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Review
. 2024 Jan-Dec:33:9636897241246351.
doi: 10.1177/09636897241246351.

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review

Tahereh Rostami  1 , Soroush Rad  2 , Mohammad Reza Rostami  1 , Seied Amirhossein Mirhosseini  3 , Hediyeh Alemi  2   4 , Naghmeh Khavandgar  2   4 , Ghasem Janbabai  1 , Azadeh Kiumarsi  5 , Amir Kasaeian  2   4   6 , Seied Asadollah Mousavi  2
Affiliations
Review

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review

Tahereh Rostami et al. Cell Transplant. 2024 Jan-Dec.

Abstract

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.

Keywords: conditioning regimen; donor selection; posttransplant outcomes; sickle cell disease; stem cell transplantation.

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Conflict of interest statement

Declaration of Conflicting InterestsThe author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

Figures

Figure 1.
Figure 1.
FDA-approved drugs and gene products for the management of SCD.
See this image and copyright information in PMC

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