Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2021 Sep;194(6):954-969.
doi: 10.1111/bjh.17403. Epub 2021 Apr 14.

Acquired severe aplastic anaemia: how medical therapy evolved in the 20th and 21st centuries

Phillip Scheinberg  1
Affiliations
Review

Acquired severe aplastic anaemia: how medical therapy evolved in the 20th and 21st centuries

Phillip Scheinberg. Br J Haematol. 2021 Sep.

Abstract

The progress in aplastic anaemia (AA) management is one of success. Once an obscure entity resulting in death in most affected can now be successfully treated with either haematopoietic stem cell transplantation (HSCT) or immunosuppressive therapy (IST). The mechanisms that underly the diminution of haematopoietic stem cells (HSCs) are now better elucidated, and include genetics and immunological alterations. Advances in supportive care with better antimicrobials, safer blood products and iron chelation have greatly impacted AA outcomes. Working somewhat 'mysteriously', anti-thymocyte globulin (ATG) forms the base for both HSCT and IST protocols. Efforts to augment immunosuppression potency have not, unfortunately, led to better outcomes. Stimulating HSCs, an often-sought approach, has not been effective historically. The thrombopoietin receptor agonists (Tpo-RA) have been effective in stimulating early HSCs in AA despite the high endogenous Tpo levels. Dosing, timing and best combinations with Tpo-RAs are being defined to improve HSCs expansion in AA with minimal added toxicity. The more comprehensive access and advances in HSCT and IST protocols are likely to benefit AA patients worldwide. The focus of this review will be on the medical treatment advances in AA.

Keywords: anti-thymocyte globulin; aplastic anaemia; bone marrow failure; cyclosporin; eltrombopag; immunosuppressive therapy.

PubMed Disclaimer

References

    1. Young NS. Aplastic anaemia. Lancet. 1995;346(8969):228-32.
    1. Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica. 2008;93(4):489-92.
    1. Issaragrisil S, Kaufman DW, Anderson T, Chansung K, Leaverton PE, Shapiro S, et al. The epidemiology of aplastic anemia in Thailand. Blood. 2006;107(4):1299-307.
    1. Scheinberg P, Chen J. Aplastic anemia: what have we learned from animal models and from the clinic. Semin Hematol. 2013;50(2):156-64.
    1. Sieff CA. Introduction to acquired and inherited bone marrow failure. Hematol Oncol Clin North Am. 2018;32(4):569-80.

MeSH terms