Etiocholanedione (ED) is under investigation for its usage in the treatment of aplastic anemia. The specific aims, in 3 steps, of this study are 1) to determine the therapeutic dose and pharmacokinetics of ED, 2) to evaluate the 14-day tolerance of ED, and 3) to evaluate the efficacy and adverse-reaction profile of ED over a 91-day period. The long-term goal is to provide aplastic anemia patients with an oral therapy that cures or ameliorates this disorder. The Step 1, single dose escalation study was finished. Pharamacokinetics at doses of 400 mg po, 800 mg po & 1200 mg po revealed a peak of the blood levels at 2-4 hrs post dose. An increase in the dose was associated with an increase in the peak levels. In addition a secondary peak of about 5 ng/ml was noted at 36 hrs post-dose. Step 2 was begun with a 1200 mg po qd dose to achieve a constant blood level of ED at about 6-8 ng/ml. However, ED induced liver enzymes as evidenced by the shorter circulation time for the drug of 6-8 hrs on Day 8 & Day 14 compared to about 10-12 hrs on Day 1. Dosing was then changed to 1200 mg po bid. Two patients in Step 2 received ED at a dose of 1200 mg po bid for 14 days. Blood for ED level measurements were sent for assay to SuperGen, Inc. The dosage to be used in the subsequent Phase II (Step 3) study will depend on the results of the blood levels obtained in patients studied in Step 2. The drug has been tolerated extremely well. No difficulty has been encountered in recruitment of minorities or females in the study.