Table 1.
PNH patient characteristics
| Patient no. | PNH class | Previous studies | LDH pre (U / l) | LDH last FU (U / l) | Total bilirubin pre (μM) | Total bilirubin last FU (μM) | Hb pre (g / dl) | Hb last FU (g / dl) | RBC pre | RBC last FU | Ferritin pre (ng / ml) | Ferritin last FU (ng / ml) | C3c / C3d+ pre | C3c / C3d+ last FU | Therapy duration (months) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 01 | 2a | S | 2944 | 226 | 140·0 | 77·0 | 7·9 | 10·7 | Yes | No | 28 | 213 | Neg | C3d+ | 60 |
| 02 | 1 | S | 2703 | 770 | 12·8 | 17· 1 | 10·7 | 12·5 | Yes | No | 27 | 441 | Neg | C3d+ | 60 |
| 03 | 1 | S | 3048 | 223 | n.a | n.a | n.a | 10·3 | Yes | Yes (reg) | 2245 | 4196 | Neg | C3d+ | 59 |
| 04 | 2a | T | 2788 | 223 | 44·0 | 60·5 | 7·8 | 11· 1 | Yes | No | 115 | 888 | Neg | C3d+ | 60 |
| 05 | 1 | N | 456 | 479 | 18·8 | 27·4 | 14·4 | 14·9 | No | No | 38 | 138 | Neg | n.a. | 19 |
| 06 | 2a | T | 3057 | 222 | 30·6 | 30·0 | 8·9 | 8·5 | Yes | Yes (int) | 477 | 1935 | Neg | C3c+, C3d+ | 61 |
| 07 | 1 | S | 3569 | 235 | 44·5 | 30·8 | 9·7 | 11·6 | Yes | Yes (int) | 124 | 606 | Neg | C3d+ | 60 |
| 08 | 2a | S | 1657 | 115 | 30·0 | 29·1 | 11·0 | 10·7 | Yes | No | 63 | 1001 | Neg | C3d+ | 55 |
| 09 | 2a | N | 3014 | 212 | 45·0 | 18·3 | 10·0 | 11·0 | Yes | No | 91 | 1300 | Neg | n.a. | 30 |
| 10 | 2a | N | 360 | 174 | 87·2 | 34·0 | 12·0 | 13·5 | Yes | No | 17 | 318 | n.a. | Neg | 30 |
| 11 | 1 | N | 2248 | 333 | 25·0 | 33·0 | 8·1 | 9·7 | Yes | No | 27 | n.a | Neg | n.a. | 3 |
| 12 | 2a | T | 1585 | 225 | 18·0 | 29·1 | 10·5 | 8·7 | Yes | Yes (int) | 18 | 168 | n.a. | C3d+ | 52 |
| 13 | 1 | N | 5191 | 307 | 50·0 | 30·8 | 7·5 | 10·8 | Yes | Yes (int) | 56 | 774 | Neg | C3d+ | 29 |
| 14 | 1 | N | 2274 | 246 | 33·3 | 35·6 | 13·0 | 9·5 | Yes | No | 75 | 378 | Neg | C3d+ | 23 |
| 15 | 1 | N | 1557 | 341 | 5·1 | 14·2 | 12·1 | 12·7 | Yes | No | 91 | 129 | Neg | n.a. | 26 |
| 16 | 2a | N | 660 | 93 | 34·0 | 26·0 | 9·1 | 8·3 | Yes | Yes (reg) | 127 | 698 | Neg | Neg | 12 |
| 17 | 1 | N | 2464 | 246 | 30·8 | 30·8 | 9·4 | 8·8 | Yes | Yes (int) | 104 | n.a | Neg | Pos | 25 |
| 18 | 1 | N | 2456 | 292 | 77·0 | 11·3 | 5·3 | 10·2 | Yes | No | 14 | n.a | Neg | Pos | 24 |
| 19 | 1 | N | 2724 | 366 | 32·5 | 56·4 | 9·1 | 10·8 | Yes | No | 163 | 145 | Neg | C3d+ | 31 |
| 20 | 2a | N | 910 | 275 | n.a | 12·0 | 5·8 | 7·6 | No | Yes | 135 | N.a | n.a. | n.a. | 5 |
| 21 | 2b | N | 1883 | 1829 | n.a | 42·8 | 7·0 | 8·1 | Yes | Yes (reg) | 15 | 68 | Neg | Neg | 2 |
| 22 | 1 | N | 551 | 136 | 10·3 | 5·1 | 10·1 | 12·0 | No | No | 17 | 46 | Neg | Neg | 17 |
| 23 | 2a | N | 975 | 207 | 31·3 | 35·7 | 12·8 | 12·5 | No | No | 41 | 39 | Neg | n.a. | 2 |
| 24 | 1 | N | 1012 | 258 | 26·0 | 49·0 | 8·9 | 12·5 | Yes | No | 11 | 166 | Neg | C3d+ | 26 |
| 25 | 2a | N | 1544 | 292 | 20·0 | 47·9 | 10·0 | 9·9 | Yes | No | 100 | 49 | C3d+ | Neg | 25 |
| 26 | 2a | N | 840 | 350 | 9·7 | 7·5 | 7·0 | 8·5 | Yes | Yes (reg) | n.a | 1883 | n.a. | Neg | 7 |
| 27 | 1 | N | 2814 | 168 | 40·0 | 27·0 | 5·0 | 11·3 | Yes | No | 18 | 182 | n.a. | Pos | 30 |
| 28 | 2a | T | 3770 | 293 | 54·7 | 20·5 | 7·8 | 9·2 | Yes | Yes (reg) | 100 | 986 | Neg | C3d+ | 63 |
| 29 | 1 | N | 723 | 201 | 29·8 | 22·8 | 6·0 | 9·3 | Yes | No | 76 | 1798 | n.a. | n.a. | 4 |
| 30 | 2a | N | 436 | 216 | 22·2 | 22·2 | 9·5 | 10·5 | Yes | Yes (reg) | 3821 | 3700 | Neg | n.a. | 1 |
| 31 | 2a | N | 787 | 228 | 38·0 | n.a | 12·3 | 13·3 | Yes | No | 172 | 116 | Neg | n.a. | 19 |
| 32 | 1 | N | 2283 | 339 | n.a | 17· 1 | 9·2 | 9·1 | Yes | No | 14 | 244 | Neg | C3d+ | 24 |
| 33 | 1 | N | 609 | 256 | 28·0 | 24·0 | 12·8 | 12·1 | No | No | 53 | 76 | Neg | C3d+ | 34 |
| 34 | 1 | N | 2014 | 268 | 29·1 | 34·2 | 8·7 | 12·0 | No | No | 30 | 67 | Neg | C3d+ | 8 |
| 35 | 1 | N | 472 | 205 | 14·0 | 18·8 | 13·9 | 13·6 | No | No | 41 | n.a | Neg | n.a. | 8 |
| 36 | 1 | N | 3793 | 309 | 30·8 | 25·7 | 11·3 | 9·9 | Yes | No | 245 | 309 | Neg | C3d+ | 7 |
| 37 | 1 | N | 1331 | 266 | 15·4 | 12·0 | 7·2 | 6·9 | No | Yes | 22 | 572 | Neg | Neg | 4 |
| 38 | 1 | N | 344 | 309 | 6·8 | 35·9 | 9·2 | 8·8 | Yes | Yes (reg) | 688 | N.a | Neg | C3d+ | 6 |
| 39 | 2a | N | 1186 | 277 | 10·0 | 30·0 | 9·1 | 10·1 | No | No | 32 | 872 | Neg | n.a. | 15 |
| 40 | 2a | N | 1070 | 298 | 28·4 | 22·6 | 9·5 | 9·5 | Yes | Yes (reg) | 1493 | 3347 | Neg | n.a. | 7 |
| 41 | 2b | N | 2722 | 1770 | 12·0 | 10·3 | 6·4 | 5·9 | Yes | Yes (reg) | 1692 | n.a | C3c+ | Neg | 2 |
Pre-eculizumab = last follow-up before eculizumab start, n. a., not available; LDH, lactate dehydrogenase; Hb, haemoglobin; RBC, red-blood-cell; int, intermittent (RBC transfusions with a time interval > every 12 weeks); reg, regular (RBC transfusions with a time interval > every 12 weeks); FU, last follow-up during eculizumab; Neg, negative; Pos, positive.
PNH classification: 1 = classical PNH; 2 = PNH in the context of another defined bone marrow disorder, either AA (2a) or myeloproliferative syndrome (2b).
Previous enrolled in the TRIUMPH (=T) or the SHEPHERD (=S) trial. N = have not been reported before.
Two patients had additional anti-erythrocytic alloantibodies or warm autoantibodies.