Revisiting acquired aplastic anaemia: current concepts in diagnosis and management

Danielle B. Clucas, Lucy C. Fox, Erica M. Wood, Frank S. Hong, John Gibson, Ashish Bajel, Jeff Szer, Piers Blombery, Zoe K. McQuilten, Devendra Hiwase, Frank Firkin, Merrole F. Cole-Sinclair, on behalf of the Australian Aplastic Anaemia Registry Steering Committee

Research output: Contribution to journalReview ArticleResearchpeer-review

11 Citations (Scopus)


Acquired aplastic anaemia is a rare, serious, immunologically mediated bone marrow failure syndrome, characterised by marrow hypoplasia of varying severity and significant pancytopenia. Careful attention and investigation, including molecular testing, is required to confirm the diagnosis and exclude other mimicking conditions, such as inherited bone marrow failure syndromes. In a proportion of patients, the disease evolves to myelodysplasia or acute myeloid leukaemia and in some there is an association with paroxysmal nocturnal haemoglobinuria. The disease has a major impact on patient quality of life. Haemopoietic stem/progenitor cell transplantation for eligible patients with an available donor is the only current curative therapy. Other patients may receive immunosuppression, most commonly with anti-thymocyte globulin and cyclosporin. An initial response to immunosuppression is often encouraging, but relapse is common. Supportive care, including management of transfusion requirements and infections, is central to management. Promising new diagnostic tools and emerging therapies will likely transform approaches to this important, chronic and life-threatening condition.

Original languageEnglish
Pages (from-to)152-159
Number of pages8
JournalInternal Medicine Journal
Issue number2
Publication statusPublished - 4 Feb 2019


  • anaemia
  • anti-thymocyte globulin
  • aplastic
  • blood transfusion
  • bone marrow transplantation
  • pancytopenia

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