Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

Leonard, Alexis; Tisdale, John; Abraham, Allistair

doi:10.1111/bjh.16437

Cited by 45Hemoglobinopathies and Related DisordersHematopoietic Stem Cell TransplantationVirus-based gene therapy research

Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

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Leonard, Alexis, Tisdale, John, Abraham, Allistair·2020·British Journal of Haematology

DOI 10.1111/bjh.16437

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Abstract

Haematopoietic stem cell transplantation (HSCT) is curative in sickle cell disease (SCD); however, the lack of available matched donors makes this therapy out of reach for the majority of patients with SCD. Alternative donor sources such as haploidentical HSCT expand the donor pool to nearly all patients with SCD, with recent data showing high overall survival, limited toxicities, and effective reduction in acute and chronic graft-versus-host disease (GVHD). Simultaneously, multiple gene therapy strategies are entering clinical trials with preliminary data showing their success, theoretically offering all patients yet another curative strategy without the morbidity and mortality of GVHD. As improvements are made for alternative donors in the allogeneic setting and as data emerge from gene therapy trials, the optimal curative strategy for any individual patient with SCD will be determined by many critical factors including efficacy, transplant morbidity and mortality, safety, patient disease status and preference, cost and applicability. Haploidentical may be the preferred choice now based mostly on availability of data; however, gene therapy is closing the gap and may ultimately prove to be the better option. Progress in both strategies, however, makes cure more attainable for the individual with SCD.

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Gene therapy will have to compete with the improving prospects for haploidentical allogeneic transplantation that could extend the proven effectiveness of allogeneic transplantation.
“Gene therapy will also have to compete with the improving prospects for haploidentical allogeneic transplantation that could extend the proven effectiveness of allogeneic transplantation.128”
From Fetal hemoglobin in sickle cell anemia (2020) · cites this paper as [128]

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Authors: Leonard, A., Tisdale, J., & Abraham, A.
Affiliations: National Institutes of Health (US)
Children's National (US)
Corresponding author: Abraham, Allistair
ORCID: Leonard, Alexis
Tisdale, John
Abraham, Allistair
Venue: British Journal of Haematology
Publisher: Wiley
Published: 2020-02-07
Volume: 189
Issue: 3
Pages: 408-423
ISSN: 0007-1048 (ISSN-L)
1365-2141
DOI: 10.1111/bjh.16437
OpenAlex: W3005407815
Cited by: 45
References: 102
Citations by year: 2026: 5 · 2025: 5 · 2024: 8 · 2023: 9 · 2022: 5 · 2021: 7 · 2020: 6
FWCI: 0.99
Type: journal article
Language: en
Open access: hybrid
doi.org
Licence: public-domain
onlinelibrary.wiley.com/termsAndConditions#vor (vor)
Locations: British Journal of Haematology (publishedVersion, public-domain)
PubMed (publishedVersion)
Funders: American Society of Hematology-Robert Wood Johnson Foundation
Topics: Health Sciences › Medicine › Genetics › Hemoglobinopathies and Related Disorders
Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Keywords: sickle cell disease, haploidentical hematopoietic stem cell transplantation, gene therapy, graft-versus-host disease, curative treatment, transplant morbidity and mortality
MeSH terms: Transplantation, Haploidentical, Anemia, Sickle Cell, Humans, Genetic Therapy, Hematopoietic Stem Cell Transplantation, Transplantation Conditioning

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Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

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