Abstract
Severe aplastic anaemia is a rare but life-threatening disorder for which allogeneic haemopoietic stem-cell transplant (HSCT) is a potential cure. In patients younger than 35 years and up to 50 years of age without substantial comorbidities with HLA-matched sibling donor, upfront HSCT is the recommended treatment. Healthy patients without a HLA-matched sibling donor are offered HSCT as a second-line treatment after failure of immunosuppressive therapy. 1 Marsh JC Ball SE Cavenagh J et al. Guidelines for the diagnosis and management of aplastic anaemia. Br J Haematol. 2009; 147: 43-70 Crossref PubMed Scopus (419) Google Scholar Although 60–70% of patients respond to immunosuppressive therapy, blood counts return to normal only in a third of patients, and another third need long-term cyclosporin therapy and the risk of relapse of aplastic anaemia and clonal evolution to myelodysplastic syndromes occurs in a further third of patients. HSCT, however, offers an opportunity for longlasting haematological response and minimises the risk of relapse and reduces the likelihood of clonal evolution. Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1–2 dose de-escalation studyCyclophosphamide at 50 mg/kg and 100 mg/kg with TBI 2 Gy, fludarabine, and anti-thymocyte globulin results in effective conditioning and few early deaths after unrelated donor transplantation for severe aplastic anaemia. These doses of cyclophosphamide provide a framework for further regimen optimisation strategies. Full-Text PDF
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