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Revisiting acquired aplastic anaemia: current concepts in diagnosis and management
Author(s) -
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,
ColeSinclair Merrole F.
Publication year - 2019
Publication title -
internal medicine journal
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.596
H-Index - 70
eISSN - 1445-5994
pISSN - 1444-0903
DOI - 10.1111/imj.14140
Subject(s) - medicine , pancytopenia , immunosuppression , bone marrow failure , disease , aplastic anemia , intensive care medicine , bone marrow , stem cell , pediatrics , immunology , haematopoiesis , biology , genetics
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.