Blood and Marrow Transplantation for Sickle Cell Disease: is Less More?

Overview

Journal Blood Rev

Publisher Elsevier

Specialty Hematology

Date 2014 Sep 14

PMID 25217413

Citations 9

Authors

Javier Bolanos-Meade

Robert A Brodsky

Affiliations

Soon will be listed here.

Abstract

Blood and marrow transplantation is a curative therapy for patients with sickle cell disease yet this option is seldom used. Clinical studies have shown however that children transplanted for this condition can achieve excellent results. In children with sickle cell disease transplanted following conditioning with busulfan, cyclophosphamide, and anti-thymocyte globulin, cure rates in excess of 80% can be obtained when an HLA-matched sibling is used as the donor. However, the large majority of patients with sickle cell disease will not have such a donor, or will not be able to tolerate high dose conditioning regimens. Therefore novel approaches such as non-myeloablative regimes, and alternative donors such as haploidentical, unrelated, or cord blood grafts are currently being explored in clinical trials. Recent reports on non-myeloablative conditioning (HLA-matched or haploidentical donors) highlight the safety and efficacy of these approaches with low mortality and high efficacy suggesting that in the near future non-myeloablation could be the preferred type of conditioning and donor availability will not be a barrier anymore to proceed to transplant. This review will focus on the results obtained when bone marrow transplants are used to treat sickle cell disease and will discuss the results obtained with these novel approaches.

Citing Articles

Burden experienced by informal caregivers of children with sickle cell disease (SCD): a qualitative exploratory study at Tamale Teaching Hospital, Ghana.

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Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet.

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Hematopoietic stem cell transplantation reverses white matter injury measured by diffusion-tensor imaging (DTI) in sickle cell disease patients.

de Mello Costa T, Chiari-Correia R, Salmon C, Darrigo-Junior L, Grecco C, Pieroni F Bone Marrow Transplant. 2021; 56(11):2705-2713.

PMID: 34234298 DOI: 10.1038/s41409-021-01365-z.

Assessment of Patient and Caregiver Attitudes and Approaches to Decision-Making Regarding Bone Marrow Transplant for Sickle Cell Disease: A Qualitative Study.

Bakshi N, Katoch D, Sinha C, Ross D, Quarmyne M, Loewenstein G JAMA Netw Open. 2020; 3(5):e206742.

PMID: 32469414 PMC: 7260617. DOI: 10.1001/jamanetworkopen.2020.6742.

Pathophysiology and recent therapeutic insights of sickle cell disease.

Shah F, Dwivedi M Ann Hematol. 2020; 99(5):925-935.

PMID: 32157419 DOI: 10.1007/s00277-020-03977-9.

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