Objective: To study the efficacy and safety of human leukocyte antigen (HLA) mismatched hematopoietic stem cell transplantation (HSCT) on severe aplastic anemia (SAA).
Methods: From January 2006 to May 2010, 17 patients received mismatched HSCT. HLA antigens were 3-loci-mismatched in 9 patients, 2-loci-mismatched in 8. All patients received recombinant human granulocyte colony-stimulating factor (rhG-CSF) primed bone marrow cells plus peripheral blood stem cells after modified busulfan/cyclophosphamide + antithymocyte immunoglobulin (BU/CY + ATG) conditioning regimen.
Results: All patients achieved full donor type engraftment. Grade III-IV graft versus host disease (GVHD) occurred in 3 patients and extensive chronic GVHD in 1. With a median following-up time of 285 (60 - 1670) d, 11 patients were alive, 9 of them had normal blood counts and the other 2 were blood transfusion independent. Six patients died of transplant-related complications.
Conclusion: Mismatched HSCT is a feasible and safe option for SAA patients without sibling identical donors.