Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review.
Tahereh RostamiSoroush RadMohammad Reza RostamiSeied Amirhossein MirhosseiniHediyeh AlemiNaghmeh KhavandgarGhasem JanbabaiAzadeh KiumarsiAmir KasaeianSeied Asadollah MousaviPublished in: Cell transplantation (2024)
While exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) have been approved by the US Food and Drug Administration (FDA) as the first cell-based gene therapies for the treatment of patients 12 years of age and older with sickle cell disease (SCD), this treatment is not universally accessible. Allogeneic hematopoietic stem cell transplant (HSCT) has the potential to eradicate the symptoms of patients with SCD, but a significant obstacle in HSCT for SCD is the availability of suitable donors, particularly human leukocyte antigen (HLA)-matched related donors. Furthermore, individuals with SCD face an elevated risk of complications during stem cell transplantation due to SCD-related tissue damage, endothelial activation, and inflammation. Therefore, it is imperative to consider optimal conditioning regimens and investigate HSCT from alternative donors. This review encompasses information on the use of HSCT in patients with SCD, including the indications for HSCT, conditioning regimens, alternative donors, and posttransplant outcomes.
Keyphrases
- hematopoietic stem cell
- stem cell transplantation
- sickle cell disease
- drug administration
- endothelial cells
- high dose
- kidney transplantation
- oxidative stress
- acute myeloid leukemia
- physical activity
- type diabetes
- stem cells
- single cell
- low dose
- genome wide
- cell therapy
- climate change
- risk assessment
- health information
- combination therapy
- depressive symptoms
- pluripotent stem cells
- replacement therapy