Table 1.
Patient characteristics (n=27)
| Initial PNH clone size (granulocytes) | |||||
|---|---|---|---|---|---|
| Characteristics | ≤15% | >15% and <40% | ≥40% | Total | |
| Sex | |||||
| Male, n (%) | 6 (22.2) | 2 (7.4) | 1 (3.7) | 9 (33.3) | |
| Female, n (%) | 10 (37) | 5 (18.5) | 3 (11.1) | 18(66.7) | |
| Age at diagnosis, Median (range) | |||||
| Male | 29 (20-41) | 30 (24-36) | 41 (41) | 24(20-41) | |
| Female | 21 (10-62) | 48 (15-68) | 31 (19-32) | 29(10-68) | |
| Therapy | |||||
| IST only | 1 | 1 | 0 | 2 | |
| 1ST→ HiCy | 5 | 0 | 2 | 7 | |
| HiCy only | 7 | 4 | 2 | 13 | |
| HiCy → BMT | 0 | 1 | 0 | 1 | |
| HiCy → Eculizumab | 1 | 0 | 0 | 1 | |
| No therapy | 2 | 1 | 0 | 3 | |
| Follow up years, Median (range) | 6.3 (1.5-11.5) | 3.3 (2.5-7.3) | 5.5 (3.2-9) | 5.1(1.5-11.5) | |
| PNH clone size | |||||
| Increase, n (%) | 6 (22.2) | 5(18.5) | 2 (7.4) | 13 (48.1) | |
| Decrease, n (%) | 3 (11.1) | 2 (7.4) | 2 (7.4) | 7 (26) | |
| Stable, n (%) | 7 (25.9) | 0 (0) | 0 (0) | 7 (26) | |
| Eventually developed PNH symptoms (n) | 3 | 3 | 1 | 7 | |
| Transfusion dependent | 3 | 3 | 1 | ||
| Worsening Fatigue | 1 | 3 | 0 | ||
| Abdominal pain | 1 | 0 | 0 | ||
| Hemoglobinuria | 1 | 0 | 0 | ||
| Thrombosis | 0 | 0 | 0 | ||