Curative therapy for hemoglobinopathies: An International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation Review

  

Authors:	Leonard, A.; Bertaina, A.; Bonfim, C.; Cohen, S.; Prockop, S.; Purtill, D.; Russell, A.; Boelens, J. J.; Wynn, R.; Ruggeri, A.; Abraham, A.
Review Title:	Curative therapy for hemoglobinopathies: An International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation
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. © 2021 International Society for Cell & Gene Therapy
Keywords:	gene therapy; thalassemia; sickle cell disease; hematopoietic cell transplant
Journal Title:	Cytotherapy
Volume:	24
Issue:	3
ISSN:	1465-3249
Publisher:	Elsevier Science Ltd.
Date Published:	2022-03-01
Start Page:	249
End Page:	261
Language:	English
DOI:	10.1016/j.jcyt.2021.09.003
PUBMED:	34879990
PROVIDER:	scopus
DOI/URL:	https://www.scopus.com/inward/record.uri?eid=2-s2.0-85120697106&doi=10.1016%2fj.jcyt.2021.09.003&partnerID=40&md5=f0e0a247de2005f7af60a03c9c8b3f65
Notes:	Article -- Export Date: 1 March 2022 -- Source: Scopus

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