Abstract
Aplastic anaemia (AA) is a rare bone marrow failure syndrome treated either by immunosuppressive therapy or allogeneic stem cell transplantation (SCT). At present, no randomised clinical trials evaluating both treatment options, and in particular SCT from unrelated donors, are available. We here report the clinical course and outcome of allogeneic SCT for 20 consecutive adult patients with AA. Newly diagnosed and untreated patients (n = 8) or patients pre-treated by immunosuppressive therapy (n = 12) were transplanted either from human-leukocyte-antigen (HLA) identical family donors (n = 13) or matched (n = 6) and mismatched (n = 1) unrelated donors, respectively. Conditioning varied depending on donor type and included cyclophosphamide with or without anti-thymocyte globulin (ATG) and fludarabine-cyclophosphamide-ATG with or without low-dose total body irradiation. With a median follow-up of more than 40 months, all patients have had favourable outcomes with stable haematopoietic engraftment and high performance scores. Six patients developed acute (five I degrees -II degrees ; one >II degrees ) and four limited chronic graft-versus-host disease. In this group of AA patients, allogeneic SCT has proven very successful, independent of donor type and pre-treatment. Studies with greater cohorts of patients are warranted to better determine indication and timing of SCT especially from unrelated donors in AA.
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