Patient characteristics at baseline
| Patient characteristics . | Values . |
|---|---|
| Age, y | |
| Median | 32.5 |
| Range | 10-69 |
| Age, n (%) | |
| <18 y | 2 (6) |
| 18-64 y | 30 (88) |
| ≥65 y | 2 (6) |
| Sex, n (%) | |
| Female | 17 (50) |
| Male | 17 (50) |
| Race/ethnicity, n(%) | |
| Asian | 4 (12) |
| African American | 5 (15) |
| Hispanic | 2 (6) |
| White | 23 (68) |
| Other | 0 (0) |
| Diagnosis, n (%) | |
| MAA | 31 (91) |
| UC-erythroid (including 1 DBA) | 3 (9) |
| No. of previous treatments (%) | |
| 0 | 19 (56) |
| 1 | 9 (26) |
| >2 | 6 (18) |
| Previous treatments, n (%) | |
| Immunosuppressive therapy with ATG, ATG/CSA, or CSA | 7 (21) |
| Androgens | 6 (18) |
| Other* | 11 (32) |
| Time since first diagnosis, median (range), mo | 11 (1-354) |
| Laboratory parameters | |
| Neutrophil count, median (range), ×10−9/L | 1.1 (0.5-5.3) |
| Reticulocyte count, median (range), ×10−9/L | 62.9 (3.1-92.2) |
| Platelet count, median (range), ×109/L | 26.0 (10.0-311.0) |
| Hemoglobin, median (range), g/dL | 8.7 (6.6-15.7) |
| Erythroid response eligible (transfusion-dependent or hemoglobin <8.5 g/dL), n (%) | 23 (68) |
| Platelet response eligible (transfusion-dependent or platelets <30 × 109/L), n (%) | 24 (71) |
| Erythroid and platelet response eligible, n (%) | 13 (38) |
| PNH clone >1%, n (%) | 11 (32.) |
| Patient characteristics . | Values . |
|---|---|
| Age, y | |
| Median | 32.5 |
| Range | 10-69 |
| Age, n (%) | |
| <18 y | 2 (6) |
| 18-64 y | 30 (88) |
| ≥65 y | 2 (6) |
| Sex, n (%) | |
| Female | 17 (50) |
| Male | 17 (50) |
| Race/ethnicity, n(%) | |
| Asian | 4 (12) |
| African American | 5 (15) |
| Hispanic | 2 (6) |
| White | 23 (68) |
| Other | 0 (0) |
| Diagnosis, n (%) | |
| MAA | 31 (91) |
| UC-erythroid (including 1 DBA) | 3 (9) |
| No. of previous treatments (%) | |
| 0 | 19 (56) |
| 1 | 9 (26) |
| >2 | 6 (18) |
| Previous treatments, n (%) | |
| Immunosuppressive therapy with ATG, ATG/CSA, or CSA | 7 (21) |
| Androgens | 6 (18) |
| Other* | 11 (32) |
| Time since first diagnosis, median (range), mo | 11 (1-354) |
| Laboratory parameters | |
| Neutrophil count, median (range), ×10−9/L | 1.1 (0.5-5.3) |
| Reticulocyte count, median (range), ×10−9/L | 62.9 (3.1-92.2) |
| Platelet count, median (range), ×109/L | 26.0 (10.0-311.0) |
| Hemoglobin, median (range), g/dL | 8.7 (6.6-15.7) |
| Erythroid response eligible (transfusion-dependent or hemoglobin <8.5 g/dL), n (%) | 23 (68) |
| Platelet response eligible (transfusion-dependent or platelets <30 × 109/L), n (%) | 24 (71) |
| Erythroid and platelet response eligible, n (%) | 13 (38) |
| PNH clone >1%, n (%) | 11 (32.) |
Includes daclizumab, methotrexate, rituximab, intravenous immunoglobulin, mycophenolate, sirolimus, erythropoietin, corticosteroids, and central venal pressure.