Table 2.
Clinical characteristics of patients divided according to PNH clone size on granulocytes at baseline.
| Clone size | Neg N = 1740 | 0.01–1% N = 215 | 1–10% N = 108 | 10–50% N = 59 | >50% N = 128 |
|---|---|---|---|---|---|
| Males, N (%) | 945 (54) | 114 (53) | 60 (56) | 30 (51) | 56 (44) |
| Females, N (%) | 795 (46) | 101 (47) | 48 (44) | 29 (49) | 72 (56) |
| Median age, years (IQR) | 55 (39–66) | 49 (34–63) | 48 (30–65) | 48 (28–60) | 44 (31–85) |
| MDS, N (%) | 616 (35) | 71 (33) | 32 (30) | 13 (22) | 7 (6)* |
| AA, N (%) | 175 (10) | 98 (46) | 72 (67) | 40 (68) | 47 (37) |
| MDS/AA, N (%) | 92 (5) | 8 (4) | 2 (2) | 0 (0) | 0 (0) |
| Acute leukemia, N (%) | 157 (9) | 11 (5)* | 0 (0) | 1 (2) | 0 (0) |
| Hemolytic PNH, N (%) | 0 (0) | 1 (0.5)a | 1 (0.5)a | 3 (5) | 74 (58)* |
| MPN, N (%) | 0 (0) | 8 (4) | 0 (0) | 0 (0) | 0 (0) |
| MDS/MPN, N (%) | 0 (0) | 4 (2) | 0 (0) | 0 (0) | 0 (0) |
| Isolated cytopenia, N (%) | 433 (25) | 19 (9) | 1 (1) | 1 (2) | 0 (0) |
| Isolated thrombosis, N (%) | 245 (14) | 6 (3)* | 1 (1) | 0 (0) | 0 (0) |
| Others, N (%) | 22 (2) | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
| N = 1740 | N = 215 | N = 108 | N = 59 | N = 128 | |
| Treated, N (%) | 425 (56) | 130 (68) | 81 (78) | 46 (78) | 114 (89)* |
| Thrombosis, N (%) | 315 (18) | 16 (7) | 6 (6) | 3 (5) | 27 (21)* |
| Death, N (%) | 536 (31) | 33 (15) | 12 (11) | 12 (20) | 9(7)* |
| N = 771 | N = 215 | N = 108 | N = 59 | N = 128 | |
| Hb < 100 g/L, N (%) | 299 (39) | 102 (47) | 58 (54) | 30 (51) | 67 (52) |
| PLT < 10 × 109/L, N (%) | 351 (45) | 133 (62) | 88 (82) | 45 (76)* | 43 (34) |
| ANC < 1.5 × 109/L, N (%) | 220 (29) | 65 (30) | 57 (53) | 29 (49) | 17 (13) |
| Median LDH U/L (range) | 197 (73–1520) | 222 (83–1200) | 198 (108–1158) | 266 (70–984) | 880 (97–4403)* |
Values are shown only for patients with evaluable granulocyte PNH clone size at baseline by FLAER and with available clinical and laboratory data (Negative, N = 1740, positive, N = 510).
MDS myelodysplastic syndromes, AA aplastic anemia, MDS/AA hypoplastic MDS, MPN myeloproliferative neoplasms, Hb hemoglobin, PLT platelets, ANC absolute neutrophil counts.
*p < 0.0005.
aTwo patients showed small and very small clones at baseline and then developed classic hemolytic PNH, with 15% and 20% clone size, respectively.