Treatment of severe aplastic anemia with antilymphocyte globulin and androgens: a report on 33 patients.

Thirty-three patients with severe aplastic anemia were treated with antilymphocyte globulin (ALG, Mérieux) and androgens (with or without corticosteroids) between 1981 and 1989; 24 patients (72.7%) were responders after one course of ALG, eight were nonresponders, and only one patient had an early death. Eighteen of the 24 responses occurred within 2 months of ALG treatment. Of note is the good response rate we obtained for very severe aplastic anemia (four responders of five evaluable patients). With a median follow-up of 36 months (range 1-97), a survival rate of 77.6% +/- 1.2% was obtained at 30 months. No predictive factor of survival could be identified except response to treatment (p = 0.0001). The duration of the disease before treatment was inversely related to survival, although this difference did not reach statistical significance (p = 0.06). Four initial responders relapsed after 14, 24, 38, and 57 months. Three of these patients received a second course of ALG and two responded. In contrast, four of the non-responders received a second course of ALG, with only one response. Toxicity of androgens was mild. No patient developed a PNH clone or myelodysplastic syndrome. Major toxicity of corticosteroids was femoral osteonecrosis in three patients. In our experience, the combination of ALG and androgens in SAA, with or without corticosteroids, was associated with a higher response rate and better survival than in many previously published reports. This could have been due to the intensive supportive care during the initial weeks of treatment. We suggest that it may also result from the addition of androgens to ALG, although this issue may only be resolved in a randomized study.