Pt. # | Gender | Genotype | Body wt. (kg) | Tx duration (days) | Liver (MN) | Liver volume (mL) | Spleen (MN) | Spleen volume (mL) | Hb (g/dL) | Platelets (x 109/l) | Chito nmol/(mL*hr) | ||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
 |  |  | BL | EOT |  | BL | EOT | BL | EOT | BL | EOT | BL | EOT | BL | EOT | BL | EOT | BL | EOT |
1 | M | N370S/unknown | 102 | – | 71 | 0.55 | – | 1420 | – | 3.40 | – | 696 | – | 15.1 | – | 127 | – | 159 | – |
2†,Ŧ | M | N370S/IVS 2 + 1 | 79.4 | 79.4 | 148 | – | 0.92 | – | 1833 | – | 5.52 | – | 876 | 15.4 | 15.6 | 93 | 95 | 781 | 1396 |
3†| F | R496H/G202R | 59.8 | 57.4 | 734 | 1.05 | 0.94 | 1564 | 1345 | SPX | – | SPX | – | 13.8 | 13.4 | 613 | 521 | 2915 | 1881 |
4†| F | N370S/L444P | 126 | 86.8 | 716 | 0.64 | 0.87 | 1999 | 1890 | SPX | – | SPX | – | 12.9 | 14.4 | 437 | 311 | – | 151 |
5†,Ŧ | F | N370S/unknown | 69.7 | 62.8 | 408 | 0.85 | 0.85 | 1474 | 1333 | 2.66 | 3.54 | 371 | 445 | 14.0 | 12.1 | 233 | 148 | 974 | 3456 |
6 | F | N370S/L444P | 86.3 | – | 75 | 0.73 | – | 1582 | – | 2.94 | – | 507 | – | 16.8 | 16.9 | 173 | 173 | 1165 | – |
7†| M | N370S/L444P | 85.0 | 79.5 | 663 | 0.73 | 0.80 | 1545 | 1593 | 2.19 | 2.68 | 373 | 425 | 15.5 | 15.6 | 162 | 127 | 4729 | 1808 |
8†,Ŧ | F | N370S/EXG DEL G | 48.0 | 46.0 | 245 | 1.49 | 1.54 | 1785 | 1774 | SPX | – | SPX | – | 12.9 | 12.3 | 161 | 195 | 2887 | 5196 |
9†| M | N370S/L444P | 91.0 | 85.0 | 732 | 0.98 | 0.89 | 2224 | 1891 | 9.90 | 10.4 | 1801 | 1761 | 14.3 | 14.0 | 100 | 98 | 1619 | 3139 |
10 | M | N370S/N370S | 71.0 | – | 50 | – | – | – | – | – | – | – | – | 15.3 | – | 139 | – | – | – |
11 | M | N370S/N370S | 70.5 | 69.7 | 729 | 0.98 | 0.92 | 1729 | 1600 | – | 3.12 | – | 435 | 14.0 | 12.7 | 167 | 100 | – | 2156 |
12†,Ŧ | F | N370S/T134P | 57.0 | 46.7 | 373 | 0.77 | 0.80 | 1100 | 935 | SPX | – | SPX | – | 13.9 | 11.6 | 271 | 127 | – | 862 |
13 | M | N370S/N370S | 76.3 | – | 3 | 0.94 | – | 1798 | – | 2.21 | – | 338 | – | 16.1 | – | 187 | – | 53 | – |
14†,Ŧ | M | N370S/K198E | 70.1 | 70.1 | 190 | 1.04 | 1.11 | 1827 | 1938 | 2.72 | 3.51 | 382 | 492 | 15.2 | 12.2 | 160 | 150 | – | 4978 |
15†,Ŧ | M | N370S/84GG | 83.6 | 75.3 | 515 | 1.49 | 1.20 | 3112 | 2258 | 4.66 | 3.77 | 779 | 568 | 13.3 | 12.4 | 199 | 149 | – | 20 |
16*,†,Ŧ | M | N370S/S107L | 78.5 | 72.3 | 405 | 0.83 | 0.91 | 1627 | 1637 | SPX | – | SPX | – | 13.4 | 11.6 | 283 | 188 | 420 | 3645 |
17 | F | N370S/D409H | 81.2 | – | 109 | 1.02 | – | 2062 | – | 4.30 | – | 699 | – | 13.4 | – | 106 | – | 839 | – |
18*,†| F | N370S/L444P | 59.2 | 57.4 | 741 | 1.33 | 1.19 | 1964 | 1715 | 2.91 | 3.11 | 344 | 357 | 13.5 | 12.1 | 177 | 99 | 1232 | 4113 |
19†| M | N370S/84GG | 74.7 | 69.6 | 743 | 0.78 | 0.72 | 1463 | 1254 | SPX | – | SPX | – | 17.0 | 13.1 | 225 | 155 | 1336 | 4200 |
20Ŧ | F | N37O/NK | 51.5 | – | 112 | 1.04 | – | 1341 | – | 3.11 | – | 320 | – | 16.8 | 16.8 | 183 | 152 | 591 | 695 |
21†| F | N370S/N370S | 73.5 | 69.0 | 729 | 0.66 | 0.67 | 1220 | 1148 | 1.44 | 1.29 | 211 | 179 | 13.8 | 13.3 | 160 | 153 | 80 | 41 |
22*,†| F | N370S/crossover | 62.5 | 58.2 | 755 | 1.00 | 1.16 | 1562 | 1687 | 3.57 | 5.11 | 447 | 595 | 16.4 | 15.4 | 167 | 111 | 2326 | 2969 |
23†| F | N370S/unknown | 82.5 | 68.5 | 718 | 0.93 | 1.00 | 1925 | 1706 | 5.03 | 6.40 | 830 | 877 | 16.6 | 16.1 | 104 | 91 | 389 | 4058 |
24†| F | N370S/N370S | 67.7 | 65.9 | 740 | 0.82 | 0.86 | 1391 | 1416 | 2.00 | 1.80 | 270 | 238 | 16.4 | 14.6 | 226 | 191 | 27 | 17 |
25*,†| M | N370S/1327TT | 72.0 | 72.0 | 754 | 1.22 | 1.22 | 2191 | 2204 | 4.51 | 6.23 | 650 | 897 | 15.0 | 14.8 | 152 | 79 | 2658 | 8348 |
26*,†| F | N370S/REC NEIL | 61.0 | 51.0 | 732 | 0.95 | 1.06 | 1444 | 1349 | 1.61 | 2.84 | 197 | 289 | 14.8 | 13.4 | 186 | 113 | 666 | 3500 |
27*,†,Ŧ | F | N370S/L444P | 55.9 | 56.8 | 478 | 1.12 | 1.25 | 1571 | 1770 | 4.01 | 5.08 | 449 | 577 | 13.6 | 10.8 | 148 | 99 | 1872 | 2897 |
28†,Ŧ | M | N370S/1035INSG | 93.7 | 87.7 | 545 | 1.02 | 1.15 | 2391 | 2530 | 1.43 | 1.94 | 268 | 340 | 11.3 | 11.1 | 151 | 155 | 5863 | 6772 |
29*,†| M | ND | 91.2 | 87.3 | 653 | 0.81 | 1.05 | 1847 | 2291 | 6.67 | 11.1 | 1217 | 1939 | 15.0 | 15.1 | 98 | 54 | 241 | 1703 |
30*,†| M | ND | 86.1 | 84.8 | 765 | 0.81 | 0.97 | 1745 | 2063 | 2.40 | 4.07 | 413 | 690 | 16.8 | 14.7 | 158 | 116 | 3398 | 7138 |
31†| M | N370S/N370S | 95.9 | 82.3 | 734 | 0.82 | 0.90 | 1964 | 1854 | SPX | – | SPX | – | 14.8 | 14.4 | 264 | 251 | – | 110 |
32†,Ŧ | M | N370S/8499 | 71.2 | 79.8 | 239 | 0.92 | 0.75 | 1638 | 1496 | 2.18 | 2.18 | 311 | 348 | 14.9 | 14.1 | 186 | 128 | 391 | 590 |
33†| F | N370S/R257X | 124 | 109 | 738 | 1.05 | 0.88 | 3274 | 2400 | SPX | – | SPX | – | 17.8 | 13.9 | 229 | 219 | 44 | 1071 |
34*,†| F | N370S/unidentified | 113 | 109 | 725 | 0.63 | 0.64 | 1788 | 1733 | 1.74 | 2.96 | 394 | 642 | 13.6 | 14.6 | 223 | 135 | 1587 | 4118 |
35*,†| F | N370S/unidentified | 89.2 | 68.8 | 725 | 0.52 | 0.85 | 1152 | 1468 | 2.23 | 3.24 | 398 | 446 | 15.3 | 14.8 | 149 | 102 | 132 | 1002 |
36*,†| M | N370S/unidentified | 70.3 | 68.9 | 725 | 0.97 | 1.09 | 1704 | 1880 | 2.94 | 4.34 | 413 | 598 | 14.2 | 14.6 | 177 | 158 |  | 13834 |
37Ŧ | M | N370S/unknown | 76.0 | – | 140 | 0.75 | – | 1434 | – | 2.11 | – | 320 | – | 14.7 | 14.4 | 141 | 141 | 438 | 510 |
38*,†| F | 84GG/N370S | 65.0 | 62.5 | 732 | 0.72 | 0.92 | 1162 | 1439 | 3.24 | 3.72 | 422 | 465 | 16.8 | 15.1 | 136 | 106 | 98 | 1271 |
39*,†| M | N370S/84GG | 88.0 | 81.0 | 730 | 0.88 | 1.11 | 1932 | 2248 | SPX | – | SPX | – | 17.1 | 14.8 | 254 | 174 | 1940 | 10636 |
40 | M | L444P/NA | 54.4 | – | 22 | 0.81 | – | 1095 | – | 2.18 | – | 238 | – | 16.2 | – | 134 | – | – | – |
41 | M | N370S/N370S | 76.7 | – | 15 | 0.80 | – | 1538 | – | 1.37 | – | 210 | – | 15.9 | – | 232 | – | – | 38 |
42†,Ŧ | F | N370S/N370S | 58.5 | 58.0 | 231 | 1.01 | 0.98 | 1471 | 1424 | 2.35 | 2.54 | 275 | 294 | 12.6 | 12.9 | 214 | 207 | 17 | 21 |