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.
Study design: 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.
Results: 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)