Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review
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 hemat...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-02-01
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| Series: | Frontiers in Medicine |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fmed.2023.1036939/full |
| _version_ | 1828006121928916992 |
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| author | Nishka Bhalla Anjali Bhargav Sandeep Kumar Yadav Aloukick Kumar Singh |
| author_facet | Nishka Bhalla Anjali Bhargav Sandeep Kumar Yadav Aloukick Kumar Singh |
| author_sort | Nishka Bhalla |
| collection | DOAJ |
| description | 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 percentage of patients. To expand the donor pool, different contrasting approaches of allogeneic HSCT like T-cell replete and deplete have been tested. None of those tested approaches have been without the risk of GvHD and graft rejection. Other limitations such as transplantation-related infections and organ dysfunction caused by the harsh conditioning regimen need to be addressed on a priority basis. In this review, we will discuss available allogeneic HSCT approaches to cure SCD, as well as recent advancements to make the approach safer. The center of interest is using megadose T-cell-depleted bone marrow in conjugation with donor-derived CD8 veto T cells to achieve engraftment and tolerance across MHC barriers, under reduced intensity conditioning (RIC). This approach is in phase I/II clinical trial at the MD Anderson Cancer Centre and is open to patients with hemoglobinopathies. |
| first_indexed | 2024-04-10T07:52:28Z |
| format | Article |
| id | doaj.art-cc8faec1571640b99f12ca68b51d7225 |
| institution | Directory Open Access Journal |
| issn | 2296-858X |
| language | English |
| last_indexed | 2024-04-10T07:52:28Z |
| publishDate | 2023-02-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Medicine |
| spelling | doaj.art-cc8faec1571640b99f12ca68b51d72252023-02-23T09:32:31ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2023-02-011010.3389/fmed.2023.10369391036939Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A reviewNishka Bhalla0Anjali Bhargav1Sandeep Kumar Yadav2Aloukick Kumar Singh3Centre for Stem Cell Research, Christian Medical College, Vellore, Tamilnadu, IndiaCentre for Stem Cell Research, Christian Medical College, Vellore, Tamilnadu, IndiaUniversity of Texas MD Anderson Cancer Center, Houston, TX, United StatesCentre for Stem Cell Research, Christian Medical College, Vellore, Tamilnadu, IndiaSickle 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 percentage of patients. To expand the donor pool, different contrasting approaches of allogeneic HSCT like T-cell replete and deplete have been tested. None of those tested approaches have been without the risk of GvHD and graft rejection. Other limitations such as transplantation-related infections and organ dysfunction caused by the harsh conditioning regimen need to be addressed on a priority basis. In this review, we will discuss available allogeneic HSCT approaches to cure SCD, as well as recent advancements to make the approach safer. The center of interest is using megadose T-cell-depleted bone marrow in conjugation with donor-derived CD8 veto T cells to achieve engraftment and tolerance across MHC barriers, under reduced intensity conditioning (RIC). This approach is in phase I/II clinical trial at the MD Anderson Cancer Centre and is open to patients with hemoglobinopathies.https://www.frontiersin.org/articles/10.3389/fmed.2023.1036939/fullsickle cell diseasehematopoietic stem cell transplantationreduced intensity conditioningveto cellbone marrow transplantation |
| spellingShingle | Nishka Bhalla Anjali Bhargav Sandeep Kumar Yadav Aloukick Kumar Singh Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review Frontiers in Medicine sickle cell disease hematopoietic stem cell transplantation reduced intensity conditioning veto cell bone marrow transplantation |
| title | Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review |
| title_full | Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review |
| title_fullStr | Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review |
| title_full_unstemmed | Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review |
| title_short | Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review |
| title_sort | allogeneic hematopoietic stem cell transplantation to cure sickle cell disease a review |
| topic | sickle cell disease hematopoietic stem cell transplantation reduced intensity conditioning veto cell bone marrow transplantation |
| url | https://www.frontiersin.org/articles/10.3389/fmed.2023.1036939/full |
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