Allogeneic Haematopoietic Cell Transplantation in Aplastic Anaemia: A Dataset of 240 Cases

Objective: To analyse factors associated with survival, rejection and graft versus host disease in aplastic anaemia patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT) from related donors.

Patients & Methods: The study included 240 consecutive aplastic anaemia patients undergoing haematopoietic stem cell transplantation from HLA matched sibling donors at this centre from July 2001 to April 2016. Potential risk factors affecting overall survival, rejection, disease free survival and graft versus host disease were analysed.

Results: One hundred and eighty eight male and fifty two female AA patients were included in the study. Median age was 18 years (3-55 years), conditioning regimens used were cyclosphosphamide (Cy) plus antilymphocyte globulin (ALG) or antithymocyte globulin (ATG) (n=105), Fludarabine (FLU)+CY+ATG (n=128), and Campath 1-H +CY in 6 cases respectively. While haplo-identical transplantation was carried out in one patient. Methotrexate was added to GVHD prophylaxis in 101 patients while the rest received only CSA plus prednisolone. At a median follow up of 1485 days OS & DFS were 76% & 70% respectively. Factors associated with better OS were male sex, Cy plus ALG/ATG conditioning and use of bone marrow as stem cell source. In a comparison between ATG and ALG-based regimens (n=205), ATG-containing regimens was found superior to the one those with ALG, in both OS (78.7% vs69.4%) and DFS (75% vs 55.6%) [p=0.018].

Conclusion: Cy plus ALG/ATG conditioning regimen, bone marrow as stem cell source and GVHD prophylaxis with CSA minus methotrexate were associated with better survival in AA.