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. 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 language | English |
|---|---|
| Pages (from-to) | 152-159 |
| Number of pages | 8 |
| Journal | Internal Medicine Journal |
| Volume | 49 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - 4 Feb 2019 |
Keywords
- anaemia
- anti-thymocyte globulin
- aplastic
- blood transfusion
- bone marrow transplantation
- pancytopenia
Cite this
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Revisiting acquired aplastic anaemia: current concepts in diagnosis and management. / Clucas, Danielle B.; Fox, Lucy C.; Wood, Erica M.; Hong, Frank S.; Gibson, John; Bajel, Ashish; Szer, Jeff; Blombery, Piers; McQuilten, Zoe K.; Hiwase, Devendra; Firkin, Frank; Cole-Sinclair, Merrole F.; on behalf of the Australian Aplastic Anaemia Registry Steering Committee.
In: Internal Medicine Journal, Vol. 49, No. 2, 04.02.2019, p. 152-159.Research output: Contribution to journal › Review Article › Research › peer-review
TY - JOUR
T1 - Revisiting acquired aplastic anaemia: current concepts in diagnosis and management
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.
AU - on behalf of the Australian Aplastic Anaemia Registry Steering Committee
PY - 2019/2/4
Y1 - 2019/2/4
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. 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.
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. 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.
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 - Internal Medicine Journal
JF - Internal Medicine Journal
SN - 1444-0903
IS - 2
ER -