Allogeneic hematopoietic stem cell transplantation as the first-line treatment option in a patient with severe aplastic anemia without a matched related donor: A case report

The outcomes of matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) in patients with severe aplastic anemia (SAA) remain controversial. The clinical outcome in patients that undergo transplantation following failed IST is typically poorer when compared with patients that initially underwent transplantation. Clinical treatment algorithms have been proposed to determine the management of such patients, and account for individual conditions, personal preferences and prognostic risk factors. The present study reports the promising outcome of a 22-year-old patient exhibiting SAA. The patient underwent peripheral blood stem cell transplantation (PBSCT) from an MUD using a fludarabine-based conditioning regimen and low-dose total body irradiation as an alternative method to first-line IST. The patient achieved rapid bone marrow reconstitution and has been in complete remission for 32 months. The aim of the fludarabine-based conditioning regimen with PBSCT was to improve the patient's therapeutic outcome and provide a convenient treatment strategy. Furthermore, this regimen extends the application of HSCT to patients who are older or those that are without a matched related donor.