Hematopoietic Stem Cell Transplantation for Acquired Aplastic Anemia

Overview

Journal Curr Opin Hematol

Specialty Hematology

Date 2016 Sep 9

PMID 27607445

Citations 10

Authors

George E Georges

Rainer Storb

Affiliations

Soon will be listed here.

Abstract

Purpose Of Review: There has been a steady improvement in outcomes with allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA), because of progress in optimization of the conditioning regimens, donor hematopoietic cell source, and supportive care. Here, we review recently published data that highlight the improvements and current issues in the treatment of SAA.

Recent Findings: Approximately one-third of aplastic anemia patients treated with immune suppressive therapy (IST) have acquired mutations in myeloid cancer candidate genes. Because of the greater probability for eventual failure of IST, human leukocyte antigen (HLA)-matched sibling donor BMT is the first-line of treatment for SAA. HLA-matched unrelated donor (URD) BMT is generally recommended for patients who have failed IST. However, in younger patients for whom a 10/10-HLA-allele matched URD can be rapidly identified, there is a strong rationale to proceed with URD BMT as first-line therapy. HLA-haploidentical BMT using posttransplant cyclophosphamide conditioning regimens is now a reasonable second-line treatment for patients who failed IST.

Summary: Improved outcomes have led to an increased first-line role of BMT for treatment of SAA. The optimal cell source from an HLA-matched donor is bone marrow. Additional studies are needed to determine the optimal conditioning regimen for HLA-haploidentical donors.

Citing Articles

The Protective Effect of Chronic Intermittent Hypobaric Hypoxia on Preventing the Destruction of CD34 Haematopoietic Stem Cells in Aplastic Anaemia by Modulating the Th1/Th2 Balance.

Guo H, Zhao Y, Mu R, Zhang G, Chen S, Cao X Stem Cell Rev Rep. 2023; 20(1):301-312.

PMID: 37831395 DOI: 10.1007/s12015-023-10631-0.

Pre-Transplant Platelet Refractoriness and Alternative Donors Are Associated With Cytomegalovirus Retinitis in Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia.

Zhang Y, Liang Y, Zhang X, Wang S, Cao J, Gao Z Front Cell Infect Microbiol. 2022; 12:870296.

PMID: 35372094 PMC: 8964998. DOI: 10.3389/fcimb.2022.870296.

Type 2 innate lymphoid cells from Id1 transgenic mice alleviate skin manifestations of graft-versus-host disease.

Srinivasan A, Bajana S, Pankow A, Yuen C, Shah R, Sun X BMC Immunol. 2021; 22(1):46.

PMID: 34256699 PMC: 8278660. DOI: 10.1186/s12865-021-00432-w.

Outcomes of haploidentical bone marrow transplantation in patients with severe aplastic anemia-II that progressed from non-severe acquired aplastic anemia.

Liu H, Zheng X, Zhang C, Xie J, Gao B, Shao J Front Med. 2021; 15(5):718-727.

PMID: 34170455 DOI: 10.1007/s11684-020-0807-4.

Current Use of Total Body Irradiation in Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation.

Jung J, Lee H, Suh Y, Eom H, Lee E J Korean Med Sci. 2021; 36(8):e55.

PMID: 33650334 PMC: 7921367. DOI: 10.3346/jkms.2021.36.e55.

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