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For children with idiopathic AA and a human leucocyte antigen (HLA)\u2010matched sibling donor (MSD), allogeneic haematopoietic stem\u2010cell transplantation (AHSCT) is the primary therapy of choice, offering long\u2010term disease\u2010free survival of 90%, although graft\u2010<jats:italic>versus<\/jats:italic>\u2010host disease remains a cause of long\u2010term morbidity. A greater treatment challenge remains for those children without a MSD. Combination immunosuppressive therapy (IST) is associated with response rates of 70% or more. However, relapse and clonal evolution with transformation to myelodysplasia or acute myeloid leukaemia remain significant problems after IST and long\u2010term event\u2010free survival rates are less impressive. For children who do not have a sustained response to IST, alternate donor AHSCT should be considered. New HLA typing technologies, novel stem cell sources, reduced\u2010intensity conditioning and graft engineering have reduced toxicity and improved the outcome after alternate donor AHSCT. Emerging therapies that capitalise on recent advances in our understanding of the pathophysiology of idiopathic AA and the immunobiology of AHSCT and IST may further improve the long\u2010term outcome of this disease.<\/jats:p>","DOI":"10.1111\/j.1365-2141.2006.06461.x","type":"journal-article","created":{"date-parts":[[2007,1,10]],"date-time":"2007-01-10T09:16:19Z","timestamp":1168420579000},"page":"549-564","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":79,"title":["An update on the management of severe idiopathic aplastic anaemia in children"],"prefix":"10.1111","volume":"136","author":[{"given":"Jeffrey K.","family":"Davies","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eva 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