Objective: Aplastic anemia is characterized by diminished or absent hematopoietic precursors in the bone marrow, most often due to injury to the pluripotent stem cell. In Pakistan, Aplastic Anemia is not uncommon and allogeneic hematopoietic stem cell transplant remains the only curative option in these patients. We aim to determine the transplant outcome of combined, G-CSF primed blood and bone marrow grafts in adult and pediatric patients with aplastic anemia.
Methodology: We retrospectively collected data of all transplant procedures performed from 2004–2019 at Aga Khan University Karachi, Pakistan. Variables analyzed included type of transplants, age, gender, type of stem cells used, conditioning regimens and overall survival for patients undergoing transplant in aplastic anemia.
Results: A total of 351 transplants were performed during the study period. Out of these, 239 were allogeneic transplants whereas 112 were autologous procedures. There were 254 males and 97 females. The main indications for allogeneic stem cell transplant were aplastic anemia (70), acute leukemia (58) and beta thalassemia major(40). Out of 70 patients with aplastic anemia, 52 were males and 18 were females. 38.6% percent of patients were from pediatric age group. The median age±SD was 17.5±9.4 years (range: 2–43 years). Cyclophosphamide/ATG was used as a conditioning regimen in 67 patients, while ATG/cyclophosphamide/fludarabine was used in 2. Haploidentical transplant was done in 1 patient. Twenty seven percent of patients underwent sex-mismatched procedures. In 52 patients, a combination of GCSF primed blood and bone marrow stem cells were used. The mean CD34 count was 5.2×10/kg. GVHD prophylaxis was done with cyclosporine and methotrexate. All patients received standard infection prophylaxis. Engraftment was achieved in 75% 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 causes of mortality included gram-negative sepsis (5), graft versus host disease (4), graft failure (4), disseminated fungal infection (2), intracranial bleed (2), bleeding diathesis (2) and transplant associated microangiopathy (1).
Conclusion: Combination of blood and bone marrow stem cells results in early engraftment with decreased frequency of GVHD in aplastic anemia. The overall survival was comparable to international literature.
