How I manage aplastic anaemia in children |
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Authors: | Sujith Samarasinghe David K. H. Webb |
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Affiliation: | 1. Paediatric Haematopoietic Stem Cell Transplant Unit, Department of Adolescent and Paediatric Haematology and Oncology, Great North Children's Hospital, Royal Victoria Infirmary, , Newcastle Upon Tyne, UK;2. Department of Haematology, Great Ormond Street Hospital, , London, UK |
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Abstract: | Aplastic anaemia (AA) is a rare heterogeneous condition in children. 15–20% 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 30–40%. 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. |
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Keywords: | paediatric aplastic anaemia inherited bone marrow failure syndrome transplantation in aplastic anaemia anti‐thymocyte globulin |
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