration of symptoms, and stabilization of organ damage. The majority of HSCT 
      has been performed in children from HLA-identical sibling donors and has resulted 
      in excellent rates of survival. The use of alternate donors such as HLA-matched 
      unrelated donors and haploidentical donors has the potential to expand the 
      applicability of HSCT for SCD. Early results in gene therapy for SCD are 
      encouraging. Conclusion: Evaluation of the long-term benefits of curative 
      therapies for SCD requires comparative clinical trials and studies of late 
      effects.
FAU - Khemani, Kirshma
AU  - Khemani K
AD  - Department of Pediatric Hematology-Oncology-Bone Marrow Transplant, Aflac Cancer 
      and Blood Disorders Center, Emory University School of Medicine, Atlanta, GA.
AD  - Children's Healthcare of Atlanta, Atlanta, GA.
FAU - Katoch, Deeksha
AU  - Katoch D
AD  - Department of Pediatric Hematology-Oncology-Bone Marrow Transplant, Aflac Cancer 
      and Blood Disorders Center, Emory University School of Medicine, Atlanta, GA.
AD  - Children's Healthcare of Atlanta, Atlanta, GA.
FAU - Krishnamurti, Lakshmanan
AU  - Krishnamurti L
AD  - Department of Pediatric Hematology-Oncology-Bone Marrow Transplant, Aflac Cancer 
      and Blood Disorders Center, Emory University School of Medicine, Atlanta, GA.
AD  - Children's Healthcare of Atlanta, Atlanta, GA.
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Ochsner J
JT  - Ochsner journal
JID - 101125795
PMC - PMC6584191
OTO - NOTNLM
OT  - Anemia-sickle cell
OT  - bone marrow transplantation
OT  - genetic therapy
OT  - stem cell transplantation
EDAT- 2019/07/02 06:00
MHDA- 2019/07/02 06:01
PMCR- 2019/06/01
CRDT- 2019/07/02 06:00
PHST- 2019/07/02 06:00 [entrez]
PHST- 2019/07/02 06:00 [pubmed]
PHST- 2019/07/02 06:01 [medline]
PHST- 2019/06/01 00:00 [pmc-release]
AID - toj.18.0044 [pii]
AID - 10.31486/toj.18.0044 [doi]
PST - ppublish
SO  - Ochsner J. 2019 Summer;19(2):131-137. doi: 10.31486/toj.18.0044.