Characteristics of patients and type and response to treatment
| Characteristic . | Value . |
|---|---|
| Number of patients | 63 |
| Median age, y (range) | 42 (18-76) |
| Disease severity at diagnosis (SAA:NSAA), n | 31:32 |
| Median disease duration, mo (range) | 41 (0.4-300) |
| Sex (M:F), n | 28:35 |
| PNH clone at time of study, n (%) | 24 (38) |
| Size of PNH clone, median % (range) | |
| Red cells | 0.9 (0-27.7) |
| Granulocytes | 2.9 (0-97.0) |
| Monocytes | 5.7 (0-54.4) |
| Etiology | |
| Idiopathic | 59 |
| Pregnancy | 3 |
| Thymoma | 1 |
| Treatment and response | |
| ATG + CSA (1 course) | 23 |
| Responders | 14 (3 CR, 11 PR) |
| ATG + CSA (2 courses) | 5 |
| Responders | 2 (1 CR, 1 PR) |
| ATG + CSA (3 or 4 courses) | 3 |
| Responders | 2 (2 PR) |
| ATG alone | 3 |
| Responders | 2 (1 CR, 1 PR) |
| ATG + oxymetholone (1 or 2 courses) | 3 |
| Responders | 3 (1 CR, 2 PR) |
| ATG + CSA + oxymetholone | 1 |
| Responders | 1 (1 PR) |
| CSA alone | 9 |
| Responders | 3 (3 PR) |
| Characteristic . | Value . |
|---|---|
| Number of patients | 63 |
| Median age, y (range) | 42 (18-76) |
| Disease severity at diagnosis (SAA:NSAA), n | 31:32 |
| Median disease duration, mo (range) | 41 (0.4-300) |
| Sex (M:F), n | 28:35 |
| PNH clone at time of study, n (%) | 24 (38) |
| Size of PNH clone, median % (range) | |
| Red cells | 0.9 (0-27.7) |
| Granulocytes | 2.9 (0-97.0) |
| Monocytes | 5.7 (0-54.4) |
| Etiology | |
| Idiopathic | 59 |
| Pregnancy | 3 |
| Thymoma | 1 |
| Treatment and response | |
| ATG + CSA (1 course) | 23 |
| Responders | 14 (3 CR, 11 PR) |
| ATG + CSA (2 courses) | 5 |
| Responders | 2 (1 CR, 1 PR) |
| ATG + CSA (3 or 4 courses) | 3 |
| Responders | 2 (2 PR) |
| ATG alone | 3 |
| Responders | 2 (1 CR, 1 PR) |
| ATG + oxymetholone (1 or 2 courses) | 3 |
| Responders | 3 (1 CR, 2 PR) |
| ATG + CSA + oxymetholone | 1 |
| Responders | 1 (1 PR) |
| CSA alone | 9 |
| Responders | 3 (3 PR) |
SAA indicates severe aplastic anemia; NSAA, nonsevere aplastic anemia; M, male; F, female; PNH, paroxysmal nocturnal hemoglobinuria; CSA, cyclosporin A; CR, complete response; and PR, partial response.