Revisiting acquired aplastic anaemia: Current concepts in diagnosis and management

on behalf of the Australian Aplastic Anaemia Registry Steering Committee, 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

Research output: Contribution to journalReview article

Abstract

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. Haematopoietic 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. This article is protected by copyright. All rights reserved.

LanguageEnglish
Pages152-159
Number of pages8
JournalInternal Medicine Journal
Volume49
Issue number2
Early online date15 Oct 2018
DOIs
Publication statusPublished - 1 Feb 2019

Keywords

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

ASJC Scopus subject areas

  • Internal Medicine

Cite this

on behalf of the Australian Aplastic Anaemia Registry Steering Committee (2019). Revisiting acquired aplastic anaemia: Current concepts in diagnosis and management. Internal Medicine Journal, 49(2), 152-159. https://doi.org/10.1111/imj.14140
on behalf of the Australian Aplastic Anaemia Registry Steering Committee. / Revisiting acquired aplastic anaemia : Current concepts in diagnosis and management. In: Internal Medicine Journal. 2019 ; Vol. 49, No. 2. pp. 152-159.
@article{a05483bb0ec84aa280c618e695aa9f62,
title = "Revisiting acquired aplastic anaemia: Current concepts in diagnosis and management",
abstract = "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. Haematopoietic 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. This article is protected by copyright. All rights reserved.",
keywords = "anaemia, anti-thymocyte globulin, aplastic, blood transfusion, bone marrow transplantation, pancytopenia",
author = "{on behalf of the Australian Aplastic Anaemia Registry Steering Committee} and Clucas, {Danielle B.} and Fox, {Lucy C.} and Wood, {Erica M.} and Hong, {Frank S.} and John Gibson and Ashish Bajel and Jeff Szer and Piers Blombery and McQuilten, {Zoe K.} and Devendra Hiwase and Frank Firkin and Cole-Sinclair, {Merrole F.}",
year = "2019",
month = "2",
day = "1",
doi = "10.1111/imj.14140",
language = "English",
volume = "49",
pages = "152--159",
journal = "Australian and New Zealand Journal of Medicine",
issn = "1444-0903",
publisher = "Wiley-Blackwell",
number = "2",

}

on behalf of the Australian Aplastic Anaemia Registry Steering Committee 2019, 'Revisiting acquired aplastic anaemia: Current concepts in diagnosis and management', Internal Medicine Journal, vol. 49, no. 2, pp. 152-159. https://doi.org/10.1111/imj.14140

Revisiting acquired aplastic anaemia : Current concepts in diagnosis and management. / on behalf of the Australian Aplastic Anaemia Registry Steering Committee.

In: Internal Medicine Journal, Vol. 49, No. 2, 01.02.2019, p. 152-159.

Research output: Contribution to journalReview article

TY - JOUR

T1 - Revisiting acquired aplastic anaemia

T2 - Australian and New Zealand Journal of Medicine

AU - on behalf of the Australian Aplastic Anaemia Registry Steering Committee

AU - Clucas, Danielle B.

AU - Fox, Lucy C.

AU - Wood, Erica M.

AU - Hong, Frank S.

AU - Gibson, John

AU - Bajel, Ashish

AU - Szer, Jeff

AU - Blombery, Piers

AU - McQuilten, Zoe K.

AU - Hiwase, Devendra

AU - Firkin, Frank

AU - Cole-Sinclair, Merrole F.

PY - 2019/2/1

Y1 - 2019/2/1

N2 - 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. Haematopoietic 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. This article is protected by copyright. All rights reserved.

AB - 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. Haematopoietic 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. This article is protected by copyright. All rights reserved.

KW - anaemia

KW - anti-thymocyte globulin

KW - aplastic

KW - blood transfusion

KW - bone marrow transplantation

KW - pancytopenia

UR - http://www.scopus.com/inward/record.url?scp=85061392191&partnerID=8YFLogxK

U2 - 10.1111/imj.14140

DO - 10.1111/imj.14140

M3 - Review article

VL - 49

SP - 152

EP - 159

JO - Australian and New Zealand Journal of Medicine

JF - Australian and New Zealand Journal of Medicine

SN - 1444-0903

IS - 2

ER -

on behalf of the Australian Aplastic Anaemia Registry Steering Committee. Revisiting acquired aplastic anaemia: Current concepts in diagnosis and management. Internal Medicine Journal. 2019 Feb 1;49(2):152-159. https://doi.org/10.1111/imj.14140