Clinical characteristics of patients
| . | n . | Value, n (%) . |
|---|---|---|
| Age, y | 544 | 29 (18-54) |
| Range | 2-82 | |
| Male sex | 544 | 304 (56) |
| Ethnicity | 544 | |
| White | 275 (51) | |
| African American | 120 (22) | |
| Hispanic | 109 (20) | |
| Asian | 35 (6) | |
| Other | 5 (1) | |
| Disease status | 544 | |
| Treatment naïve severe AA | 515 (95) | |
| Relapsed or refractory AA | 29 (5) | |
| Pretreatment blood values | ||
| Neutrophil count, ×109/L | 515 | 0.29 (0.09-0.51) |
| Lymphocyte count, ×109/L | 515 | 1.27 (0.92-1.63) |
| Reticulocyte count, ×109/L | 515 | 15.3 (6.5-32.0) |
| Platelet count, ×109/L | 515 | 9 (5-13) |
| Thrombopoietin, ng/mL | 140 | 2610 (2220-3080) |
| PNH clone ≥1% | 470 | 177 (38) |
| hATG-based IST | 416 | |
| hATG and CsA | 102 (25) | |
| hATG, CsA, and MMF | 103 (25) | |
| hATG, CsA, and rapamycin | 35 (8) | |
| hATG, CsA, and EPAG | 176 (42) | |
| Response to IST | 416 | |
| Overall response | 293 (70) | |
| Complete response | 101 (24) | |
| Clonal evolution | 416 | |
| High-risk clonal evolution | 31 (7) | |
| Low-risk clonal evolution | 26 (6) |
| . | n . | Value, n (%) . |
|---|---|---|
| Age, y | 544 | 29 (18-54) |
| Range | 2-82 | |
| Male sex | 544 | 304 (56) |
| Ethnicity | 544 | |
| White | 275 (51) | |
| African American | 120 (22) | |
| Hispanic | 109 (20) | |
| Asian | 35 (6) | |
| Other | 5 (1) | |
| Disease status | 544 | |
| Treatment naïve severe AA | 515 (95) | |
| Relapsed or refractory AA | 29 (5) | |
| Pretreatment blood values | ||
| Neutrophil count, ×109/L | 515 | 0.29 (0.09-0.51) |
| Lymphocyte count, ×109/L | 515 | 1.27 (0.92-1.63) |
| Reticulocyte count, ×109/L | 515 | 15.3 (6.5-32.0) |
| Platelet count, ×109/L | 515 | 9 (5-13) |
| Thrombopoietin, ng/mL | 140 | 2610 (2220-3080) |
| PNH clone ≥1% | 470 | 177 (38) |
| hATG-based IST | 416 | |
| hATG and CsA | 102 (25) | |
| hATG, CsA, and MMF | 103 (25) | |
| hATG, CsA, and rapamycin | 35 (8) | |
| hATG, CsA, and EPAG | 176 (42) | |
| Response to IST | 416 | |
| Overall response | 293 (70) | |
| Complete response | 101 (24) | |
| Clonal evolution | 416 | |
| High-risk clonal evolution | 31 (7) | |
| Low-risk clonal evolution | 26 (6) |
Values are n (%) or median (IQR).
CsA, cyclosporine; MMF, mycophenolate mofetil.