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How I manage aplastic anaemia in children

Lookup NU author(s): Dr Sujith Samarasinghe


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Aplastic anaemia (AA) is a rare heterogeneous condition in children. 1520% of cases are constitutional and correct diagnosis of these inherited causes of AA is important for appropriate management. For idiopathic severe aplastic anaemia, a matched sibling donor (MSD) haematopoietic stem cell transplant (HSCT) is the treatment of choice. If a MSD is not available, the options include immunosuppressive therapy (IST) or unrelated donor HSCT. IST with horse anti-thymocyte globulin (ATG) is superior to rabbit ATG and has good long-term results. In contrast, IST with rabbit ATG has an overall response of only 3040%. Due to improvements in outcome over the last two decades in matched unrelated donor (MUD) HSCT, results are now similar to that of MSD HSCT. The decision to proceed with IST with ATG or MUD HSCT will depend on the likelihood of finding a MUD and the differing risks and benefits that each therapy provides.

Publication metadata

Author(s): Samarasinghe S, Webb DKH

Publication type: Review

Publication status: Published

Journal: British Journal of Haematology

Year: 2012

Volume: 157

Issue: 1

Pages: 26-40

Print publication date: 20/02/2012

ISSN (print): 0007-1048

ISSN (electronic): 1365-2141



DOI: 10.1111/j.1365-2141.2012.09058.x