Table 3.
Age/ Sex |
Disease phenotype |
Length of Dx (in months) |
Indication for treatment with eculizumab |
Pre-tx Hb (g/dL) |
Post-tx Hb (g/dL) |
Pre-tx LDH (U/L) |
Post-tx LDH (U/L) |
Pre-tx Retic (K/cu mm) |
Post-tx Retic (K/cu mm) |
Pre-tx RBC clone size (%) |
Post-tx RBC clone size (%) |
Pre-tx Gran clone size (%) |
Post-tx Gran clone size (%) |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|
(A) | |||||||||||||
33 M | Classical | 13 | Anemia from hemolysis | 8.6 | 17.4 | 1670 | 274 | NA | 133.5 | 46 | 6.9 | 87 | 24 |
31 M | AA/PNH | 82 | Thrombosis, anemia from hemolysis | 7.4 | 15.6 | 771 | 311 | 82.6 | 150.7 | 73 | 11.9 | 99 | 99 |
28 M | Classical | 4 | Thrombosis, anemia from hemolysis | 10.1 | 16.2 | 888 | 261 | 110 | 109.7 | 51 | 14 | 98 | 99 |
36 M | Classical | 192 | PNH sx, Thrombosis, anemia from hemolysis | 8.4 | 15.9 | 3490 | 135 | 324.3 | 101.1 | 63 | 6 | 99 | 23 |
(B) | |||||||||||||
27 M | Classical | 2 | Anemia from hemolysis, PNH sx | 8.9 | 12.7 | 6218 | 329 | 180 | 164.6 | 6.66 | 49 | 73 | 87 |
35 F | Classical | 84 | Anemia from hemolysis, PNH sx | 6.6 | 10.3 | H | 196 | 20 | NA | 54 | NA | 99 | NA |
56 F | AA/PNH | 288 | Anemia from hemolysis, PNH sx | 8.4 | 8.9 | 2042 | 223 | 101.5 | 79.5 | 25 | 44 | 85 | 96 |
21 M | Classical | 60 | Anemia from hemolysis, PNH sx | 9.6 | 12 | 1969 | 279 | 208.9 | 162.3 | 64 | NA | 96 | NA |
31 M | Classical | 168 | Thrombosis, anemia from hemolysis | 11 | 12.81 | 1591 | 209 | 129.2 | 129.8 | 83 | 100 | 99 | 99 |
64 F | Classical | 108 | Anemia from hemolysis, PNH sx | 9.2 | 11.7 | 1583 | 247 | 161.4 | 86.5 | 86 | 99.7 | 87 | 75.9 |
24 F2 | AA/PNH | 36 | Thrombosis | 9.9 | 14.7 | 157 | 168 | 8.2 | 0.9 | 1 | 0 | 65 | 0 |
32 M | Classical | 13 | Anemia from hemolysis, PNH sx | 12.1 | 12.6 | 2555 | 240 | 299.5 | 325.1 | 28 | NA | 88 | NA |
22 F | Classical | 122 | Anemia from hemolysis, PNH sx | 5.8 | 10.9 | 924 | 195 | 266.1 | 179.8 | 77 | 94 | 92 | 99 |
18 F | Classical | 26 | Anemia from hemolysis, PNH sx | 6.3 | 10 | 1760 | 234 | 219.8 | 179.8 | 13 | 95 | 55 | 99 |
28 F | AA/PNH | 12 | Anemia from hemolysis, PNH sx | 11.1 | 9.3 | 529 | 180 | 69.2 | 68 | 34 | NA | 72 | NA |
48 M | Classical | 36 | Anemia from hemolysis, PNH sx | 6.8 | 10.3 | 2343 | 317 | 190.1 | 160.4 | 38 | 68 | 41 | 62 |
47 F | Classical | 2 | Thrombosis, anemia from hemolysis, PNH sx | 10 | 10.9 | 1440 | 268 | 160.9 | 5.1 | 43 | 55 | 90 | 91 |
18 F | Classical | 4 | Anemia from hemolysis | 5.1 | 10.0 | 1767 | NA | 169.7 | NA | 37 | 66 | 93 | 98 |
48 M | Classical | 204 | Anemia from hemolysis | 7.6 | 10.5 | 4835 | 277 | 451.8 | 321.8 | 42 | 99 | 97 | 96 |
57 M | Classical | 150 | Anemia from hemolysis | 11.1 | 10.1 | 1537 | NA | 174 | NA | 34 | NA | 90 | NA |
(C) | |||||||||||||
77 F | AA/PNH | 9 | Anemia from hemolysis | 7.8 | 11.41 | 1085 | 303 | 68.1 | 113.9 | 8 | 7.91 | 86 | 59 |
40 F | AA/PNH | 12 | Thrombosis | 8.2 | 8.2 | 622 | 252 | 112.2 | 114.8 | 9 | 97 | 66 | 97 |
16 F | Classical | 24 | Thrombosis, anemia from hemolysis | 9.2 | 9.8 | 282 | 362 | 251.1 | NA | 76 | NA | 97 | NA |
28 M | Classical | 1 | Anemia from hemolysis, PNH sx | 8.2 | 7.9 | 2023 | 1637 | NA | NA | 13 | NA | 71 | NA |
63 M | AA/PNH | 36 | Anemia from hemolysis, PNH sx | 8.5 | 8.5 | 417 | 278 | 32.6 | 28 | 7 | 4 | 58 | 72 |
35 M | AA/PNH | 2 | Thrombosis, anemia from hemolysis | 10 | 7.6 | NA | 919 | NA | 84 | 80 | 4.51 | 75 | 92 |
46 F | Classical | 3 | Thrombosis, anemia from hemolysis | 8.1 | 9.1 | 1551 | 200 | 139.7 | 222.9 | 32 | 50 | 83 | 94 |
39 M2 | Classical | 72 | Thrombosis | 9.9 | 8.1 | H | 185 | 351.4 | 340 | 99 | 86 | 99 | 100 |
63 M | Classical | 14 | Thrombosis, anemia from hemolysis | 10.4 | 10.91 | 981 | 189 | 142.8 | 187.3 | 16 | 341 | 78 | 96 |
51 F | AA/PNH | 4 | Thrombosis, anemia from hemolysis | 9.1 | 11.71 | 568 | 222 | 151.1 | 106.3 | 11 | 3.2 | 56 | 18 |
PNH, paroxysmal nocturnal hemoglobinuria; H, hemolyzed; NA, not available (for patient with treatment initiated at primary institution but followed elsewhere, only information received at time of analysis).
Values for post-treatment performed after minimum of 6 months from initiation of treatment.
Transfused value.
S/p bone marrow transplantation.