Abstract
Thalassemia and sickle cell disease (SCD) are the most common monogenic diseases in
the world and represent a growing global health burden. Management is limited by a
paucity of disease-modifying therapies; however, allogeneic hematopoietic stem cell
transplantation (HSCT) and autologous HSCT after genetic modification offer patients
a curative option. Allogeneic HSCT is limited by donor selection, morbidity and mortality
from transplant conditioning, graft-versus-host disease and graft rejection, whereas
significant concerns regarding long-term safety, efficacy and cost limit the broad
applicability of gene therapy. Here the authors review current outcomes in allogeneic
and autologous HSCT for transfusion-dependent thalassemia and SCD and provide our
perspective on issues surrounding accessibility and costs as barriers to offering
curative therapy to patients with hereditary hemoglobinopathies.
Key Words
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Article info
Publication history
Published online: December 06, 2021
Accepted:
September 4,
2021
Received:
June 10,
2021
Identification
Copyright
© 2021 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.
