2023
DOI: 10.3389/fmed.2023.1036939
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Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review

Abstract: Sickle cell disease (SCD) had first been mentioned in the literature a century ago. Advancement in the molecular basis of the pathophysiology of the disease opens the door for various therapeutic options. Though life-extending treatments are available for treating patients with SCD, allogeneic hematopoietic stem cell transplantation (HSCT) is the only option as of yet. A major obstacle before HSCT to cure patients with SCD is the availability of donors. Matched sibling donors are available only for a small per… Show more

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Cited by 14 publications
(7 citation statements)
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“…HSCT is currently a curative treatment for severe SCD 7 10 . Five-year overall survival (OS) and event-free survival (EFS) of patients who received allo-HSCT from a human leukocyte antigen (HLA)-identical sibling donor have been reported to be 92%–94% and 84%–92%, respectively, with an improved OS in children younger than 16 years (95% vs 81%, P < 0.001) 11 .…”
Section: Allogeneic Hsct In Scdmentioning
confidence: 99%
“…HSCT is currently a curative treatment for severe SCD 7 10 . Five-year overall survival (OS) and event-free survival (EFS) of patients who received allo-HSCT from a human leukocyte antigen (HLA)-identical sibling donor have been reported to be 92%–94% and 84%–92%, respectively, with an improved OS in children younger than 16 years (95% vs 81%, P < 0.001) 11 .…”
Section: Allogeneic Hsct In Scdmentioning
confidence: 99%
“…Reduce the burden of sickled cells [94,95] Hydroxyurea Increase fetal hemoglobin (HbF) to stop polymers forming in the sickle hemoglobin [96] Hematopoietic stem cell transplantation Reverse the sickle phenotype [97,98] L-glutamine Antioxidant effects [99] Hemoglobin S (HbS) polymerization inhibitors Prevent HbS polymerization [100] Monoclonal antibody (crizanlizumab) Reduce selectin-mediated adhesion [101] Gene editing therapy (Casgevy™) Editing faulty gene in a patient's bone marrow stem cells [102] Recent Advanced Therapeutic Approaches for Sickle Cell Disease…”
Section: Blood Transfusionmentioning
confidence: 99%
“…However, due in part to the cytotoxicity of conventional conditioning regimens, the benefits of HSCT must be weighed against the risks of treatment-related adverse events, which may be severe enough to prevent older or infirmed patients from accessing the curative potential of transplantation 3 . Furthermore, they may impede the safe application of HSCT for non-malignant blood diseases such as sickle cell anemia 4 .…”
Section: Introductionmentioning
confidence: 99%