RESUMO
INTRODUCTION: Aplastic anemia (AA) is characterized by diminished hematopoietic precursors in the bone marrow, most often due to injury to the pluripotent stem cell. In Pakistan, AA is not uncommon, and allogeneic hematopoietic stem cell transplant remains the only curative option for these patients. OBJECTIVE: The objective of this study was to determine the transplant outcome of combined granulocyte colony-stimulating factor (G-CSF) primed blood and bone marrow grafts in adult and pediatric patients with AA. METHODS: We retrospectively collected the data of all transplant procedures performed from 2004 to 2019 at Aga Khan University in Karachi, Pakistan. Variables analyzed included age, sex, type of stem cells used, conditioning regimens, and overall survival for patients undergoing transplant in AA. RESULTS: A total of 351 transplants were performed during the study period. Out of these, 239 were allogeneic transplants, whereas 112 were autologous procedures. We performed 70 transplants for AA during the study period, of which 52 were male patients and 18 were female patients. The median age ± standard deviation (SD) was 17.5 ± 9.4 years (range, 2-43 years). Cyclophosphamide/antithymocyte globulin (ATG) was used as a conditioning regimen in 65 patients, while ATG/cyclophosphamide/fludarabine was used in 5 patients. In 60 patients, a combination of G-CSF primed blood and bone marrow stem cells were used. The mean CD34 count was 5.2 × 106/kg. Graft-vs-host disease (GVHD) prophylaxis was done with cyclosporine and methotrexate. All patients received standard infection prophylaxis. Engraftment was achieved in 86% of patients. The median day of myeloid engraftment was 15 (range, 10-22 days). Chronic GVHD was present in 3 patients while 4 had acute GVHD. The overall survival was 71.2% (median duration of 80 months). The main cause of mortality was gram-negative sepsis. CONCLUSION: A combination of blood and bone marrow stem cells results in early engraftment with decreased frequency of GVHD in AA. The overall survival was comparable to international literature.