PMID- 37800996
OWN - NLM
STAT- MEDLINE
DCOM- 20231120
LR  - 20240124
IS  - 1747-4094 (Electronic)
IS  - 1747-4094 (Linking)
VI  - 16
IP  - 11
DP  - 2023 Jul-Dec
TI  - Outcomes and long-term effects of hematopoietic stem cell transplant in sickle 
      cell disease.
PG  - 879-903
LID - 10.1080/17474086.2023.2268271 [doi]
AB  - INTRODUCTION: Hematopoietic stem cell transplant (HSCT) is the only readily 
      available curative option for sickle cell disease (SCD). Cure rates following 
      human leukocyte antigen (HLA)-matched related donor HSCT with myeloablative or 
      non-myeloablative conditioning are >90%. Alternative donor sources, including 
      haploidentical donor and autologous with gene therapy, expand donor options but 
      are limited by inferior outcomes, limited data, and/or shorter follow-up and 
      therefore remain experimental. AREAS COVERED: Outcomes are improving with time, 
      with donor type and conditioning regimens having the greatest impact on long-term 
      complications. Patients with stable donor engraftment do not experience 
      SCD-related symptoms and have stabilization or improvement of end-organ 
      pathology; however, the long-term effects of curative strategies remain to be 
      fully established and have significant implications in a patient's decision to 
      seek therapy. This review covers currently published literature on HSCT outcomes, 
      including organ-specific outcomes implicated in SCD, as well as long-term 
      effects. EXPERT OPINION: HSCT, both allogeneic and autologous gene therapy, in 
      the SCD population reverses the sickle phenotype, prevents further organ damage, 
      can resolve prior organ dysfunction in both pediatric and adult patients. Data 
      support greater success with HSCT at a younger age, thus, curative therapies 
      should be discussed early in the patient's life.
FAU - Inam, Zaina
AU  - Inam Z
AD  - Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood 
      Institute, National Institutes of Health, Bethesda, MD, USA.
AD  - Center for Cancer and Blood Disorders, Children's National Hospital, Washington, 
      DC, USA.
FAU - Tisdale, John F
AU  - Tisdale JF
AD  - Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood 
      Institute, National Institutes of Health, Bethesda, MD, USA.
FAU - Leonard, Alexis
AU  - Leonard A
AD  - Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood 
      Institute, National Institutes of Health, Bethesda, MD, USA.
AD  - Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, 
      USA.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20231117
PL  - England
TA  - Expert Rev Hematol
JT  - Expert review of hematology
JID - 101485942
SB  - IM
MH  - Adult
MH  - Humans
MH  - Child
MH  - *Hematopoietic Stem Cell Transplantation/adverse effects
MH  - *Anemia, Sickle Cell/complications
MH  - Transplantation, Homologous
MH  - Tissue Donors
MH  - Transplantation Conditioning/adverse effects
MH  - *Graft vs Host Disease/etiology
OTO - NOTNLM
OT  - Allogeneic transplantation
OT  - alternative donor transplants
OT  - autologous transplantation
OT  - gene therapy
OT  - hematopoietic stem cell transplant
OT  - long-term effects
OT  - organ function
OT  - sickle cell disease
EDAT- 2023/10/06 12:45
MHDA- 2023/11/20 06:54
CRDT- 2023/10/06 09:42
PHST- 2023/11/20 06:54 [medline]
PHST- 2023/10/06 12:45 [pubmed]
PHST- 2023/10/06 09:42 [entrez]
AID - 10.1080/17474086.2023.2268271 [doi]
PST - ppublish
SO  - Expert Rev Hematol. 2023 Jul-Dec;16(11):879-903. doi: 
      10.1080/17474086.2023.2268271. Epub 2023 Nov 17.