Abstract
Objective: The aim of this study was to reveal the efficacy and safety of rabbit antithymocyte globulin (rATG) in patients with aplastic anemia (AA).
Patients: This study included 17 consecutive patients in Tenri Hospital from May 2009 through November 2013 who were diagnosed with AA and first treated with rATG in combination with or without cyclosporine (CS), including one who had been previously treated with CS alone.
Results: Ages ranged from 8 to 81, with a median of 68. The diseases included 5 non-severe (NSAA), 7 severe (SAA), and 5 very severe AA (VSAA). Paroxysmal nocturnal hemoglobinuria (PNH)-type cells were present in 11. Rabbit ATG was administered at a dose of 2.5 or 3.75 mg/kg/day. Cyclosporine was concurrently given to all but one. At 6 months, 10 (71%) of 14 evaluable patients achieved a partial response (PR) and 2 finally fulfilled the criteria for a complete response. Nine (75%) of the 12 NSAA/SAA patients achieved a PR or more, while the hematological response in 3 of 4 VSAA patients at >6 months did not reach the level of PR. The percentage of PNH-type cells showed no significant change after treatment. Three patients aged >75 years old died, including 2 with unexpected cardiopulmonary arrest. With a median follow-up of 504 days, the overall survival rate at 2 years was 79%.
Conclusion: This study indicates that rATG is the treatment of choice for NSAA/SAA patients who are ineligible for allogeneic transplantation. However, we should be cautious when using rATG for aged patients.
