PMID- 36096996
OWN - NLM
STAT- MEDLINE
DCOM- 20221025
LR  - 20221025
IS  - 1473-0502 (Print)
IS  - 1473-0502 (Linking)
VI  - 61
IP  - 5
DP  - 2022 Oct
TI  - Umbilical cord blood as a hematopoietic stem cell source in transplantation for 
      pediatric sickle cell disease: current challenges and strategies.
PG  - 103554
LID - S1473-0502(22)00238-5 [pii]
LID - 10.1016/j.transci.2022.103554 [doi]
AB  - Allogeneic hematopoietic stem cell transplant (HSCT) is the only established cure 
      for sickle cell disease (SCD), a hemolytic disorder that arises due to a point 
      mutation in the hemoglobin A gene. The result is an abnormal sickle hemoglobin 
      (HbS) replacing hemoglobin A. Of the spectrum of sickle hemoglobinoapthies, 
      homozygous (HbSS) and HbSbeta(0) thalassemia manifest severe forms of disease 
      characterized by chronic endothelial injury/vasculopathy, ischemic pain, and 
      vital organ damage that commence in childhood and escalate with age resulting in 
      impaired quality of life, increased healthcare burden, and early mortality. HSCT 
      has demonstrated durable disease control and regression of symptoms. Human 
      leukocyte antigen (HLA) matched sibling donor (MSD) transplantation can achieve 
      disease-free survival of > 90%. However, < 18% of SCD patients in the United 
      States have a MSD. Familial mismatched and unrelated donors from registries 
      provide alternate stem cell sources. Umbilical cord blood (UCB) from family or 
      cord blood banks expand donor sources and are attractive due to donor-independent 
      ease of use and availability. These naive cells tolerate greater degrees of 
      HLA-mismatch. The primary challenge with UCB is optimizing cell dose toward 
      successful engraftment and timely immune reconstitution while minimizing 
      graft-versus-host disease (GVHD). This review summarizes evidence that UCB 
      remains a promising stem cell source where modern methods of graft expansion, 
      conditioning, GVHD prophylaxis, and infection control can overcome these 
      challenges and retain the value of this intervention.
CI  - Copyright (c) 2022 Elsevier Ltd. All rights reserved.
FAU - Malhotra, Megha
AU  - Malhotra M
AD  - Washington University School of Medicine, Division of Pediatric Hematology 
      Oncology, Department of Pediatrics, 660 S. Euclid Avenue, Campus Box 8116, St. 
      Louis, MO 63110, USA. Electronic address: m.malhotra@wustl.edu.
FAU - Shenoy, Shalini
AU  - Shenoy S
AD  - Washington University School of Medicine, Division of Pediatric Hematology 
      Oncology, Department of Pediatrics, 660 S. Euclid Avenue, Campus Box 8116, St. 
      Louis, MO 63110, USA. Electronic address: shalinishenoy@wustl.edu.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20220829
PL  - England
TA  - Transfus Apher Sci
JT  - Transfusion and apheresis science : official journal of the World Apheresis 
      Association : official journal of the European Society for Haemapheresis
JID - 101095653
RN  - 0 (Hemoglobin, Sickle)
RN  - 0 (HLA Antigens)
MH  - Humans
MH  - Child
MH  - *Graft vs Host Disease
MH  - Fetal Blood
MH  - Hemoglobin, Sickle
MH  - Quality of Life
MH  - Unrelated Donors
MH  - *Hematopoietic Stem Cell Transplantation/methods
MH  - *Anemia, Sickle Cell/therapy
MH  - *Cord Blood Stem Cell Transplantation
MH  - Hematopoietic Stem Cells
MH  - HLA Antigens
OTO - NOTNLM
OT  - Allogeneic hematopoietic stem cell transplant
OT  - Engraftment
OT  - Graft-versus-host disease
OT  - Immune reconstitution
OT  - Sickle cell disease
OT  - Umbilical cord blood transplant
COIS- Declaration of Competing Interest None.
EDAT- 2022/09/13 06:00
MHDA- 2022/10/26 06:00
CRDT- 2022/09/12 23:50
PHST- 2022/09/13 06:00 [pubmed]
PHST- 2022/10/26 06:00 [medline]
PHST- 2022/09/12 23:50 [entrez]
AID - S1473-0502(22)00238-5 [pii]
AID - 10.1016/j.transci.2022.103554 [doi]
PST - ppublish
SO  - Transfus Apher Sci. 2022 Oct;61(5):103554. doi: 10.1016/j.transci.2022.103554. 
      Epub 2022 Aug 29.