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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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Autores principales: Bhalla, Nishka, Bhargav, Anjali, Yadav, Sandeep Kumar, Singh, Aloukick Kumar
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9995916/
https://www.ncbi.nlm.nih.gov/pubmed/36910492
http://dx.doi.org/10.3389/fmed.2023.1036939
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author Bhalla, Nishka
Bhargav, Anjali
Yadav, Sandeep Kumar
Singh, Aloukick Kumar
author_facet Bhalla, Nishka
Bhargav, Anjali
Yadav, Sandeep Kumar
Singh, Aloukick Kumar
author_sort Bhalla, Nishka
collection PubMed
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.
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spelling pubmed-99959162023-03-10 Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review Bhalla, Nishka Bhargav, Anjali Yadav, Sandeep Kumar Singh, Aloukick Kumar Front Med (Lausanne) Medicine 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. Frontiers Media S.A. 2023-02-23 /pmc/articles/PMC9995916/ /pubmed/36910492 http://dx.doi.org/10.3389/fmed.2023.1036939 Text en Copyright © 2023 Bhalla, Bhargav, Yadav and Singh. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Medicine
Bhalla, Nishka
Bhargav, Anjali
Yadav, Sandeep Kumar
Singh, Aloukick Kumar
Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review
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 Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9995916/
https://www.ncbi.nlm.nih.gov/pubmed/36910492
http://dx.doi.org/10.3389/fmed.2023.1036939
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