A randomized study comparing ciclosporin A and antithymocyte globulin for treatment of severe aplastic anemia

This report summarizes results of the treatment of SAA with ciclosporin A (CSA) or antithymocyte globulin (ATG) in 82 patients (pts) randomized since January 1986 from 28 centers.

at diagnosis, pts with established international criteria of SAA and without an HLA identical sib donor were randomized between 2 groups: CyA 4 to 6 mg/kg/d orally for 6 months, or H. ATG 15 mg/kg IV for 5 days associated with Methyl-Prednisolone 5 mg/kg for 5 days then gradually tapered off until day 60. At 3 months follow up, pts with a complete response (CR) or a partial response (PR) received no further therapy, pts with a failure (F) or minimal response (MR) were switched in the other group. All patients were followed regularly for at least 1 year.

at 3 months, all pts with CyA were alive (36 pts) and only 26/35 in the ATG group because of 9 early deaths due to severe infection. In the CyA group, 5 were in CR or PR and 31 in MR or F. Subsequently, 5 received a mismatched BMT and died, 8 remained on CyA, 20 had ATG with, at 1 year, 3 F and 11 PR + CR, 5 pts are too early to evaluate. In the ATG group, 26 pts were alive at 3 months, 6 had a CR or PR, 20 failed. Two pts received a mismatched BMT and died. Fifteen received CyA with, at 1 year, 1 failure and 7 responses (7 pts are too early to evaluate at 1 year).(ABSTRACT TRUNCATED AT 250 WORDS)